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GLASSIA Safety, Immunogenicity, and Bronchoalveolar Lavage Study

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02525861
Recruitment Status : Completed
First Posted : August 18, 2015
Results First Posted : October 14, 2021
Last Update Posted : October 14, 2021
Sponsor:
Information provided by (Responsible Party):
Takeda ( Baxalta now part of Shire )

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Alpha1-antitrypsin Deficiency
Intervention Biological: GLASSIA
Enrollment 34
Recruitment Details The study was conducted at 21 centers in the United States and 1 center in Canada between 08 Mar 2016 (first participant first visit) and 29 Jul 2020 (last participant last visit).
Pre-assignment Details A total of 34 participants were randomized and received the study treatment.
Arm/Group Title Cohort I: GLASSIA (High-end) Cohort II: GLASSIA (Low-end)
Hide Arm/Group Description Participants received weekly intravenous (IV) infusions of GLASSIA (lot with particle loads representing the high end within the normal range) at 60 milligrams per kilogram (mg/kg) body weight (BW) active Alpha1-Proteinase Inhibitor (A1PI) protein at a rate of 0.2 milliliters per kilogram per minute (ml/kg/min) for 25 weeks (25 planned infusions). Participants received weekly IV infusions of GLASSIA (lot with particle loads representing the low end within the normal range) at 60 mg/kg BW active A1PI protein at a rate of 0.2 ml/kg/min for 25 weeks (25 planned infusions).
Period Title: Overall Study
Started 18 16
Completed 17 14
Not Completed 1 2
Reason Not Completed
Adverse Event             1             0
Withdrawal by Subject             0             1
Physician Decision             0             1
Arm/Group Title Cohort I: GLASSIA (High-end) Cohort II: GLASSIA (Low-end) Total
Hide Arm/Group Description Participants received weekly intravenous (IV) infusions of GLASSIA (lot with particle loads representing the high end within the normal range) at 60 milligrams per kilogram (mg/kg) body weight (BW) active Alpha1-Proteinase Inhibitor (A1PI) protein at a rate of 0.2 milliliters per kilogram per minute (ml/kg/min) for 25 weeks (25 planned infusions). Participants received weekly IV infusions of GLASSIA (lot with particle loads representing the low end within the normal range) at 60 mg/kg BW active A1PI protein at a rate of 0.2 ml/kg/min for 25 weeks (25 planned infusions). Total of all reporting groups
Overall Number of Baseline Participants 18 16 34
Hide Baseline Analysis Population Description
Safety analysis set consisted of all enrolled participants who received any amount of investigational product (IP), regardless of protocol deviations or non-adherence to study procedures.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 18 participants 16 participants 34 participants
57.9  (8.95) 58.9  (9.32) 58.4  (9.00)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 18 participants 16 participants 34 participants
Female
8
  44.4%
6
  37.5%
14
  41.2%
Male
10
  55.6%
10
  62.5%
20
  58.8%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 18 participants 16 participants 34 participants
Hispanic or Latino
1
   5.6%
2
  12.5%
3
   8.8%
Not Hispanic or Latino
17
  94.4%
14
  87.5%
31
  91.2%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 18 participants 16 participants 34 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
White
18
 100.0%
16
 100.0%
34
 100.0%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
1.Primary Outcome
Title Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Potentially Related to Presence of Particle Load in the GLASSIA Solution
Hide Description An Adverse Events (AEs) was defined as any untoward medical occurrence in a participant administered an IP that does not necessarily have a causal relationship with the treatment. Any AE that occurs on or after the first dose of IP infusion will be considered a TEAE. A TEAE that is considered potentially related to the presence of protein aggregates (particle load) in the GLASSIA solution is defined as any embolic or thrombotic event. Number of participants with TEAEs potentially related to the presence of protein aggregates (particle load) in the GLASSIA solution were reported.
Time Frame From start of study treatment up to Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
Safety analysis set consisted of all enrolled participants who received any amount of IP, regardless of protocol deviations or non-adherence to study procedures.
Arm/Group Title Cohort I: GLASSIA (High-end) Cohort II: GLASSIA (Low-end)
Hide Arm/Group Description:
Participants received weekly intravenous (IV) infusions of GLASSIA (lot with particle loads representing the high end within the normal range) at 60 milligrams per kilogram (mg/kg) body weight (BW) active Alpha1-Proteinase Inhibitor (A1PI) protein at a rate of 0.2 milliliters per kilogram per minute (ml/kg/min) for 25 weeks (25 planned infusions).
Participants received weekly IV infusions of GLASSIA (lot with particle loads representing the low end within the normal range) at 60 mg/kg BW active A1PI protein at a rate of 0.2 ml/kg/min for 25 weeks (25 planned infusions).
Overall Number of Participants Analyzed 18 16
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
2.Primary Outcome
Title Number of Participants With Treatment-Emergent Adverse Reactions (ARs) Plus Suspected Adverse Reactions (ARs) Within 24 Hours Following the End of IP Infusion
Hide Description An Treatment-emergent AR and suspected AR was any TEAE which met any of the following criteria: a; A TEAE that began during infusion or within 24 hours (or 1 day where time of onset is not available) following the end of IP infusion, or b; A TEAE considered by either the investigator and/or the sponsor to be possibly or probably related to IP administration, or c; A TEAE for which causality assessment was missing or indeterminate. Number of participants with both treatment-emergent ARs plus suspected ARs were collectively reported.
Time Frame From start of study treatment up to 24 hours post infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Safety analysis set consisted of all enrolled participants who received any amount of IP, regardless of protocol deviations or non-adherence to study procedures.
Arm/Group Title Cohort I: GLASSIA (High-end) Cohort II: GLASSIA (Low-end)
Hide Arm/Group Description:
Participants received weekly intravenous (IV) infusions of GLASSIA (lot with particle loads representing the high end within the normal range) at 60 milligrams per kilogram (mg/kg) body weight (BW) active Alpha1-Proteinase Inhibitor (A1PI) protein at a rate of 0.2 milliliters per kilogram per minute (ml/kg/min) for 25 weeks (25 planned infusions).
