BAX 855 Continuation

• ClinicalTrials.gov Identifier: NCT01945593
• Recruitment Status: Completed
• First Posted: September 18, 2013
• Results First Posted: May 1, 2019
• Last Update Posted: May 24, 2021
• Sponsor: Baxalta now part of Shire
• Collaborator: Baxalta Innovations GmbH, now part of Shire
• Information provided by (Responsible Party): Takeda ( Baxalta now part of Shire )

Study Description

Brief Summary:

To continue the evaluation of the safety and efficacy of BAX 855 for prophylaxis and treatment of bleeding episodes in adult and pediatric previously treated patients (PTPs) aged ≤ 75 years of age with severe hemophilia A.

Condition or disease	Intervention/treatment	Phase
Hemophilia A	Biological: BAX855	Phase 3

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 218 participants
• Allocation: Non-Randomized
• Intervention Model: Parallel Assignment
• Masking: None (Open Label)
• Primary Purpose: Prevention
• Official Title: A Phase 3b Continuation Study of the Safety and Efficacy of PEGylated Recombinant Factor VIII (PEG-rFVIII; BAX 855) in Prophylaxis of Bleeding in Previously Treated Patients With Severe Hemophilia A
• Actual Study Start Date: October 15, 2013
• Actual Primary Completion Date: March 2, 2018
• Actual Study Completion Date: March 2, 2018

Arms and Interventions

Arm	Intervention/treatment
Experimental: Fixed BAX855 prophylaxis; 45-80 IU/kg twice weekly to once per week.	Biological: BAX855; Antihemophilic Factor (Recombinant), PEGylated; Other Names: ADYNOVATE; BAX 855; PEGylated Recombinant Factor VIII
Experimental: Pharmacokinetic (PK)-tailored BAX 855 prophylaxis; PK-tailored prophylactic BAX855 regimen based on participant's individual PK profile to maintain a Factor VIII (FVIII) trough level	Biological: BAX855; Antihemophilic Factor (Recombinant), PEGylated; Other Names: ADYNOVATE; BAX 855; PEGylated Recombinant Factor VIII

Outcome Measures

Primary Outcome Measures :

1. Number of Participants With Inhibitory Antibodies to Factor VIII (FVIII) [ Time Frame: Baseline through end of study (53 months) ]
   Inhibitory antibodies to Factor VIII were measured by the Nijmegen modification of the Bethesda assay. Inhibitors had to be confirmed by 2 separate assessments within a 2 to 4 week period from the central laboratory.
2. Annualized Bleed Rate (ABR) - Spontaneous Bleeds [ Time Frame: Baseline through end of study (53 months) ]
   The ABR was assessed based upon each individual bleeding episode. A bleeding episode was defined as subjective (pain consistent with a joint bleed) or objective evidence of bleeding which may or may not require treatment with FVIII. The ABR of spontaneous bleeds was reported separately for twice weekly, PK-t R, each of the every 5 days and every 7 days treatment regimens at the time of bleed.

Secondary Outcome Measures :

