BAX 855 PK-guided Dosing (PROPEL)

• ClinicalTrials.gov Identifier: NCT02585960
• Recruitment Status: Completed
• First Posted: October 26, 2015
• Results First Posted: August 26, 2019
• Last Update Posted: May 25, 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:

1. To compare the efficacy and safety of pharmacokinetic (PK)-guided treatment with BAX 855 targeting FVIII trough levels of 1-3% and approximately 10% (8-12%)
2. To further characterize pharmacokinetic (PK) and pharmacodynamic (PD) parameters of BAX 855

Condition or disease	Intervention/treatment	Phase
Hemophilia A	Biological: PEGylated Recombinant Factor VIII	Phase 3

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 135 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: None (Open Label)
• Primary Purpose: Prevention
• Official Title: Phase 3, Prospective, Randomized, Multi-center Clinical Study Comparing the Safety and Efficacy of BAX 855 Following PK-guided Prophylaxis Targeting Two Different FVIII Trough Levels in Subjects With Severe Hemophilia A
• Actual Study Start Date: November 23, 2015
• Actual Primary Completion Date: August 5, 2018
• Actual Study Completion Date: August 5, 2018

Arms and Interventions

Arm	Intervention/treatment
Experimental: Pharmacokinetic (PK) evaluation of BAX 855; Participants will first undergo an initial pharmacokinetic (PK) assessment. Following the PK assessment participants will be randomized to one of 2 dosing regimens.	Biological: PEGylated Recombinant Factor VIII; Pharmacokinetic (PK) evaluation; Other Names: BAX855; BAX 855
Experimental: FVIII trough target 1-3%; Standard treatment arm - PK-guided dosing schedule to achieve a Factor VIII (FVIII) trough of 1-3%	Biological: PEGylated Recombinant Factor VIII; Standard treatment; Other Names: BAX 855; BAX855
Experimental: FVIII trough target 8-12%; Intensified treatment arm - PK-guided dosing schedule to achieve a Factor VIII (FVIII) trough of 8-12%	Biological: PEGylated Recombinant Factor VIII; Intensified treatment; Other Names: BAX855; BAX 855

Outcome Measures

Primary Outcome Measures :

1. Percentage of Participants With a Total Annualized Bleeding Rate (ABR) of Zero for Second Six Months [ Time Frame: Day 183 to Day 364 (6 months) ]
   Annualized bleeding rate was determined by dividing the number of bleeds by observation period in years.

Secondary Outcome Measures :

