Clinical trials: Haemophilia B
Hemophilia B
Post Marketing Surveillance To Observe Safety and Efficacy Of BeneFIX In Patients With Hemophilia B
To provide safety and effectiveness information of BeneFIX during the post-marketing period as required by Korea FDA regulations, to identify any potential drug related treatment factors in Korean population including: 1) Unknown…
- Trial name
- NCT00484185
- Testing
- BeneFIX (coagulation factor IX (recombinant))
- Where
- South Korea
- Volunteers
- 183 people
- Sponsor
- Pfizer
Congenital Bleeding Disorder, Haemophilia B
Efficacy and Safety of NNC-0156-0000-0009 During Surgical Procedures in Subjects With Haemophilia B
This trial is conducted in Africa, Asia, Europe and the United States of America (USA). The aim of the trial is to evaluate the safety and efficacy of NNC-0156-0000-0009 (nonacog beta pegol) during surgical procedures in patients…
- Trial name
- paradigm™ 3 (NCT01386528)
- Testing
- nonacog beta pegol
- Where
- 41 sites in United States, Austria and 15 more
- Volunteers
- 13 people
- Sponsor
- Novo Nordisk A/S
Congenital Bleeding Disorder, Haemophilia B
Safety and Efficacy of NNC-0156-0000-0009 in Haemophilia B Patients
This trial is conducted in Africa, Asia, Europe, Japan and North America. The aim of this trial is to evaluate the safety and efficacy, including pharmacokinetics (the rate at which the body eliminates the trial drug), of…
- Trial name
- paradigm™ 2 (NCT01333111)
- Testing
- nonacog beta pegol
- Where
- 43 sites in United States, Canada and 13 more
- Volunteers
- 74 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A or B
PF-06741086 Multiple Dose Study in Severe Hemophilia
This study is designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of multiple subcutaneous and/or intravenous doses of PF-06741086 in subjects with severe hemophilia.
- Trial name
- NCT02974855
- Testing
- PF-06741086
- Where
- 10 sites in United States, Chile and 4 more
- Volunteers
- 27 people
- Sponsor
- Pfizer
Hemophilia B
Evaluation of a Recombinant Factor IX Product, APVO101, in Previously-Treated Pediatric Patients With Hemophilia B
Phase 3/4, single arm, open-label study to evaluate PK, safety, and efficacy of APVO101 prophylaxis in severe or moderately severe hemophilia B subjects < 12 years of age.
- Trial name
- NCT03855280
- Testing
- APVO101
- Where
- 11 sites in Brazil, Georgia and 4 more
- Volunteers
- 21 people
- Sponsor
- Medexus Pharma, Inc.
Hemophilia B
RIXUBIS Drug Use-Result Survey (Japan)
The purpose of this survey is to understand the following items observed in the actual clinical use of RIXUBIS. 1. Unexpected adverse drug reactions 2. Occurrence of adverse drug reactions in the actual clinical use 3.
- Trial name
- NCT02937831
- Testing
- RIXUBIS
- Where
- 5 sites in Japan
- Volunteers
- 6 people
- Sponsor
- Baxalta now part of Shire
Hemophilia
A Study of Fitusiran in Severe Hemophilia A and B Patients Previously Receiving Factor or Bypassing Agent Prophylaxis
Primary Objective: To characterize the frequency of bleeding episodes (BE) while receiving fitusiran treatment, relative to the frequency of bleeding episodes while receiving factor concentrate or bypassing agent (BPA)…
- Trial name
- ATLAS-PPX (NCT03549871)
- Testing
- Fitusiran, BPA prophylaxis, Factor (FVIII or FIX) prophylaxis
- Where
- 35 sites in United States, Australia and 13 more
- Volunteers
- 80 people
- Sponsor
- Genzyme, a Sanofi Company
Hemophilia B
Study of Next-Generation Recombinant Factor IX Variant in Adult Subjects With Hemophilia B
Phase 2b, single-center, open-label study designed to evaluate the pharmacokinetics, pharmacodynamics, efficacy and safety of subcutaneous (SC) prophylaxis treatment regimens with CB2679d in 6 adult, male subjects with severe…
- Trial name
- NCT03995784
- Testing
- Coagulation Factor IX variant
- Where
- South Africa
- Volunteers
- 6 people
- Sponsor
- Catalyst Biosciences
Hemophilia B
HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients
This is an open-label, single-dose, multi-center, multinational trial to demonstrate the efficacy of AMT-061 and to further describe its safety profile. The study drug is identified as AAV5-hFIXco-Padua (AMT- 061).
- Trial name
- NCT03569891
- Testing
- AAV5-hFIXco-Padua, Factor IX (FIX)
- Where
- 33 sites in United States, Belgium and 6 more
- Volunteers
- 67 people
- Sponsor
- CSL Behring
Hemophilia A, Hemophilia B
Study of Ataluren (PTC124) in Hemophilia A and B
Hemophilia A (HA) and hemophilia B (HB) are inherited bleeding disorders caused by mutations in the gene for factor VIII (FVIII) and factor IX (FIX), respectively. These proteins are essential for blood clotting.