1. Total Annualized Bleed Rate (ABR) [ Time Frame: Baseline through end of study (53 months) ]
   The ABR was assessed based upon each individual bleeding episode. A bleeding episode was defined as subjective (pain consistent with a joint bleed) or objective evidence of bleeding which may or may not require treatment with FVIII. Bleeding occurring at multiple locations related to the same injury (e.g., knee and ankle bleed following a fall) was counted as a single bleeding episode. Total annualized bleed rate (spontaneous and traumatic bleeding episodes) was reported.
2. Overall Hemostatic Efficacy Rating of BAX 855 for Treatment of Breakthrough Bleeding Episodes [ Time Frame: Baseline through end of study (53 months) ]
   The participant or caregiver rated the overall treatment response at 24 (+/- 2) hours after the initiation of treatment using a 4-point efficacy rating scale as Excellent: Full relief of pain and cessation of objective signs of bleeding after a single infusion and no additional infusion is required for the control of bleeding; Good: Definite pain relief and/or improvement in signs of bleeding after a single infusion and possibly requires more than 1 infusion for complete resolution; Fair: Slight relief of pain and slight improvement in signs of bleeding after a single infusion and required more than 1 infusion for complete resolution and None: No improvement or condition worsens.
3. BAX 855 Infusions Needed to Treat Bleeding Episodes [ Time Frame: Baseline through end of study (53 months) ]
   The BAX 855 infusions to treat each bleeding episode was determined by the participant, the participant's caregiver, and/or investigator, and was based upon the participant's response to treatment. A bleeding episode was defined as subjective (pain consistent with a joint bleed) or objective evidence of bleeding which may or may not require treatment with FVIII.
4. Total Time Intervals Between Bleeding Episodes [ Time Frame: Baseline through end of study (53 months) ]
   The time interval between bleeding episodes was calculated based upon the date and time reported for each bleeding episode. A bleeding episode was defined as subjective (pain consistent with a joint bleed) or objective evidence of bleeding which may or may not require treatment with FVIII.
5. Average Dose of BAX 855 Per Prophylactic Infusion [ Time Frame: Baseline through end of study (53 months) ]
   The average dose of BAX 855 per prophylactic infusion was reported.
6. Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Baseline through end of study (53 months) ]
   An AE was any unfavorable and unintended sign (eg, an abnormal laboratory finding), symptom (eg, rash, pain, discomfort, fever, dizziness, etc.), disease (eg, peritonitis, bacteremia, etc.), or outcome of death temporally associated with the use of an investigational product (IP), whether or not considered causally related to the IP. A serious adverse event (SAE) was defined as an untoward medical occurrence that at any dose met one or more of the following criteria: outcome was fatal/resulted in death; was life-threatening; required inpatient hospitalization or resulted in prolongation of an existing hospitalization; resulted in persistent or significant disability/incapacity; was a congenital anomaly/birth defect; was a medically important event.
7. Change From Baseline in Body Temperature [ Time Frame: Baseline, end of study (53 months) ]
   Change in body temperature at pre-infusion and post-infusion at end of the study was reported. In the below table, FDR refers to fixed dose regimen, PK-tR refers to PK tailored regimen at the time of sampling.
8. Change From Baseline in Pulse Rate [ Time Frame: Baseline, end of study (53 months) ]
   Change in pulse rate at pre-infusion and post-infusion at end of the study was reported. In the below table, FDR refers to fixed dose regimen, PK-tR refers to PK tailored regimen at the time of sampling.
9. Change From Baseline in Respiratory Rate [ Time Frame: Baseline, end of study (53 months) ]
   Change in respiratory rate at pre-infusion and post-infusion at end of the study was reported. In the below table, FDR refers to fixed dose regimen, PK-tR refers to PK tailored regimen at the time of sampling.
10. Change From Baseline in Blood Pressure [ Time Frame: Baseline, end of study (53 months) ]
    Change in systolic and diastolic blood pressure at pre-infusion and post-infusion at end of the study were reported. In the below table, FDR refers to fixed dose regimen, PK-tR refers to PK tailored regimen at the time of sampling, SBP refers to systolic blood pressure, DBP refers to diastolic blood pressure.
11. Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments. [ Time Frame: Baseline through end of study (53 months) ]
    The number of participants with clinically significant shifts from "normal" "abnormal clinically significant (CS)" and "abnormal not clinically significant (abnormal NCS)" at baseline to "normal" "abnormal clinically significant (CS) and abnormal clinically significant (NCS)" at completion were reported. In the below table, FDR refers to fixed dose regimen at the time of sampling, AlA refers to alanine aminotransferase, AP refers to alkaline phosphatase, AsA refers to aspartate aminotransferase.
12. Number of Participants With Shifts in Hematology Laboratory Assessments [ Time Frame: Baseline through end of study (53 months) ]