Participants received weekly IV infusions of GLASSIA (lot with particle loads representing the low end within the normal range) at 60 mg/kg BW active A1PI protein at a rate of 0.2 ml/kg/min for 25 weeks (25 planned infusions).
Overall Number of Participants Analyzed 18 16
Measure Type: Count of Participants
Unit of Measure: Participants
7
  38.9%
3
  18.8%
3.Primary Outcome
Title Number of Participants With Treatment-Emergent Adverse Reactions (ARs) Plus Suspected Adverse Reactions (ARs) Within 72 Hours Following the End of IP Infusion
Hide Description An Treatment-emergent AR and suspected AR was any TEAE which met any of the following criteria: a; A TEAE that began during infusion or within 72 hours (or 3 days where time of onset is not available) following the end of IP infusion, or b; A TEAE considered by either the investigator and/or the sponsor to be possibly or probably related to IP administration, or c; A TEAE for which causality assessment was missing or indeterminate. Number of participants with both treatment-emergent ARs plus suspected ARs were collectively reported.
Time Frame From start of study treatment up to 72 hours post infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Safety analysis set consisted of all enrolled participants who received any amount of IP, regardless of protocol deviations or non-adherence to study procedures.
Arm/Group Title Cohort I: GLASSIA (High-end) Cohort II: GLASSIA (Low-end)
Hide Arm/Group Description:
Participants received weekly intravenous (IV) infusions of GLASSIA (lot with particle loads representing the high end within the normal range) at 60 milligrams per kilogram (mg/kg) body weight (BW) active Alpha1-Proteinase Inhibitor (A1PI) protein at a rate of 0.2 milliliters per kilogram per minute (ml/kg/min) for 25 weeks (25 planned infusions).
Participants received weekly IV infusions of GLASSIA (lot with particle loads representing the low end within the normal range) at 60 mg/kg BW active A1PI protein at a rate of 0.2 ml/kg/min for 25 weeks (25 planned infusions).
Overall Number of Participants Analyzed 18 16
Measure Type: Count of Participants
Unit of Measure: Participants
10
  55.6%
7
  43.8%
4.Primary Outcome
Title Number of Infusions Discontinued, Slowed, or Interrupted Due to TEAEs
Hide Description An AE was defined as any untoward medical occurrence in a participant administered an IP that does not necessarily have a causal relationship with the treatment. Infusions may be interrupted or discontinued in an individual participant in the event of intolerable moderate to severe infusion-related AEs and/or at the discretion of the investigator. Number of infusions that are discontinued, slowed, or interrupted due to TEAEs were reported.
Time Frame From start of study treatment up to Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
Safety analysis set consisted of all enrolled participants who received any amount of IP, regardless of protocol deviations or non-adherence to study procedures.
Arm/Group Title Cohort I: GLASSIA (High-end) Cohort II: GLASSIA (Low-end)
Hide Arm/Group Description:
Participants received weekly intravenous (IV) infusions of GLASSIA (lot with particle loads representing the high end within the normal range) at 60 milligrams per kilogram (mg/kg) body weight (BW) active Alpha1-Proteinase Inhibitor (A1PI) protein at a rate of 0.2 milliliters per kilogram per minute (ml/kg/min) for 25 weeks (25 planned infusions).
Participants received weekly IV infusions of GLASSIA (lot with particle loads representing the low end within the normal range) at 60 mg/kg BW active A1PI protein at a rate of 0.2 ml/kg/min for 25 weeks (25 planned infusions).
Overall Number of Participants Analyzed 18 16
Measure Type: Number
Unit of Measure: Number of Infusions
Number of Infusions Discontinued due to TEAEs 0 0
Number of Infusions Slowed due to TEAEs 1 0
Number of Infusions Interrupted due to TEAEs 0 0
5.Primary Outcome
Title Number of Participants Who Developed Binding and/or Neutralizing Anti- Alpha1-Proteinase Inhibitor(A1PI) Antibodies
Hide Description Development of Binding/Neutralizing Anti-A1PI Antibodies=Negative or missing at Baseline and confirmed positive at any post-infusion time point. Participants that had a positive result at Baseline and missing at all post-infusion time points were included as "No Development". Neutralizing anti-A1PI antibodies were only assessed in case of positive binding anti-A1PI antibodies. Anti-A1PI antibodies was detected using validated binding and neutralizing anti-A1PI antibody assays at a qualified immunoassay laboratory. Number of participants who developed binding and/or neutralizing anti-A1PI antibodies were reported.
Time Frame From start of study treatment up to Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
Safety analysis set consisted of all enrolled participants who received any amount of IP, regardless of protocol deviations or non-adherence to study procedures. Here, number analyzed signifies participants who were evaluable for this outcome measure at specific category.
Arm/Group Title Cohort I: GLASSIA (High-end) Cohort II: GLASSIA (Low-end)
Hide Arm/Group Description:
Participants received weekly intravenous (IV) infusions of GLASSIA (lot with particle loads representing the high end within the normal range) at 60 milligrams per kilogram (mg/kg) body weight (BW) active Alpha1-Proteinase Inhibitor (A1PI) protein at a rate of 0.2 milliliters per kilogram per minute (ml/kg/min) for 25 weeks (25 planned infusions).
Participants received weekly IV infusions of GLASSIA (lot with particle loads representing the low end within the normal range) at 60 mg/kg BW active A1PI protein at a rate of 0.2 ml/kg/min for 25 weeks (25 planned infusions).