1. Total Annualized Bleeding Rate for Second Six Months [ Time Frame: Day 183 to Day 364 (6 months) ]
   Annualized bleeding rate was determined by dividing the number of bleeds by observation period in years.
2. Annualized Spontaneous Bleeding Rate for Second Six Months [ Time Frame: Day 183 to Day 364 (6 months) ]
   Annualized spontaneous bleeding rate was determined by dividing the number of spontaneous bleeds by observation period in years. A bleed was defined as spontaneous if it was not related to injury/trauma.
3. Annualized Traumatic Bleeding Rate for Second Six Months [ Time Frame: Day 183 to Day 364 (6 months) ]
   Annualized traumatic bleeding rate was determined by dividing the number of traumatic bleeds by observation period in years. A bleed was defined as traumatic if it was related to injury/trauma.
4. Annualized Joint Bleeding Rate (AJBR) for Second Six Months [ Time Frame: Day 183 to Day 364 (6 months) ]
   Annualized joint bleeding rate was determined by dividing the number of joint bleeds by observation period in years. An acute joint bleed include some or all of the following: 'aura', pain, swelling, warmth of the skin over the joint, decreased range of motion and difficulty in using the limb compared with baseline or loss of function.
5. Total Weight-adjusted Consumption of BAX 855 [ Time Frame: From start of study treatment up to 12 months (completion or termination) ]
   Total weight-adjusted consumption of BAX 855 were reported.
6. Number of Bleeding Episodes: Overall Hemostatic Efficacy Rating at Number of Infusions [ Time Frame: 8 hours after study drug administration ]
   The participant or caregiver rated the overall treatment response 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: Probable and/or 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.
7. Number of Bleeding Episodes: Overall Hemostatic Efficacy Rating at Bleed Resolution [ Time Frame: From start of study treatment up to bleed resolution (up to 12 months) ]
   The participant or caregiver rated the overall treatment response 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: Probable and/or 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.
8. Treatment of Bleeding Episodes: Number of BAX 855 Infusions Per Bleeding Episode Required Until Bleed Resolution [ Time Frame: From start of study treatment up to 12 months (completion or termination) ]
   Infusions of BAX 855 that were required until bleed resolution were reported.
9. Change From Baseline in Hemophilia Joint Health Score (HJHS)- Total Score [ Time Frame: Baseline, Month 12 ]
   HJHS was assessed based on the following components of the elbow, knee, and ankle joints: swelling, duration of swelling, muscle atrophy, crepitus on motion, flexion loss, extension loss, joint pain, and strength, together with an assessment of the global gait. The HJHS is a validated 11-item scoring tool based on radiologic and clinical evaluation, sensitive to detect early signs and minor changes. HJHS ranges from 0 to 124. Higher values in the HJHS represent worse situation for the participant.
10. Number of Participants With Hemostatic Efficacy Ratings for BAX 855 Treatment of Operative Bleeds [ Time Frame: Day 0 through discharge or 14 days post-surgery ]
    The participant or caregiver rated the overall treatment response 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: Probable and/or 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. Hemostatic efficacy was evaluated intra-operatively (from start to end of the procedure), post-operatively (from the end of procedure up to 24 h post procedure), and perioperatively (from the start of procedure to participant discharge from hospital or 14 days after completion of procedure; whichever was first).
11. Blood Loss Per Participant in Case of Surgery [ Time Frame: Day 0 through discharge or 14 days post-surgery ]
    The intraoperative blood loss was measured by determining the volume of blood and fluid removal through suction into the collection container (waste box and/or cell saver) and the estimated blood loss into swabs and towels during the procedure, per the anesthesiologist's record. Postoperatively, blood loss was determined by the drainage volume collected, which mainly consisted of drainage fluid via vacuum or gravity drain, as applicable. In cases where no drain was present, blood loss was determined by the surgeon's clinical judgment, as applicable or entered as "not available". Blood loss was evaluated intra-operatively (from start to end of the procedure), post-operatively (from the end of procedure up to 24 h post procedure), and perioperatively (from the start of procedure to participant discharge from hospital or 14 days after completion of procedure; whichever was first).
12. Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: From start of study treatment up to 12 months (completion or termination) ]
    An AE was any unfavorable and unintended sign (an abnormal laboratory finding), symptom (rash, pain, discomfort, fever, dizziness, etc.), disease (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 SAE was defined as an untoward medical occurrence that at any dose met one or more of the following criteria: outcome was fatal/results in death, life-threatening, required in-patient 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 that was not immediately life-threatening or resulted in death or required hospitalization but jeopardize the participant or required medical or surgical intervention to prevent any of the above outcomes.
13. Number of Participants With Clinically Significant Changes in Vital Signs Reported as Treatment Related Adverse Events [ Time Frame: From start of study treatment up to 12 months (completion or termination) ]
    Vital signs included systolic and diastolic blood pressure, pulse rate, respiratory rate, body temperature.
14. Number of Participants With Clinically Significant Changes in Clinical Laboratory Parameters Reported as Treatment Related Adverse Events [ Time Frame: From start of study treatment up to 12 months (completion or termination) ]
    Clinical laboratory assessments included clinical chemistry, hematology, lipid panel, genetics, T-cell, B-cell and NK cell (TBNK) and viral serology.
15. Number of Participants With Positive Inhibitory Antibodies and Binding Antibodies to Factor VIII (FVIII), BAX 855, Polyethylene Glycol (PEG), and Chinese Hamster Ovary (CHO) Protein [ Time Frame: From start of study treatment up to 12 months (completion or termination) ]
    Positive Inhibitory Antibodies and Binding Antibodies to Factor VIII (FVIII), BAX 855, Polyethylene Glycol (PEG), and Chinese Hamster Ovary (CHO) Protein were reported here.
16. Change From Baseline in Physical Component Scores (PCS) of the Short Form-36 (SF-36) Health Survey [ Time Frame: Baseline, Month 12 (completion or termination) ]
    Short Form (36) Health Survey (SF-36) is a 36-item validated, generic health related quality of life (HR QoL) instrument. PCS is a summary scale of the dimensions physical functioning, role physical, bodily pain, and general health. The component score is normalized to a standard population. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both sub-scores and summary scores.
17. Area Under the Plasma Concentration of BAX 855 From Zero to Infinity (AUC0-inf) [ Time Frame: Pre-infusion, 15 - 30 minutes, 3, 8, 24, 48, 72 and 96 hours post-infusion ]
    Area under the plasma concentration versus time curve from time 0 to infinity of BAX 855 were reported.
18. Incremental Recovery (IR) at Maximum Plasma Concentration (Cmax) of BAX 855 [ Time Frame: Pre-infusion, 15 - 30 minutes, 3, 8, 24, 48, 72 and 96 hours post-infusion ]
    IR at Cmax of BAX 855 were reported.
19. Plasma Half-life (T1/2) of BAX 855 [ Time Frame: Pre-infusion, 15 - 30 minutes, 3, 8, 24, 48, 72 and 96 hours post-infusion ]
    T1/2 of BAX 855 in plasma were reported.
20. Mean Residence Time (MRT) of BAX 855 [ Time Frame: Pre-infusion, 15 - 30 minutes, 3, 8, 24, 48, 72 and 96 hours post-infusion ]
    MRT of BAX 855 were reported.
21. Maximum Plasma Concentration (Cmax) of BAX 855 [ Time Frame: Pre-infusion, 15 - 30 minutes, 3, 8, 24, 48, 72 and 96 hours post-infusion ]
    Cmax of BAX 855 were reported.
22. Time to Maximum Concentration of BAX 855 in Plasma (Tmax) [ Time Frame: Pre-infusion, 15 - 30 minutes, 3, 8, 24, 48, 72 and 96 hours post-infusion ]
    Tmax of BAX 855 were reported.
23. Total Body Clearance (CL) of BAX 855 [ Time Frame: Pre-infusion, 15 - 30 minutes, 3, 8, 24, 48, 72 and 96 hours post-infusion ]
    Total body clearance of BAX 855 from blood by the kidney were reported.
24. Volume of Distribution at Steady State (Vss) [ Time Frame: Pre-infusion, 15 - 30 minutes, 3, 8, 24, 48, 72 and 96 hours post-infusion ]
    Volume of distribution was defined as the theoretical volume in which the total amount of drug was uniformly distributed to produce the desired blood concentration of a drug. Vss is the apparent volume of distribution at steadystate.
25. Incremental Recovery (IR) Over Time [ Time Frame: Baseline, Month 3, 6, 7.5, 9, 10.5, 12 (Completion or termination) ]
    Incremental recovery was calculated by BAX 855 increment (IU/dL) / BAX 855 dose (IU/kg).