    The number of participants with clinically significant shifts from "normal" "abnormal clinically significant (CS)" and "abnormal not clinically significant (abnormal NCS)" at baseline to "normal" "abnormal clinically significant (CS) and abnormal clinically significant (NCS)" at completion were reported. In the below table, FDR refers to fixed dose regimen, PK-tR refers to PK tailored regimen at the time of sampling, Leu refers to leukocytes, MCV refers to mean corpuscular volume, Lym/Leu refers to lymphocytes/leukocytes.
13. Number of Participants With Shifts in Lipid Panel Assessments [ Time Frame: Baseline through end of study (53 months) ]
    The number of participants with clinically significant shifts from "normal" "abnormal clinically significant (CS)" and "abnormal not clinically significant (abnormal NCS)" at baseline to "normal" "abnormal clinically significant (CS) and abnormal clinically significant (NCS)" at completion were reported.. In the below table, HDL refers to high density lipoprotein, LDL refers to low density lipoprotein, VLDL refers to very low density lipoprotein.
14. Number of Participants With Binding Antibodies [ Time Frame: Baseline through end of study (53 months) ]
    Binding antibodies (IgG and IgM) against FVIII, polyethylene glycol (PEG) and PEGylated FVIII (PEG-FVIII) were analyzed using enzyme-linked immunosorbent assay (ELISA).
15. Number of Participants With Anti-Chinese Hamster Ovary (CHO) Antibodies [ Time Frame: Baseline through end of study (53 months) ]
    Testing for binding of anti-CHO protein antibodies was performed on citrate-anti-coagulated plasma using an ELISA employing polyclonal antihuman IgG antibodies.
16. Change From Baseline in Bleed Severity [ Time Frame: Baseline, end of study (53 months) ]
    Hemophilia symptom (haemo-SYM) questionnaire has two subscales: pain and bleed. It was used to asses the bleed severity for participants >=18 years of age as: severity of spontaneous bleeding in my joints (unrelated to injury or activity), spontaneous bleeding in my muscles (unrelated to injury or activity), prolonged bleeding after injury in spite of treatment, intense pain because of bleeding event, joint pain due to active bleed and bleeding during personal hygiene routine, blood in my urine, nose bleeds and assigned a score of 0=Absent, 1=very mild, 2=mild, 3=moderate, 4=severe and 5=very severe. The score was determined as (mean score/5)*100 where mean score is the mean of the available results in the particular subscale. Higher scores on the Haemo-SYM indicate more severe symptoms. Therefore, negative change scores indicate that symptoms have improved. Here 'n' refers to the number of participants evaluable for this endpoint.
17. Change From Baseline in Pain Severity [ Time Frame: Baseline, end of study (53 months) ]
    Hemophilia symptom (haemo-SYM) questionnaire has two subscales: pain and bleed. It was used to asses the pain severity for participants >=18 years of age as: pain because of swelling in my joints, climbing stairs, upon waking in the morning, active arthritis; constant pain, in my muscles, that needs medication; joint sensitivity to weather conditions; reduced range of joint movement, joint deformity, sleep disturbance because of pain or bleeds, blood in my urine, nose bleeds and assigned a score of 0=Absent, 1=very mild, 2=mild, 3=moderate, 4=severe and 5=very severe. The score was determined as (mean score/5)*100 where mean score is the mean of the available results in the particular subscale. Higher scores on the Haemo-SYM indicate more severe symptoms. Therefore, negative change scores indicate that symptoms have improved. Here 'n' refers to the number of participants evaluable for this endpoint.
18. Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Short Form-36 (SF-36) [ Time Frame: Baseline, end of study (53 months) ]
    HRQoL in participants aged >=14 years was measured using the SF-36 questionnaire. The questionnaire was divided into 8 domains and scored as: physical functioning (1=yes, limited a lot to 3=no, not limited at all), role-physical (1=all of the time to 5=none of the time), bodily pain (1=very severe to 6=none), general health (1=poor to 5=excellent), vitality (1=none of the time to 5=all of the time), social functioning (1=all of the time: to 5=none of the time), role emotional (1=all of the time to 5=none of the time) and mental health (1=all of the time to 5=none of the time). The score for each domain is then to be transformed to a 0-100 range as [(actual raw score-lowest possible raw score)/possible raw score range]*100. Positive change scores indicate improved HRQoL. in the below table 'FDR' indicates fixed dose regimen, 'PK-tr' indicates pharmacokinetically tailored regimen and 'n' refers to the number of participants evaluable for this endpoint.
19. Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Pediatrics Quality of Life (PedsQL) Questionnaire [ Time Frame: Baseline, end of study (53 months) ]
    HRQoL in participants aged <14 years was measured using the PedsQL. It capture data for the following domains: physical functioning, emotional functioning, social functioning, school functioning, psychosocial functioning, physical health and a total score. Each question of the PedsQL was scored as Never: 100, almost never: 75, sometimes: 50, often: 25, almost always: 0. The mean of the individual question scores was calculated. Lower scores on the PedsQL indicating worse HRQoL. Here, FDR refers to fixed dose regimen, PK-t R refers to PK-tailored regimen. Here 'n' refers to the number of participants evaluable for this endpoint. Here 'n' refers to the number of participants evaluable for this endpoint.