Overall Number of Participants Analyzed 18 16
Measure Type: Count of Participants
Unit of Measure: Participants
Number of participants with binding Anti-A1PI antibodies Number Analyzed 18 participants 16 participants
1
   5.6%
1
   6.3%
Number of participants with neutralizing Anti-A1PI antibodies Number Analyzed 2 participants 1 participants
1
  50.0%
1
 100.0%
6.Primary Outcome
Title Change From Baseline in Antigenic Alpha1-Proteinase Inhibitor (A1PI) Levels in Epithelial Lining Fluid (ELF)
Hide Description Change from baseline in antigenic A1PI levels in ELF up to Week 14 was reported. Bronchoalveolar Lavage (BAL) procedures were performed at baseline and on-treatment BAL visit during GLASSIA augmentation therapy. Data for this outcome measure was analyzed and planned to be reported based on overall arm (Arms/Groups were combined as pre-specified in the study protocol).
Time Frame Baseline up to Week 14
Hide Outcome Measure Data
Hide Analysis Population Description
BAL analysis set included a subset of the full analysis set (FAS). FAS included all enrolled participants who received at least 1 IP infusion and have at least 1 available antigenic or functional A1PI measurement (either from plasma or from ELF) during the treatment period.
Arm/Group Title All GLASSIA 60 mg/kg/Week
Hide Arm/Group Description:
All participants who received weekly IV infusions of GLASSIA (lot with particle loads representing the High and low end within the normal range) at 60 mg/kg BW active A1PI protein at a rate of 0.2 ml/kg/min for 25 weeks (25 planned infusions).
Overall Number of Participants Analyzed 16
Median (Full Range)
Unit of Measure: Micromolar (mcM)
0.515
(0.08 to 2.41)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection All GLASSIA 60 mg/kg/Week
Comments Statistical analysis was collected and assessed based on A1P1 Levels at baseline and On-treatment BAL visit.
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments [Not Specified]
Method Mixed-effects model
Comments [Not Specified]
Method of Estimation Estimation Parameter Geometric Mean Ratio
Estimated Value 5.398
Estimation Comments [Not Specified]
7.Primary Outcome
Title Change From Baseline in Functional Alpha1-Proteinase Inhibitor(A1PI) Levels in Epithelial Lining Fluid (ELF)
Hide Description Change from baseline in functional A1PI (also known as Anti-Neutrophil Elastase Capacity [ANEC]) levels in ELF up to Week 14 was reported. BAL procedures were performed at baseline and on-treatment BAL visit during GLASSIA augmentation therapy. Data for this outcome measure was analyzed and planned to be reported based on overall arm (Arms/Groups were combined as pre-specified in the study protocol).
Time Frame Baseline up to Week 14
Hide Outcome Measure Data
Hide Analysis Population Description
BAL analysis set included a subset of the FAS. FAS included all enrolled participants who received at least 1 IP infusion and have at least 1 available antigenic or functional A1PI measurement (either from plasma or from ELF) during the treatment period.
Arm/Group Title All GLASSIA 60 mg/kg/Week
Hide Arm/Group Description:
All participants who received weekly IV infusions of GLASSIA (lot with particle loads representing the High and low end within the normal range) at 60 mg/kg BW active A1PI protein administered at a rate of 0.2 ml/kg/min for 25 weeks (25 planned infusions).
Overall Number of Participants Analyzed 16
Median (Full Range)
Unit of Measure: mcM
0.256
(-0.28 to 1.54)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection All GLASSIA 60 mg/kg/Week
Comments Statistical analysis was collected and assessed based on functional A1P1 Levels at baseline and On-treatment BAL visit.
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments [Not Specified]
Method mixed-effects model
Comments [Not Specified]
Method of Estimation Estimation Parameter Geometric Mean Ratio
Estimated Value 2.259
Estimation Comments [Not Specified]
8.Secondary Outcome
Title Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Hide Description An AEs was defined as any untoward medical occurrence in a participant administered an IP that does not necessarily have a causal relationship with the treatment. Any AE that occurs on or after the first dose of IP infusion will be considered a TEAE.
Time Frame From start of study treatment up to Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
Safety analysis set consisted of all enrolled participants who received any amount of IP, regardless of protocol deviations or non-adherence to study procedures.
Arm/Group Title Cohort I: GLASSIA (High-end) Cohort II: GLASSIA (Low-end)
Hide Arm/Group Description:
Participants received weekly intravenous (IV) infusions of GLASSIA (lot with particle loads representing the high end within the normal range) at 60 milligrams per kilogram (mg/kg) body weight (BW) active Alpha1-Proteinase Inhibitor (A1PI) protein at a rate of 0.2 milliliters per kilogram per minute (ml/kg/min) for 25 weeks (25 planned infusions).
Participants received weekly IV infusions of GLASSIA (lot with particle loads representing the low end within the normal range) at 60 mg/kg BW active A1PI protein at a rate of 0.2 ml/kg/min for 25 weeks (25 planned infusions).
Overall Number of Participants Analyzed 18 16
Measure Type: Count of Participants
Unit of Measure: Participants
13
  72.2%
9
  56.3%
9.Secondary Outcome
Title Number of Participants Who Experienced a Shift From Normal or Clinically Non-significant Abnormal Laboratory Values at Baseline to Clinically Significant Abnormal Laboratory Values at Week 13, 25, and 26
Hide Description Clinical laboratory values included Hematology (hemoglobin, leukocytes, neutrophils, reticulocytes/erythrocytes [Ret/Ery], platelets); Chemistry (sodium, potassium, albumin, alanine aminotransferase [AA], aspartate aminotransferase [ASA], alkaline phosphatase [AP], lactate dehydrogenase [LD], gamma glutamyl transferase [GGT], bilirubin, direct bilirubin [DB], creatinine, creatine kinase [CK], glucose); Urinalysis (erythrocytes urine [EU], protein urine [PU], specific gravity [SG], pH) and Immunology (Complement C3, Complement C4). Assessment if a value was normal, clinically non-significant abnormal, or clinically significant abnormal was done by the investigator. Number of participants who experienced a shift from normal or clinically non-significant (NCS) abnormal laboratory values at baseline [BL] to clinically significant (CS) abnormal laboratory values at Week 13, 25 and 26 were reported.