Eligibility Criteria

• Ages Eligible for Study: 12 Years to 65 Years (Child, Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

INCLUSION CRITERIA:

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

  1. Participant has completed the end of study visit of a BAX 855 study or is transitioning from the ongoing Baxalta Continuation Study 261302.
  2. Participant is either receiving on-demand treatment or prophylactic treatment with BAX 855 and had an Annual Bleed Rate (ABR) of ≥ 2 documented and treated during the past 12 months.
  3. Participant is human immunodeficiency virus negative (HIV-); or HIV+ with stable disease and CD4+ count ≥ 200 cells/mm^3, as confirmed by central laboratory.
  4. Participant is willing and able to comply with the requirements of the protocol.

• Newly recruited participants (ie not transitioning from another BAX 855 study) including BAX855 naïve participants who meet ALL of the following criteria are eligible for this study:

  1. Participant has severe hemophilia A (FVIII clotting activity < 1%) as confirmed by central laboratory OR by historically documented FVIII clotting activity performed by a certified clinical laboratory, optionally supported by a FVIII gene mutation consistent with severe hemophilia A
  2. Participant has been previously treated with plasma-derived FVIII concentrates or recombinant FVIII for ≥ 150 documented exposure days (EDs)
  3. Participant is either receiving on-demand treatment or prophylactic treatment and had an annual bleeding rate of ≥ 2 documented and treated during the past 12 months.
  4. Participant has a Karnofsky performance score of ≥ 60 at screening
  5. Participant is HIV-; or HIV+ with stable disease and CD4+ count ≥ 200 cells/mm^3, as confirmed by central laboratory at screening
  6. Participant is hepatitis C virus negative (HCV-) by antibody (if positive, additional PCR testing will be performed), as confirmed by central laboratory at screening; or HCV+ with chronic stable hepatitis
  7. 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
  8. Participant is willing and able to comply with the requirements of the protocol.

EXCLUSION CRITERIA:

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

  1. Participant has developed a confirmed inhibitory antibody to FVIII with a titer of ≥ 0.6 BU using the Nijmegen modification of the Bethesda assay as determined at the central laboratory during the course of the previous BAX 855 study.
  2. Participant has been diagnosed with an acquired hemostatic defect other than hemophilia A.
  3. The participant's weight is < 35 kg or > 100 kg.
  4. Participant's platelet count is < 100,000/mL.
  5. Participant has an abnormal renal function (serum creatinine > 1.5 times the upper limit of normal).
  6. Participant has active hepatic disease with alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) levels ≥ 5 times the upper limit of normal.
  7. Participant is scheduled to receive a systemic immunomodulating drug (e.g. corticosteroid agents at a dose equivalent to hydrocortisone greater than 10 mg/day, or α-interferon) other than anti-retroviral chemotherapy during the study.
  8. Participant has a clinically significant medical, psychiatric, or cognitive illness, or recreational drug/alcohol use that, in the opinion of the investigator, would affect participant's safety or compliance.
  9. Participant is planning to take part in any other clinical study during the course of the study.
  10. Participant is a member of the team conducting this study or is in a dependent relationship with one of the study team members. Dependent relationships include close relatives (ie, children, partner/spouse, siblings, parents) as well as employees of the investigator or site personnel conducting the study.

Newly recruited participants (ie not transitioning from another BAX 855 study) 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 a history of confirmed FVIII inhibitors with a titer ≥ 0.6 Bethesda Units (BU) (as determined by the Nijmegen modification of the Bethesda assay or the assay employed with the respective cut-off in the local laboratory) 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. The participant's weight is < 35 kg or > 100 kg.
5. Participant's platelet count is < 100,000/mL.
6. Participant has known hypersensitivity towards mouse or hamster proteins, PEG or Tween 80.
7. Participant has severe chronic hepatic dysfunction [eg, ≥ 5 times upper limit of normal alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST), as confirmed by central laboratory at screening, or a documented INR > 1.5].
8. Participant has severe renal impairment (serum creatinine > 1.5 times the upper limit of normal).
9. Participant has current or recent (< 30 days) use of other pegylated drugs prior to study participation or is scheduled to use such drugs during study participation.
10. Participant is scheduled to receive during the course of the study, a systemic immunomodulating drug (e.g. corticosteroid agents at a dose equivalent to hydrocortisone greater than 10 mg/day, or α-interferon) other than anti-retroviral chemotherapy.
11. Participant has participated in another clinical study involving an IP or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study.
12. Participant has a medical, psychiatric, or cognitive illness or recreational drug/alcohol use that, in the opinion of the investigator, would affect participant safety or compliance.
13. Participant is a member of the team conducting this study or is in a dependent relationship with one of the study team members. Dependent relationships include close relatives (ie, children, partner/spouse, siblings, parents) as well as employees of the investigator or site personnel conducting the study.