Eligibility Criteria

• Ages Eligible for Study: up to 75 Years (Child, Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

INCLUSION CRITERIA

Participants Transitioning from Other BAX 855 Studies:

Participants transitioning from other BAX 855 studies can be provided with the continuation study informed consent form (ICF) prior to the end of study visit to review and consider participation in this continuation study. These participants will complete any additional screening assessments within 2 weeks of the previous study's end of study visit and will return to the study site within 6 (± 1) weeks of the previous study end of study visit to confirm eligibility for this continuation study.

• Participants transitioning from other BAX 855 studies who meet ALL of the following criteria are eligible for this study:

  1. Participant has completed a previous BAX 855 study and is willing to immediately transition into this continuation study.
  2. Participant is ≤75 years of age at screening of the previous BAX 855 study.
  3. Participant continues to have a Karnofsky (for participants aged ≥ 16 years) or Lansky (for participants aged < 16 years) performance score of ≥ 60.
  4. Participant is human immunodeficiency virus negative (HIV-); or HIV+ with stable disease and CD4+ count ≥ 200 cells/mm^3, as confirmed by central laboratory at screening.
  5. Participant is hepatitis C virus negative (HCV-) by antibody or polymerase chain reaction (PCR) testing (if positive, antibody titer will be confirmed by PCR), as confirmed by central laboratory at screening; or HCV+ with chronic stable hepatitis.
  6. If female of childbearing potential, participant presents with a negative urine pregnancy test and agrees to employ adequate birth control measures for the duration of the study.
  7. Participant and/or legally authorized representative is willing and able to comply with the requirements of the protocol.
• BAX 855 Naïve Participants:

BAX 855 naïve participants who are ≥ 12 years of age can only be enrolled in this continuation study after enrollment in the phase 2/3 pivotal study is closed. BAX 855 naïve participants who are < 12 years of age can only be enrolled in this continuation study after enrollment in the pediatric previously treated patient (PTP) study is closed.

- Enrolment of BAX 855 naïve participants will only start once the sponsor has notified the study sites accordingly.

BAX 855 naïve participants who meet ALL of the following criteria are eligible for this study:

1. Participant is ≤75 years of age at screening.
2. Participant is naïve to BAX 855.
3. Participant has severe hemophilia A (FVIII clotting activity < 1%) as confirmed by central laboratory at screening after at least a 72-hour washout period.
4. Participant aged ≥ 6 years has documented previous treatment with plasma-derived FVIII or rFVIII for ≥ 150 exposure days (EDs).
5. Participant aged < 6 years has documented previous treatment with plasma-derived FVIII concentrates or rFVIII for ≥ 50 EDs.
6. Participant is currently receiving prophylaxis or on-demand therapy with FVIII.
7. Participant has a Karnofsky (for participants aged ≥ 16 years) or Lansky (for participants aged < 16 years) performance score of ≥ 60.
8. Participant is HIV-; or HIV+ with stable disease and CD4+ count ≥ 200 cells/mm^3, as confirmed by central laboratory at screening.
9. Participant is HCV- by antibody or PCR testing (if positive, antibody titer will be confirmed by PCR), as confirmed by central laboratory at screening; or HCV+ with chronic stable hepatitis.
10. If female of childbearing potential, participant presents with a negative urine pregnancy test and agrees to employ adequate birth control measures for the duration of the study.
11. Participant and/or legally authorized representative is willing and able to comply with the requirements of the protocol.