Time Frame Baseline, Week 13, 25 and 26
Hide Outcome Measure Data
Hide Analysis Population Description
Safety analysis set consisted of all enrolled participants who received any amount of IP, regardless of protocol deviations or non-adherence to study procedures. Here, number of participants analyzed signifies participants who were evaluable for this outcome measure and number analyzed signifies participants who were evaluable for at specific time point.
Arm/Group Title Cohort I: GLASSIA (High-end) Cohort II: GLASSIA (Low-end)
Hide Arm/Group Description:
Participants received weekly intravenous (IV) infusions of GLASSIA (lot with particle loads representing the high end within the normal range) at 60 milligrams per kilogram (mg/kg) body weight (BW) active Alpha1-Proteinase Inhibitor (A1PI) protein at a rate of 0.2 milliliters per kilogram per minute (ml/kg/min) for 25 weeks (25 planned infusions).
Participants received weekly IV infusions of GLASSIA (lot with particle loads representing the low end within the normal range) at 60 mg/kg BW active A1PI protein at a rate of 0.2 ml/kg/min for 25 weeks (25 planned infusions).
Overall Number of Participants Analyzed 17 15
Measure Type: Count of Participants
Unit of Measure: Participants
Hemoglobin: Normal at BL→Abnormal-CS at Week 13 Number Analyzed 14 participants 14 participants
0 0
Hemoglobin: Abnormal-NCS at BL→Abnormal-CS at Week 13 Number Analyzed 14 participants 14 participants
0 0
Hemoglobin: Normal at BL→Abnormal-CS at Week 25 Number Analyzed 16 participants 14 participants
0 0
Hemoglobin: Abnormal-NCS at BL→Abnormal-CS at Week 25 Number Analyzed 16 participants 14 participants
0 0
Hemoglobin: Normal at BL→Abnormal-CS at Week 26 Number Analyzed 17 participants 12 participants
0 0
Hemoglobin: Abnormal-NCS at BL→Abnormal-CS at Week 26 Number Analyzed 17 participants 12 participants
0 0
Leukocytes: Normal at BL→Abnormal-CS at Week 13 Number Analyzed 13 participants 14 participants
0 0
Leukocytes: Abnormal-NCS at BL→Abnormal-CS at Week 13 Number Analyzed 13 participants 14 participants
0 0
Leukocytes: Normal at BL→Abnormal-CS at Week 25 Number Analyzed 16 participants 14 participants
0 0
Leukocytes: Abnormal-NCS at BL→Abnormal-CS at Week 25 Number Analyzed 16 participants 14 participants
0 0
Leukocytes: Normal at BL→Abnormal-CS at Week 26 Number Analyzed 17 participants 12 participants
0 0
Leukocytes: Abnormal-NCS at BL→Abnormal-CS at Week 26 Number Analyzed 17 participants 12 participants
0 0
Neutrophils: Normal at BL→Abnormal-CS at Week 13 Number Analyzed 13 participants 14 participants
0 0
Neutrophils: Abnormal-NCS at BL→Abnormal-CS at Week 13 Number Analyzed 13 participants 14 participants
0 0
Neutrophils: Normal at BL→Abnormal-CS at Week 25 Number Analyzed 16 participants 14 participants
1 0
Neutrophils: Abnormal-NCS at BL→Abnormal-CS at Week 25 Number Analyzed 16 participants 14 participants
0 0
Neutrophils: Normal at BL→Abnormal-CS at Week 26 Number Analyzed 17 participants 12 participants
0 0
Neutrophils: Abnormal-NCS at BL→Abnormal-CS at Week 26 Number Analyzed 17 participants 12 participants
0 0
Ret/Ery: Normal at BL→Abnormal-CS at Week 13 Number Analyzed 14 participants 14 participants
1 0
Ret/Ery: Abnormal-NCS at BL→Abnormal-CS at Week 13 Number Analyzed 14 participants 14 participants
0 0
Ret/Ery: Normal at BL→Abnormal-CS at Week 25 Number Analyzed 16 participants 14 participants
0 0
Ret/Ery: Abnormal-NCS at BL→Abnormal-CS at Week 25 Number Analyzed 16 participants 14 participants
0 0
Ret/Ery: Normal at BL→Abnormal-CS at Week 26 Number Analyzed 17 participants 12 participants
1 0
Ret/Ery: Abnormal-NCS at BL→Abnormal-CS at Week 26 Number Analyzed 17 participants 12 participants
0 0
Platelets: Normal at BL→Abnormal-CS at Week 13 Number Analyzed 13 participants 14 participants
0 0
Platelets: Abnormal-NCS at BL→Abnormal-CS at Week 13 Number Analyzed 13 participants 14 participants
0 0
Platelets: Normal at BL→Abnormal-CS at Week 25 Number Analyzed 15 participants 14 participants
0 0
Platelets: Abnormal-NCS at BL→Abnormal-CS at Week 25 Number Analyzed 15 participants 14 participants
0 0
Platelets: Normal at BL→Abnormal-CS at Week 26 Number Analyzed 16 participants 12 participants
0 0
Platelets: Abnormal-NCS at BL→Abnormal-CS at Week 26 Number Analyzed 16 participants 12 participants
0 0
Sodium: Normal at BL→Abnormal-CS at Week 13 Number Analyzed 13 participants 13 participants
0 0
Sodium: Abnormal-NCS at BL→Abnormal-CS at Week 13 Number Analyzed 13 participants 13 participants
0 0