Contacts and Locations

Locations

United States, Arizona

Phoenix Childrens Hospital

Phoenix, Arizona, United States, 85016-7710

Arizona Hemophilia & Thrombosis Center, located within The University of Arizona Cancer Center

Tucson, Arizona, United States, 85724

United States, California

Children's Hospital Los Angeles

Los Angeles, California, United States, 90027

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

United States, Georgia

Emory University-ECC

Atlanta, Georgia, United States, 30322

United States, Kentucky

University of Kentucky Medical Center

Lexington, Kentucky, United States, 40504

University of Louisville KCPCRU

Louisville, Kentucky, United States, 40202

United States, Louisiana

Tulane University

New Orleans, Louisiana, United States, 70112

United States, Massachusetts

Boston Children's Hospital

Boston, Massachusetts, United States, 02115

United States, Nebraska

University of Nebraska Medical Center

Omaha, Nebraska, United States, 68198-5456

United States, Texas

Gulf States Hemophilia Centre

Houston, Texas, United States, 77030

United States, Utah

University of Utah

Salt Lake City, Utah, United States, 84132

United States, Washington

University of Washington

Seattle, Washington, United States, 98104

Australia, Queensland

Royal Brisbane Women's Hospital

Herston, Queensland, Australia, 4006

Australia, Western Australia

The Perth Blood Institute

Nedlands, Western Australia, Australia, 6009

Austria

AKH - Medizinische Universität Wien

Vienna, Austria, 1090

Bulgaria

UMHAT "Sv. Georgi", EAD

Plovdiv, Bulgaria, 4002

UMHAT 'Tsaritsa Yoanna - ISUL', EAD

Sofia, Bulgaria, 1527

MHAT 'Sv. Marina', EAD, Clinic of Clinical Hematology

Varna, Bulgaria, 9010

France

CHU Nice- Service hematologie

Nice, Alpes Maritimes, France, 06200

Hôpital Morvan

Brest Cedex, Finistere, France, 29609

CHU Rennes - Hopital Pontchaillou

Rennes cedex 09, Ille Et Vilaine, France, 35033

CHU de Caen - Hôpital Côte de Nacre

Caen, France, 14003

CHU Charles Nicolle

Rouen, France, 76031

Germany

HZRM Hamophilie Zentrum Rhein Main GmbH

Mörfelden-Walldorf, Hessen, Germany, 65446

Inst. f. Experimentelle Hamatologie u. Transfusionsmedizin

Bonn, Nordrhein Westfalen, Germany, 53127

COAGULATION RESEARCH CENTRE GmbH

Duisburg, Nordrhein Westfalen, Germany, 47051

Hamophiliezentrum/Gerinnungssprechstunde

Berlin, Germany, 10249

MVZ Labor Dr. Reising-Ackermann

Leipzig, Germany, 04289

Hong Kong

University of Hong Kong

Hong Kong, Hong Kong

Prince of Wales Hospital

Shatin, Hong Kong, 00000

Hungary

Magyar Honvedseg EK

Budapest, Hungary, 1134

DE OEC Belgyógyászati Int

Debrecen, Hungary, 4032

PTE ÁOK

Pecs, Hungary, 7624

Israel

Chaim Sheba Medical Center

Tel-Hashomer, Israel, 5262000

Italy

UOC Ematologia, Azienda ULSS 8 Asolo, Regione Veneto

Castelfranco Veneto, Treviso, Italy, 31033

Presidio Osped. Ferrarotto

Catania, Italy, 90124

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milano, Italy, 20122

Umberto I Pol. di Roma-Università di Roma La Sapienza

Roma, Italy, 00144

Fondazione Policlinico Universitario A. Gemelli

Roma, Italy, 00168

AOU Citta della Salute e della Scienza - Presidio Molinette

Torino, Italy, 10126

ULSS n. 6 "Vicenza"