EXCLUSION CRITERA

- Participants Transitioning from Other BAX 855 Studies:

Participants transitioning from other BAX 855 studies who meet ANY of the following criteria are not eligible for this study:

1. Participant had detectable factor VIII (FVIII) inhibitory antibodies (≥ 0.6 Bethesda unit (BU) using the Nijmegen modification of the Bethesda assay) as confirmed by central laboratory at screening.
2. Participant has developed FVIII inhibitory antibodies (≥ 0.6 BU using the Nijmegen modification of the Bethesda assay as determined at central laboratory in a previous BAX 855 study).
3. Participant has acquired a hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand's disease) in a previous BAX 855 study.
4. Participant has severe chronic hepatic dysfunction (eg, ≥ 5 times upper limit of normal alanine aminotransferase [ALT], as confirmed by central laboratory at screening).
5. Participant has severe renal impairment (serum creatinine > 2.0 mg/dL), as confirmed by central laboratory at screening.
6. Participant experienced a life-threatening or gastrointestinal bleeding episode within 3 months prior to study entry.
7. Participant is scheduled to use other PEGylated drugs during study participation.
8. Participant is planning to take part in any other clinical study during the course of the continuation study, with the exception of any other parallel BAX 855 study.
9. Participant has medical, psychiatric, or cognitive illness or recreational drug/alcohol use that, in the opinion of the investigator, would affect participant safety or compliance.

10. Participant is a family member or employee of the investigator.

    • BAX 855 Naïve Participants:

BAX 855 naïve participants who meet ANY of the following criteria are not eligible for this study:

1. Participant has detectable FVIII inhibitory antibodies (≥ 0.6 BU using the Nijmegen modification of the Bethesda assay) as confirmed by central laboratory at screening.
2. Participant has history of FVIII inhibitory antibodies (≥ 0.6 BU using the Nijmegen modification of the Bethesda assay or the Bethesda assay) at any time prior to screening.
3. Participant has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand's disease).
4. Participant has known hypersensitivity towards mouse or hamster proteins, polyethylene glycol (PEG), or Tween 80.
5. Participant has severe chronic hepatic dysfunction eg, ≥ 5 times upper limit of normal ALT, as confirmed by central laboratory at screening).
6. Participant has severe renal impairment (serum creatinine > 2.0 mg/dL), as confirmed by central laboratory at screening.
7. Participant experienced a life-threatening or gastrointestinal bleeding episode within 3 months prior to study entry.
8. Participant has current or recent (< 30 days) use of other PEGylated drugs prior to study participation or scheduled use of such drugs during study participation.
9. Participant has participated in another clinical study involving an IP other than BAX 855 or device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an investigational product (IP) or investigational device during the course of this study.
10. Participant has medical, psychiatric, or cognitive illness or recreational drug/alcohol use that, in the opinion of the investigator, would affect participant safety or compliance.
11. Participant is a family member or employee of the investigator.