Sodium: Normal at BL→Abnormal-CS at Week 25 Number Analyzed 15 participants 14 participants
0 0
Sodium: Abnormal-NCS at BL→Abnormal-CS at Week 25 Number Analyzed 15 participants 14 participants
0 0
Sodium: Normal at BL→Abnormal-CS at Week 26 Number Analyzed 17 participants 13 participants
0 0
Sodium: Abnormal-NCS at BL→Abnormal-CS at Week 26 Number Analyzed 17 participants 13 participants
0 0
Potassium: Normal at BL→Abnormal-CS at Week 13 Number Analyzed 13 participants 13 participants
0 0
Potassium: Abnormal-NCS at BL→Abnormal-CS at Week 13 Number Analyzed 13 participants 13 participants
0 0
Potassium: Normal at BL→Abnormal-CS at Week 25 Number Analyzed 15 participants 14 participants
0 0
Potassium: Abnormal-NCS at BL→Abnormal-CS at Week 25 Number Analyzed 15 participants 14 participants
0 0
Potassium: Normal at BL→Abnormal-CS at Week 26 Number Analyzed 17 participants 13 participants
0 0
Potassium: Abnormal-NCS at BL→Abnormal-CS at Week 26 Number Analyzed 17 participants 13 participants
0 0
Albumin: Normal at BL→Abnormal-CS at Week 13 Number Analyzed 13 participants 13 participants
0 0
Albumin: Abnormal-NCS at BL→Abnormal-CS at Week 13 Number Analyzed 13 participants 13 participants
0 0
Albumin: Normal at BL→Abnormal-CS at Week 25 Number Analyzed 15 participants 14 participants
0 0
Albumin: Abnormal-NCS at BL→Abnormal-CS at Week 25 Number Analyzed 15 participants 14 participants
0 0
Albumin: Normal at BL→Abnormal-CS at Week 26 Number Analyzed 17 participants 13 participants
0 0
Albumin: Abnormal-NCS at BL→Abnormal-CS at Week 26 Number Analyzed 17 participants 13 participants
0 0
AA: Normal at BL→Abnormal-CS at Week 13 Number Analyzed 13 participants 13 participants
0 0
AA: Abnormal-NCS at BL→Abnormal-CS at Week 13 Number Analyzed 13 participants 13 participants
0 0
AA: Normal at BL→Abnormal-CS at Week 25 Number Analyzed 15 participants 14 participants
0 0
AA: Abnormal-NCS at BL→Abnormal-CS at Week 25 Number Analyzed 15 participants 14 participants
0 0
AA: Normal at BL→Abnormal-CS at Week 26 Number Analyzed 17 participants 13 participants
0 0
AA: Abnormal-NCS at BL→Abnormal-CS at Week 26 Number Analyzed 17 participants 13 participants
0 0
ASA: Normal at BL→Abnormal-CS at Week 13 Number Analyzed 13 participants 13 participants
0 0
ASA: Abnormal-NCS at BL→Abnormal-CS at Week 13 Number Analyzed 13 participants 13 participants
0 0
ASA: Normal at BL→Abnormal-CS at Week 25 Number Analyzed 14 participants 14 participants
0 0
ASA: Abnormal-NCS at BL→Abnormal-CS at Week 25 Number Analyzed 14 participants 14 participants
0 0
ASA: Normal at BL→Abnormal-CS at Week 26 Number Analyzed 17 participants 13 participants
0 0
ASA: Abnormal-NCS at BL→Abnormal-CS at Week 26 Number Analyzed 17 participants 13 participants
0 0
AP: Normal at BL→Abnormal-CS at Week 13 Number Analyzed 13 participants 13 participants
0 0
AP: Abnormal-NCS at BL→Abnormal-CS at Week 13 Number Analyzed 13 participants 13 participants
0 0
AP: Normal at BL→Abnormal-CS at Week 25 Number Analyzed 15 participants 14 participants
0 0
AP: Abnormal-NCS at BL→Abnormal-CS at Week 25 Number Analyzed 15 participants 14 participants
0 0
AP: Normal at BL→Abnormal-CS at Week 26 Number Analyzed 17 participants 13 participants
0 0
AP: Abnormal-NCS at BL→Abnormal-CS at Week 26 Number Analyzed 17 participants 13 participants
0 0
LD: Normal at BL→Abnormal-CS at Week 13 Number Analyzed 13 participants 13 participants
0 0
LD: Abnormal-NCS at BL→Abnormal-CS at Week 13 Number Analyzed 13 participants 13 participants
0 0
LD: Normal at BL→Abnormal-CS at Week 25 Number Analyzed 14 participants 14 participants
0 0
LD: Abnormal-NCS at BL→Abnormal-CS at Week 25 Number Analyzed 14 participants 14 participants
0 0
LD: Normal at BL→Abnormal-CS at Week 26 Number Analyzed 17 participants 13 participants
0 0
LD: Abnormal-NCS at BL→Abnormal-CS at Week 26 Number Analyzed 17 participants 13 participants
0 0
GGT: Normal at BL→Abnormal-CS at Week 13 Number Analyzed 13 participants 13 participants
0 0
GGT: Abnormal-NCS at BL→Abnormal-CS at Week 13 Number Analyzed 13 participants 13 participants
0 0
GGT: Normal at BL→Abnormal-CS at Week 25 Number Analyzed 15 participants 14 participants
0 0
GGT: Abnormal-NCS at BL→Abnormal-CS at Week 25 Number Analyzed 15 participants 14 participants
0 0
GGT: Normal at BL→Abnormal-CS at Week 26 Number Analyzed 17 participants 13 participants
0 0
GGT: Abnormal-NCS at BL→Abnormal-CS at Week 26 Number Analyzed 17 participants 13 participants
0 0
Bilirubin: Normal at BL→Abnormal-CS at Week 13 Number Analyzed 13 participants 13 participants