- Trial name
- NCT00947193
- Testing
- Ataluren
- Where
- 13 sites in United States, Canada and 2 more
- Volunteers
- 13 people
- Sponsor
- PTC Therapeutics
Hemophilia A, Hemophilia B
Understanding Hemophilia A and B Drug Dosage Administration Patterns
Study Design A prospective observational, cross-sectional epidemiological study in U.S. site-based clinical practice settings. 30 sites will enroll approximately 300 patients Participating patients - or their caregiver in the…
- Trial name
- NCT03248141
- Testing
- Hemophilia B standard half-life, Hemophilia B extended half-life, Hemophilia A standard half-life
- Where
- 3 sites in United States
- Volunteers
- 11 people
- Sponsor
- Pfizer
Hemophilia, Hemophilia B
Study Evaluating Liver Transplantation in Haemophilia Patients in Spain
This study will analyze survival and outcome of patients with severe haemophilia who undergo liver transplantation in Spain
- Trial name
- NCT00927992
- Testing
- Epidemiological Non interventional
- Volunteers
- 11 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Hemophilia B
A Phase 1/2 Study of SHP648, an Adeno-Associated Viral Vector for Gene Transfer in Hemophilia B Subjects
The purpose of this study is to evaluate the safety and dose escalation of SHP648 an adeno-associated viral vector for gene transfer in hemophilia B participants.
- Trial name
- NCT04394286
- Testing
- SHP648
- Where
- 2 sites in Spain, Turkey (Türkiye)
- Volunteers
- 2 people
- Sponsor
- Baxalta now part of Shire
Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
Research Study to Look at How Well the Drug Concizumab Works in Your Body if You Have Haemophilia With Inhibitors
This study will test how well a new medicine called concizumab works in the body of people with haemophilia A or B with inhibitors. The purpose is to show that concizumab can prevent bleeds in the body and is safe to use.
- Trial name
- explorer7 (NCT04083781)
- Testing
- Concizumab
- Where
- 99 sites in United States, Algeria and 27 more
- Volunteers
- 134 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A
Study Evaluating On-Demand Treatment Of Xyntha In Chinese Subjects
This study will evaluate the safety and efficacy of on-demand treatment with Xyntha in Chinese hemophilia A subjects.
- Trial name
- NCT00868530
- Testing
- Xyntha
- Where
- 6 sites in China
- Volunteers
- 53 people
- Sponsor
- Pfizer
Hemophilia A or B
PF-06741086 Long-term Treatment in Severe Hemophilia
This study is designed to evaluate the safety, tolerability and efficacy of long-term treatment with PF-06741086 in subjects with severe hemophilia who participated in the 3-month Phase 1b/2 B7841002 study.
- Trial name
- NCT03363321
- Testing
- PF-06741086
- Where
- 8 sites in United States, Brazil and 5 more
- Volunteers
- 20 people
- Sponsor
- Pfizer
Hemophilia B
Dose Confirmation Trial of AAV5-hFIXco-Padua
This is an open-label, single-dose, single-arm, multi-center trial, with a screening, a treatment + post-treatment follow-up phase, and a long-term follow-up phase.
- Trial name
- NCT03489291
- Testing
- AAV5-hFIXco-Padua (AMT-061)
- Where
- 4 sites in United States
- Volunteers
- 3 people
- Sponsor
- CSL Behring
Hemophilia B
Registry For Patients Treated With BeneFix In Usual Care Setting In Germany
The purpose of this observational study is to describe the incidence of adverse events among patients treated with BeneFix® in usual health care settings in Germany.
- Trial name
- NCT00714415
- Testing
- BeneFIX
- Where
- 21 sites in Austria, Germany
- Volunteers
- 80 people
- Sponsor
- Pfizer
Hemophilia B
Study Evaluating On-Demand Treatment With BeneFIX In Chinese Subjects
This study will evaluate the safety and efficacy of on-demand treatment with BeneFIX in Chinese hemophilia B subjects.
- Trial name
- NCT00866606
- Testing
- Benefix
- Where
- 6 sites in China
- Volunteers
- 35 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Reversal of Coagulopathy
An Open-label, Randomized, Multicenter Phase IIIb Study to Assess the Efficacy, Safety and Tolerance of BERIPLEX® P/N (Kcentra) Compared With Plasma for Rapid Reversal of Coagulopathy Induced by Vitamin K Antagonists in Subjects Requiring an Urgent Surgical Procedure
The purpose of this study is to evaluate efficacy, safety and tolerance of Beriplex® P/N (Kcentra) compared with plasma in regard to rapid reversal of coagulopathy induced by vitamin K antagonists in subjects who require…
- Trial name
- BE1116_3003 (NCT00803101)
- Testing
- Beriplex® P/N (Kcentra), Fresh frozen plasma
- Where
- 30 sites in United States, Belarus and 4 more
- Volunteers
- 176 people
- Sponsor
- CSL Behring