Contacts and Locations

Locations

United States, Arizona

Phoenix Childrens Hospital

Phoenix, Arizona, United States, 85016-7710

United States, Colorado

University of Colorado

Aurora, Colorado, United States, 80045

United States, Florida

University of Florida College of Medicine

Gainesville, Florida, United States, 32610-0278

United States, Illinois

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States, 60611-2605

Bleeding and Clotting Disorders Institute

Peoria, Illinois, United States, 61614

United States, Kentucky

University of Kentucky Medical Center

Lexington, Kentucky, United States, 40536-0284

University of Louisville

Louisville, Kentucky, United States, 40202

United States, Louisiana

Tulane University School of Medicine

New Orleans, Louisiana, United States, 70112

United States, Missouri

Children's Mercy Hospitals & Clinics

Kansas City, Missouri, United States, 64108

United States, New York

North Shore-Long Island Jewish Health System

New Hyde Park, New York, United States, 11040

New York - Presbyterian/Weill Cornell Medical Center

New York, New York, United States, 10065

United States, North Carolina

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, United States, 27599-7016

Duke University Medical Center

Durham, North Carolina, United States, 27705

United States, Ohio

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States, 45229

Cleveland Clinic Foundation

Cleveland, Ohio, United States, 44195

Nationwide Children's Hospital

Columbus, Ohio, United States, 43205

United States, Oklahoma

University of Oklahoma

Oklahoma City, Oklahoma, United States, 73104

United States, Pennsylvania

Penn State Hershey Cancer Center

Hershey, Pennsylvania, United States, 17033

United States, South Carolina

Palmetto Health Richland

Columbia, South Carolina, United States, 29203-6863

United States, Utah

University of Utah

Salt Lake City, Utah, United States, 84108

United States, Washington

Puget Sound Blood Group

Seattle, Washington, United States, 98104

Australia, South Australia

Royal Adelaide Hospital

Adelaide, South Australia, Australia, 5000

Australia, Victoria

The Alfred Hospital

Melbourne, Victoria, Australia, 3004

Australia, Western Australia

Fremantle Hospital

Fremantle, Western Australia, Australia, 6160

Austria

Landes-Frauen-und Kinderklinik Linz

Linz, Austria, 4020

Universitatsklinik fur Innere Medizin I

Vienna, Austria, 1090

Bulgaria

UMHAT "Sv. Georgi", EAD

Plovdiv, Bulgaria, 4002

SHAT of Oncohaematology Diseases

Sofia, Bulgaria, 1527

MHAT 'Sv. Marina', EAD

Varna, Bulgaria, 9010

Czechia

Fakultni nemocnice Brno

Brno, Czechia, 625 00

Fakultni nemocnice Olomouc

Olomouc, Czechia, 775 20

Fakultni nemocnice v Motole

Praha 5, Czechia, 150 06

Germany

Werlhof-Institut GmbH

Hannover, Niedersachsen, Germany, 30159

Gerinnungszentrum Rhein-Ruhr

Duisburg, Nordrhein Westfalen, Germany, 47051

Vivantes Klinikum im Friedrichshain - Landsberger Allee

Berlin, Germany, 10249

Universitaetsklinikum Hamburg-Eppendorf

Hamburg, Germany, 20246

Hong Kong

Prince of Wales Hospital

Shatin, Hong Kong, 00000

Israel

Rambam Health Care Campus

Haifa, Israel, 3109601

Chaim Sheba Medical Center

Ramat-Gan, Israel, 52621

Japan

Nagoya University Hospital

Nagoya-shi, Aichi-Ken, Japan, 466-8560

University of Occupational and Environmental Health Hospital

Kitakyushu-shi, Fukuoka-Ken, Japan, 807-8556

Hiroshima University Hospital

Hiroshima-shi, Hiroshima-Ken, Japan, 734-8551

St. Marianna University School of Medicine Hospital

Kawasaki-shi, Kanagawa-Ken, Japan, 216-8511

Nara Medical University Hospital

Kashihara-shi, Nara-Ken, Japan, 634-8522

Tokyo Medical University Hospital

Shinjuku-ku, Tokyo-To, Japan, 160-0023

Ogikubo Hospital

Suginami City, Tokyo, Japan, 167-8515

Korea, Republic of

Chonnam National University Hwasun Hospital

Hwasun-gun, Jeollanam-do, Korea, Republic of, 519-763

Pusan National University Hospital

Busan, Korea, Republic of, 602-739

Eulji University Hospital

Daejeon, Korea, Republic of, 302-799

Kyung Hee University Hospital at Gangdong

Seoul, Korea, Republic of, 134-727

Ulsan University Hospital

Ulsan, Korea, Republic of, 682-714

Lithuania

Vilnius University Hospital Santariskiu Clinics, Public Institution

Vilnius, Lithuania, 8661

Children's Hospital, Affiliate of Vilnius University Hospital Santariskiu Klinikos