0 0
Bilirubin: Abnormal-NCS at BL→Abnormal-CS at Week 13 Number Analyzed 13 participants 13 participants
0 0
Bilirubin: Normal at BL→Abnormal-CS at Week 25 Number Analyzed 15 participants 14 participants
0 0
Bilirubin: Abnormal-NCS at BL→Abnormal-CS at Week 25 Number Analyzed 15 participants 14 participants
0 0
Bilirubin: Normal at BL→Abnormal-CS at Week 26 Number Analyzed 17 participants 13 participants
0 0
Bilirubin: Abnormal-NCS at BL→Abnormal-CS at Week 26 Number Analyzed 17 participants 13 participants
0 0
DB: Normal at BL→Abnormal-CS at Week 13 Number Analyzed 13 participants 13 participants
0 0
DB: Abnormal-NCS at BL→Abnormal-CS at Week 13 Number Analyzed 13 participants 13 participants
0 0
DB: Normal at BL→Abnormal-CS at Week 25 Number Analyzed 14 participants 14 participants
0 0
DB: Abnormal-NCS at BL→Abnormal-CS at Week 25 Number Analyzed 14 participants 14 participants
0 0
DB: Normal at BL→Abnormal-CS at Week 26 Number Analyzed 17 participants 13 participants
0 0
DB: Abnormal-NCS at BL→Abnormal-CS at Week 26 Number Analyzed 17 participants 13 participants
0 0
Creatinine: Normal at BL→Abnormal-CS at Week 13 Number Analyzed 13 participants 13 participants
0 0
Creatinine: Abnormal-NCS at BL→Abnormal-CS at Week 13 Number Analyzed 13 participants 13 participants
0 0
Creatinine: Normal at BL→Abnormal-CS at Week 25 Number Analyzed 15 participants 14 participants
0 0
Creatinine: Abnormal-NCS at BL→Abnormal-CS at Week 25 Number Analyzed 15 participants 14 participants
0 0
Creatinine: Normal at BL→Abnormal-CS at Week 26 Number Analyzed 17 participants 13 participants
0 0
Creatinine: Abnormal-NCS at BL→Abnormal-CS at Week 26 Number Analyzed 17 participants 13 participants
0 0
CK: Normal at BL→Abnormal-CS at Week 13 Number Analyzed 13 participants 13 participants
0 0
CK: Abnormal-NCS at BL→Abnormal-CS at Week 13 Number Analyzed 13 participants 13 participants
0 0
CK: Normal at BL→Abnormal-CS at Week 25 Number Analyzed 15 participants 14 participants
1 0
CK: Abnormal-NCS at BL→Abnormal-CS at Week 25 Number Analyzed 15 participants 14 participants
0 0
CK: Normal at BL→Abnormal-CS at Week 26 Number Analyzed 17 participants 13 participants
0 0
CK: Abnormal-NCS at BL→Abnormal-CS at Week 26 Number Analyzed 17 participants 13 participants
0 0
Glucose: Normal at BL→Abnormal-CS at Week 13 Number Analyzed 13 participants 13 participants
0 0
Glucose: Abnormal-NCS at BL→Abnormal-CS at Week 13 Number Analyzed 13 participants 13 participants
0 0
Glucose: Normal at BL→Abnormal-CS at Week 25 Number Analyzed 15 participants 14 participants
0 0
Glucose: Abnormal-NCS at BL→Abnormal-CS at Week 25 Number Analyzed 15 participants 14 participants
0 0
Glucose: Normal at BL→Abnormal-CS at Week 26 Number Analyzed 17 participants 13 participants
0 0
Glucose: Abnormal-NCS at BL→Abnormal-CS at Week 26 Number Analyzed 17 participants 13 participants
0 0
EU: Normal at BL→Abnormal-CS at Week 13 Number Analyzed 1 participants 1 participants
0 0
EU: Abnormal-NCS at BL→Abnormal-CS at Week 13 Number Analyzed 1 participants 1 participants
0 0
EU: Normal at BL→Abnormal-CS at Week 25 Number Analyzed 0 participants 0 participants
EU: Abnormal-NCS at BL→Abnormal-CS at Week 25 Number Analyzed 0 participants 0 participants
EU: Normal at BL→Abnormal-CS at Week 26 Number Analyzed 1 participants 0 participants
0 0
EU: Abnormal-NCS at BL→Abnormal-CS at Week 26 Number Analyzed 1 participants 0 participants
0 0
PU: Normal at BL→Abnormal-CS at Week 13 Number Analyzed 14 participants 14 participants
0 0
PU: Abnormal-NCS at BL→Abnormal-CS at Week 13 Number Analyzed 14 participants 14 participants
0 0
PU: Normal at BL→Abnormal-CS at Week 25 Number Analyzed 16 participants 15 participants
0 0
PU: Abnormal-NCS at BL→Abnormal-CS at Week 25 Number Analyzed 16 participants 15 participants
0 0
PU: Normal at BL→Abnormal-CS at Week 26 Number Analyzed 16 participants 13 participants
0 0
PU: Abnormal-NCS at BL→Abnormal-CS at Week 26 Number Analyzed 16 participants 13 participants
0 0
SG: Normal at BL→Abnormal-CS at Week 13 Number Analyzed 14 participants 14 participants
0 0
SG: Abnormal-NCS at BL→Abnormal-CS at Week 13 Number Analyzed 14 participants 14 participants
0 0
SG: Normal at BL→Abnormal-CS at Week 25 Number Analyzed 16 participants 15 participants
0 0
SG: Abnormal-NCS at BL→Abnormal-CS at Week 25 Number Analyzed 16 participants 15 participants
0 0
SG: Normal at BL→Abnormal-CS at Week 26 Number Analyzed 16 participants 13 participants
0 0