Vicenza, Italy, 36100

Malaysia

Hospital Ampang

Ampang, Kuala Lumpur, Malaysia, 68000

Hospital Queen Elizabeth

Kota Kinabalu, Sabah, Malaysia, 88586

Hospital Kuala Lumpur

Kuala Lumpur, Malaysia, 50586

Hospital Melaka

Melaka, Malaysia, 75400

Hospital Pulau Pinang

Pulau Pinang, Malaysia, 10450

Norway

Oslo Universitetssykehus HF

Oslo, Norway, 0372

Poland

Wojewodzki Szpital Specjalistyczny im. Mikolaja Kopernika, Klinika Hematologii

Lodz, Poland, 93-510

Alvamed

Poznań, Poland, 61-828

Instytut Hematologii Ii Transfuzjologii

Warszawa, Poland, 02-776

SP Szpital Kliniczny Nr 1 we Wroclawiu

Wroclaw, Poland, 50-367

Romania

Spitalul Clinic Judetean de Urgenta Brasov

Brasov, Romania, 500365

Institutul Oncologic ClNa.

Cluj Napoca, Romania, 400124

Singapore

National University Hospital

Singapore, Singapore, 119074

Singapore General Hospital- Parent

Singapore, Singapore, 169608

KK Women's And Children's Hospital

Singapore, Singapore, 229899

Spain

Hospital Universitari Son Espases

Palma de Mallorca, Baleares, Spain, 07010

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

Sweden

Sahlgrenska Universitetssjukhuset

Gothenburg, Sweden, S-41345

Karolinska Universitetssjukhuset

Stockholm, Sweden, 17176

Switzerland

Universitätsspital Zürich

Zürich, Switzerland, 8091

Taiwan

China Medical University Hospital

Taichung, Taiwan, 40447

Taichung Veterans General Hospital

Taichung, Taiwan, 40705

Tri-Service General Hospital

Taipei, Taiwan, 11490

Turkey

Acibadem Hastanesi

Adana, Turkey, 01130

Akdeniz University

Antalya, Turkey, 07070

Istanbul University

Istanbul, Turkey, 34098

Ege University

Izmir, Turkey, 35040

Ukraine

NChSH Okhmatdyt of MoHU Center of Children Oncohematology and Bone Marrow Transplantation

Kyiv, Ukraine, 1135

Kyiv CCH #9 Dept of Surgery City SPC of Diagnostics & Treatment of Patients with HP

Kyiv, Ukraine, 4112

SI Institute of Blood Pathology and Transfusion Medicine of NAMSU

Lviv, Ukraine, 79044

M.V. Sklifosovskyi Poltava RCH Dept of Gematology HSEIU Ukrainian Medical Stomatological Academy

Poltava, Ukraine, 36011

United Kingdom

Bristol Royal Hospital for Children

Bristol, Avon, United Kingdom, BS2 8BJ

Royal Free Hospital

London, Greater London, United Kingdom, NW3 2QG

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

Churchill Hospital

Oxford, Oxfordshire, United Kingdom, OX3 7LJ

University Hospital of Wales

Cardiff, West Glamorgan, United Kingdom, CF14 4XN

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] January 13, 2015

Study Protocol: Amendment 1  [PDF] March 20, 2015

Study Protocol: Amendment 2  [PDF] May 12, 2015

Study Protocol: Amendment 3  [PDF] September 4, 2015

Study Protocol: Amendment 5  [PDF] October 18, 2016

Statistical Analysis Plan  [PDF] September 19, 2018

More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Klamroth R, Windyga J, Radulescu V, Collins PW, Stasyshyn O, Ibrahim HM, Engl W, Tangada SD, Savage W, Ewenstein B. Rurioctocog alfa pegol PK-guided prophylaxis in hemophilia A: results from the phase 3 PROPEL study. Blood. 2021 Apr 1;137(13):1818-1827. doi: 10.1182/blood.2020005673.

• Responsible Party: Baxalta now part of Shire
• ClinicalTrials.gov Identifier: NCT02585960
• Other Study ID Numbers: 261303; 2014-005477-37 ( EudraCT Number )
• First Posted: October 26, 2015
• Results First Posted: August 26, 2019
• Last Update Posted: May 25, 2021
• Last Verified: May 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/

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