Vilnius, Lithuania, LT-08406

Malaysia

Penang General Hospital

George Town, Penang, Malaysia, 10990

Hospital Umum Sarawak

Kuching, Sarawak, Malaysia, 93586

Hospital Sibu

Sibu, Sarawak, Malaysia, 96000

Hospital Ampang

Ampang, Selangor, Malaysia, 68000

Pusat Darah Negara

Kuala Lumpur, Malaysia, 50400

Hospital Pulau Pinang

Pulau Pinang, Malaysia, 10450

Netherlands

Academisch Medisch Centrum

Amsterdam, Netherlands, 1105 AZ

Poland

Uniwersyteckie Centrum Kliniczne

Gdansk, Poland, 80-952

Wojewodzki Szpital Specjalistyczny im.M.Kopernika w Lodzi

Lodz, Poland, 93-510

Romania

Sanador SRL

Bucuresti, Romania, 11038

Russian Federation

LLC "Alba Dent"

Kirov, Russian Federation, 610000

Regional Clinical Hospital

Krasnoyarsk, Russian Federation, 660022

Spain

Hospital Universitari Son Espases

Palma de Mallorca, Baleares, Spain, 7010

Complejo Hospitalario Universitario A Coruña

A Coruña, La Coruña, Spain, 15006

Hospital Regional Universitario de Malaga

Malaga, Málaga, Spain, 29010

Hospital Universitario La Paz

Madrid, Spain, 28046

Hospital Universitari i Politecnic La Fe

Valencia, Spain, 46026

Sweden

Sjukhusapoteket Malmo

Malmo, Sweden, 21428

Karolinska

Stockholm, Sweden, 17164

Switzerland

Universitaetsspital Zuerich

Zuerich, Switzerland, 8091

Taiwan

Taichung Veterans General Hospital

Taichung, Taiwan, 40705

Tri-Service General Hospital

Taipei, Taiwan, 11490

Turkey

Ankara University Medical Faculty

Ankara, Turkey, 063100

Akdeniz University Faculty of Medicine

Antalya, Turkey, 07059

Istanbul University Cerrahpasa Medical Faculty

Istanbul, Turkey, 34098

Ukraine

SI V.K.Gusak Emergency and Reconstructive Surgery Institute of NAMSU

Donetsk, Ukraine, 83045

SI Institute of Blood Pathology and Transfusion Medicine of NAMSU

Lviv, Ukraine, 79044

United Kingdom

Bristol Royal Hospital for Children

Bristol, Avon, United Kingdom, BS2 8BJ

St Thomas' Hospital

London, Greater London, United Kingdom, SE1 7EH

Royal Free Hospital

London, Greater London, United Kingdom, WC1E 6AG

Great Ormond Street Hospital for Children

London, Greater London, United Kingdom, WC1N 3JH

Royal Manchester Children's Hospital

Manchester, Greater Manchester, United Kingdom, M13 9WL

Southampton General Hospital

Southampton, Hampshire, United Kingdom, SO16 6YD

Leicester Royal Infirmary

Leicester, Leicestershire, United Kingdom, LE1 5WW

Birmingham Children's Hospital

Birmingham, West Midlands, United Kingdom, B4 6NH

The Churchill Hospital

Oxford, United Kingdom, OX3 7LE

Sponsors and Collaborators

Baxalta now part of Shire

Baxalta Innovations GmbH, now part of Shire

Investigators

• Study Director: Study Director; Takeda

  Study Documents (Full-Text)

Documents provided by Takeda ( Baxalta now part of Shire ):

Study Protocol: Protocol  [PDF] June 18, 2013

Study Protocol: Protocol Amendment 1  [PDF] October 2, 2013

Study Protocol: Protocol Amendment 4  [PDF] May 23, 2014

Study Protocol: Protocol Amendment 7  [PDF] March 20, 2015

Statistical Analysis Plan  [PDF] April 18, 2018

More Information

• Responsible Party: Baxalta now part of Shire
• ClinicalTrials.gov Identifier: NCT01945593
• Other Study ID Numbers: 261302; 2013-002236-24 ( EudraCT Number )
• First Posted: September 18, 2013
• Results First Posted: May 1, 2019
• Last Update Posted: May 24, 2021
• Last Verified: April 2021

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: Yes
• Plan Description: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
• Supporting Materials: Study Protocol; Statistical Analysis Plan (SAP); Informed Consent Form (ICF); Clinical Study Report (CSR)
• Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
• URL: https://vivli.org/ourmember/takeda/

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Hemophilia A; Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Factor VIII; BAX 855; Coagulants