SG: Abnormal-NCS at BL→Abnormal-CS at Week 26 Number Analyzed 16 participants 13 participants
0 0
pH: Normal at BL→Abnormal-CS at Week 13 Number Analyzed 14 participants 14 participants
0 0
pH: Abnormal-NCS at BL→Abnormal-CS at Week 13 Number Analyzed 14 participants 14 participants
0 0
pH: Normal at BL→Abnormal-CS at Week 25 Number Analyzed 16 participants 15 participants
0 0
pH: Abnormal-NCS at BL→Abnormal-CS at Week 25 Number Analyzed 16 participants 15 participants
0 0
pH: Normal at BL→Abnormal-CS at Week 26 Number Analyzed 16 participants 13 participants
0 0
pH: Abnormal-NCS at BL→Abnormal-CS at Week 26 Number Analyzed 16 participants 13 participants
0 0
C3: Normal at BL→Abnormal-CS at Week 13 Number Analyzed 12 participants 14 participants
0 0
C3: Abnormal-NCS at BL→Abnormal-CS at Week 13 Number Analyzed 12 participants 14 participants
0 0
C3: Normal at BL→Abnormal-CS at Week 25 Number Analyzed 14 participants 15 participants
0 0
C3: Abnormal-NCS at BL→Abnormal-CS at Week 25 Number Analyzed 14 participants 15 participants
0 0
C3: Normal at BL→Abnormal-CS at Week 26 Number Analyzed 16 participants 13 participants
0 0
C3: Abnormal-NCS at BL→Abnormal-CS at Week 26 Number Analyzed 16 participants 13 participants
0 0
C4: Normal at BL→Abnormal-CS at Week 13 Number Analyzed 12 participants 14 participants
0 0
C4: Abnormal-NCS at BL→Abnormal-CS at Week 13 Number Analyzed 12 participants 14 participants
0 0
C4: Normal at BL→Abnormal-CS at Week 25 Number Analyzed 14 participants 15 participants
0 0
C4: Abnormal-NCS at BL→Abnormal-CS at Week 25 Number Analyzed 14 participants 15 participants
0 0
C4: Normal at BL→Abnormal-CS at Week 26 Number Analyzed 16 participants 13 participants
0 0
C4: Abnormal-NCS at BL→Abnormal-CS at Week 26 Number Analyzed 16 participants 13 participants
0 0
10.Secondary Outcome
Title Number of Participants With Treatment-Emergent Seroconversion or Positive Nucleic Acid Test (NAT) for Parvovirus B19 (B19V)
Hide Description Viral testing for B19V consisted of viral serology or NAT for B19V (Parvovirus B19 IgG Antibody, Parvovirus B19 IgM Antibody, Parvovirus B19 DNA Quant real-time polymerase chain reaction [RT-PCR]). Number of participants with treatment-emergent seroconversion or positive NAT for B19V were reported.
Time Frame From start of study treatment up to Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
Safety analysis set consisted of all enrolled participants who received any amount of IP, regardless of protocol deviations or non-adherence to study procedures. Here, "number analyzed" signifies those participants who were evaluable for this outcome measure at specific category.
Arm/Group Title Cohort I: GLASSIA (High-end) Cohort II: GLASSIA (Low-end)
Hide Arm/Group Description:
Participants received weekly intravenous (IV) infusions of GLASSIA (lot with particle loads representing the high end within the normal range) at 60 milligrams per kilogram (mg/kg) body weight (BW) active Alpha1-Proteinase Inhibitor (A1PI) protein at a rate of 0.2 milliliters per kilogram per minute (ml/kg/min) for 25 weeks (25 planned infusions).
Participants received weekly IV infusions of GLASSIA (lot with particle loads representing the low end within the normal range) at 60 mg/kg BW active A1PI protein at a rate of 0.2 ml/kg/min for 25 weeks (25 planned infusions).
Overall Number of Participants Analyzed 18 16
Measure Type: Count of Participants
Unit of Measure: Participants
Number of participants with parvovirus B19 IgG antibody Number Analyzed 18 participants 16 participants
0
   0.0%
1
   6.3%
Number of participants with parvovirus B19 IgM antibody Number Analyzed 18 participants 15 participants
0
   0.0%
0
   0.0%
Number of participants with Parvovirus B19 DNA Quant RT-PCR Number Analyzed 18 participants 15 participants
0
   0.0%
0
   0.0%
Time Frame From start of study treatment up to Week 26
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title GLASSIA (High-end) GLASSIA (Low-end)
Hide Arm/Group Description Participants received weekly intravenous (IV) infusions of GLASSIA (lot with particle loads representing the high end within) at 60 milligrams per kilogram (mg/kg) body weight (BW) active A1PI protein at a rate of 0.2 milliliters per kilogram per minute (ml/kg/min) for 25 weeks (25 planned infusions). Participants received weekly IV infusions of GLASSIA (lot with particle loads representing the low end within the normal range) at 60 mg/kg BW active A1PI protein at a rate of 0.2 ml/kg/min for 25 weeks (25 planned infusions).
All-Cause Mortality
GLASSIA (High-end) GLASSIA (Low-end)
Affected / at Risk (%) Affected / at Risk (%)
Total   0/18 (0.00%)      0/16 (0.00%)    
Hide Serious Adverse Events
GLASSIA (High-end) GLASSIA (Low-end)
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   1/18 (5.56%)      1/16 (6.25%)    
Infections and infestations     
Influenza * 1  1/18 (5.56%)  1 0/16 (0.00%)  0
Pneumonia * 1  1/18 (5.56%)  1 0/16 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
Pneumothorax * 1  0/18 (0.00%)  0 1/16 (6.25%)  1
1
Term from vocabulary, MedDRA 23.0
*
Indicates events were collected by non-systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
GLASSIA (High-end) GLASSIA (Low-end)
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   13/18 (72.22%)      9/16 (56.25%)    
Eye disorders     
Eye irritation * 1  0/18 (0.00%)  0 1/16 (6.25%)  1
Gastrointestinal disorders     
Diarrhoea * 1  3/18 (16.67%)  3 0/16 (0.00%)  0
Dry mouth * 1  0/18 (0.00%)  0 1/16 (6.25%)  1
Lip dry * 1  0/18 (0.00%)  0 1/16 (6.25%)  1
Nausea * 1  2/18 (11.11%)  2 0/16 (0.00%)  0
Vomiting * 1  2/18 (11.11%)  2 0/16 (0.00%)  0
General disorders     
Chills * 1  1/18 (5.56%)  1 0/16 (0.00%)  0
Fatigue * 1  1/18 (5.56%)  1 0/16 (0.00%)  0
Influenza like illness * 1  1/18 (5.56%)  1 0/16 (0.00%)  0
Pyrexia * 1  1/18 (5.56%)  1 0/16 (0.00%)  0
Hepatobiliary disorders     
Hepatic cirrhosis * 1  0/18 (0.00%)  0 1/16 (6.25%)  1
Infections and infestations     
Body tinea * 1  0/18 (0.00%)  0 1/16 (6.25%)  1
Bronchitis * 1  1/18 (5.56%)  1 1/16 (6.25%)  1
Ear infection * 1  1/18 (5.56%)  1 0/16 (0.00%)  0
Fungal infection * 1  0/18 (0.00%)  0 1/16 (6.25%)  1
Lower respiratory tract infection * 1  1/18 (5.56%)  1 0/16 (0.00%)  0
Nasopharyngitis * 1  5/18 (27.78%)  6 1/16 (6.25%)  2
Rhinitis * 1  1/18 (5.56%)  1 0/16 (0.00%)  0
Sinusitis * 1  0/18 (0.00%)  0 2/16 (12.50%)  2
Upper respiratory tract infection * 1  0/18 (0.00%)  0 3/16 (18.75%)  4
Viral infection * 1  0/18 (0.00%)  0 1/16 (6.25%)  1
Vulvovaginal mycotic infection * 1  0/18 (0.00%)  0 1/16 (6.25%)  1
Investigations     
Blood bicarbonate decreased * 1  1/18 (5.56%)  1 0/16 (0.00%)  0
Blood creatine phosphokinase increased * 1  1/18 (5.56%)  1 0/16 (0.00%)  0
Oxygen saturation decreased * 1  1/18 (5.56%)  1 0/16 (0.00%)  0
Metabolism and nutrition disorders     
Hyperglycaemia * 1  1/18 (5.56%)  1 0/16 (0.00%)  0
Musculoskeletal and connective tissue disorders     
Back pain * 1  2/18 (11.11%)  2 1/16 (6.25%)  1
Flank pain * 1  1/18 (5.56%)  1 0/16 (0.00%)  0
Musculoskeletal pain * 1  0/18 (0.00%)  0 1/16 (6.25%)  1
Tendonitis * 1  1/18 (5.56%)  1 0/16 (0.00%)  0
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Basal cell carcinoma * 1  1/18 (5.56%)  1 0/16 (0.00%)  0
Nervous system disorders     
Headache * 1  0/18 (0.00%)  0 1/16 (6.25%)  2
Lethargy * 1  1/18 (5.56%)  1 0/16 (0.00%)  0
Presyncope * 1  1/18 (5.56%)  1 0/16 (0.00%)  0
Sciatica * 1  0/18 (0.00%)  0 1/16 (6.25%)  1
Psychiatric disorders     
Anxiety * 1  1/18 (5.56%)  1 0/16 (0.00%)  0
Depression * 1  1/18 (5.56%)  1 0/16 (0.00%)  0
Renal and urinary disorders     
Haematuria * 1  1/18 (5.56%)  1 0/16 (0.00%)  0
Polyuria * 1  1/18 (5.56%)  1 0/16 (0.00%)  0
Urine abnormality * 1  0/18 (0.00%)  0 1/16 (6.25%)  1
Respiratory, thoracic and mediastinal disorders     
Chronic obstructive pulmonary disease * 1  0/18 (0.00%)  0 1/16 (6.25%)  1
Cough * 1  2/18 (11.11%)  2 0/16 (0.00%)  0
Dysphonia * 1  1/18 (5.56%)  1 0/16 (0.00%)  0
Dyspnoea * 1  2/18 (11.11%)  2 1/16 (6.25%)  1
Hiccups * 1  1/18 (5.56%)  1 0/16 (0.00%)  0
Nasal congestion * 1  1/18 (5.56%)  1 0/16 (0.00%)  0
Oropharyngeal pain * 1  2/18 (11.11%)  2 1/16 (6.25%)  1
Pneumonia aspiration * 1  0/18 (0.00%)  0 1/16 (6.25%)  1
Respiratory tract congestion * 1  1/18 (5.56%)  1 0/16 (0.00%)  0
Sinus congestion * 1  0/18 (0.00%)  0 1/16 (6.25%)  1
Tachypnoea * 1  1/18 (5.56%)  1 0/16 (0.00%)  0
Skin and subcutaneous tissue disorders     
Urticaria * 1  0/18 (0.00%)  0 1/16 (6.25%)  1
1
Term from vocabulary, MedDRA 23.0
*
Indicates events were collected by non-systematic assessment
[Not Specified]
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
If a multicenter publication is not submitted within twelve (12) months after conclusion, abandonment or termination of the Study at all sites, or after Sponsor confirms there shall be no multicenter Study publication, the Institution and/or such Principal Investigator may publish the results from the Institution site individually.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Study Director
Organization: Baxalta
Phone: +1 866 842 5335
EMail: ClinicalTransparency@takeda.com
Layout table for additonal information
Responsible Party: Takeda ( Baxalta now part of Shire )
ClinicalTrials.gov Identifier: NCT02525861    
Other Study ID Numbers: 471101
First Submitted: August 14, 2015
First Posted: August 18, 2015
Results First Submitted: July 29, 2021
Results First Posted: October 14, 2021
Last Update Posted: October 14, 2021