Clinical trials: Haemophilia A
Hemophilia
Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX
Primary Objective: To characterize the long-term safety and tolerability of fitusiran Secondary Objectives: To characterize the efficacy and long-term efficacy of fitusiran as assessed by the frequency of: Bleeding episodes…
- Trial name
- ATLAS-OLE (NCT03754790)
- Testing
- Fitusiran
- Where
- 79 sites in United States, Australia and 18 more
- Volunteers
- 281 people
- Sponsor
- Genzyme, a Sanofi Company
Hemophilia B
Study Evaluating BeneFIX in Patients With Haemophilia B, Previously Treated With Plasma Derived Factor IX
The purpose of this study is to collect data around the period of the conversion from plasma-derived Factor IX (pdFIX) to BeneFIX.
- Trial name
- NCT00749476
- Testing
- Factor IX recovery
- Where
- 8 sites in France
- Volunteers
- 1 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Hemophilia A
Relative Bioavailability Of Two Formulations Of Moroctocog Alfa (AF-CC)
This study is being conducted to compare how moroctocog alfa (AF-CC) acts in the body when administered as 2 different dose presentations.
- Trial name
- NCT01579903
- Testing
- moroctocog alfa (AF-CC)
- Where
- 3 sites in Bulgaria, Hungary and 1 more
- Volunteers
- 16 people
- Sponsor
- Pfizer
Hemophilia A
An Observational Study to Learn More About How Well Damoctocog Alfa Pegol Works in Previously Treated Children With Hemophilia A
This is an observational study of children with mild, moderate, or severe hemophilia A who are receiving damoctocog alfa pegol, and are between 7 to <12 years of age at the time of enrolment.
- Trial name
- HEM-POWR Kids (NCT07088458)
- Testing
- Damoctocog alfa pegol (Jivi, Bay94-9027)
- Where
- 12 sites in United States, Brazil and 9 more
- Volunteers
- 40 people
- Sponsor
- Bayer
Hemophilia B, Allergic Reactions
Study to Describe the Allergic Reactions to Factor IX in Patients With Hemophilia B
Retrospective, multicenter study in patients with hemophilia B. This study will be placed at 20 to 50 hemophilia treatment centers in North America and the EU, and is designed to estimate the frequency of Class II and III…
- Trial name
- NCT00195221
- Where
- 38 sites in United States, Austria and 7 more
- Volunteers
- 166 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Congenital Bleeding Disorder, Haemophilia A
Safety and Efficacy of Turoctocog Alfa (N8) in Prevention and On-demand Treatment of Bleeding Episodes in Subjects With Haemophilia A: An Extension to Trials NN7008-3543, NN7008-3545, NN7008-3600, NN7008-3893 and NN7008-4015
This trial is conducted in Asia, Europe, Japan, Oceania, North America and South America. The aim of the trial is to investigate the safety and efficacy of turoctocog alfa (N8) in Haemophilia A patients.
- Trial name
- NCT00984126
- Testing
- turoctocog alfa
- Where
- 57 sites in United States, Brazil and 18 more
- Volunteers
- 214 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A, Hemophilia B
A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors
Primary Objective: -To evaluate the efficacy of fitusiran compared to on-demand treatment with factor concentrates, as determined by the frequency of bleeding episodes.
- Trial name
- NCT03417245
- Testing
- fitusiran, factor concentrates
- Where
- 64 sites in United States, Australia and 17 more
- Volunteers
- 120 people
- Sponsor
- Genzyme, a Sanofi Company
Hemophilia B
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
This study will evaluate the efficacy and safety of PF-06838435 (a gene therapy drug) in adult male participants with moderately severe to severe hemophilia B (participants that have a Factor IX circulating activity of 2% or…
- Trial name
- BENEGENE-2 (NCT03861273)
- Testing
- PF-06838435/ fidanacogene elaparvovec
- Where
- 65 sites in United States, Australia and 14 more
- Volunteers
- 51 people
- Sponsor
- Pfizer
Hemophilia A
A Safety, Tolerability, and Pharmacokinetics Study of a Single Intravenous Injection of Recombinant Coagulation Factor VIII Fc - Von Willebrand Factor - XTEN Fusion Protein (rFVIIIFc-VWF-XTEN) (BIVV001) in Previously Treated Adults With Severe Hemophilia A (EXTEN-A)
The primary purpose was to assess the safety and tolerability of a single intravenous (IV) administration of BIVV001 in adult previously treated patients (PTPs) with severe hemophilia A.
- Trial name
- NCT03205163
- Testing
- Advate (Low Dose), Advate (High Dose), BIVV001 (Low Dose)
- Where
- 10 sites in United States, Japan
- Volunteers
- 16 people
- Sponsor
- Bioverativ, a Sanofi company
Hemophilia
Comparison of Efficacy, Safety and Costs of Recombinant FVIII Products Between On-demand and Secondary Prophylaxis Groups in Haemophilia A Patients
The project is an observational, multi-central, prospective, non-interventional and open-label data collection study on secondary prophylaxis with recombinant FVIII products in adolescents and adults with severe hemophilia A…
- Trial name
- NCT01817868
- Testing
- Recombinant Factor VIII (Kogenate, BAY14-2222)
- Where
- Turkey (Türkiye)
- Volunteers
- 73 people
- Sponsor
- Bayer
Hemophilia B
Dose-escalation Study to Investigate the Safety, PK, and PD of ISU304/CB2679d in Hemophilia B Patients
This study is a phase 1, open-label, multi-center, dose-escalation study to investigate the safety, pharmacokinetics and pharmacodynamics of ISU304/CB2679d in previously treated hemophilia B patients.
- Trial name
- NCT03186677
- Testing
- ISU304/CB2679d/Dalcinonacog alfa 75~150 IU/kg, BeneFIX
- Where
- 3 sites in South Korea
- Volunteers
- 11 people
- Sponsor
- ISU Abxis Co., Ltd.
Congenital Bleeding Disorder, Haemophilia A
Safety and Efficacy of Turoctocog Alfa in Previously Treated Male Children With Haemophilia A
This trial is conducted in Asia, Europe, and North and South America. The aim of this clinical trial is to investigate the safety and efficacy of turoctocog alfa (recombinant factor VIII, rFVIII (N8)) in male previously treated…
- Trial name
- guardian™ 3 (NCT01138501)
- Testing
- turoctocog alfa
- Where
- 38 sites in United States, Brazil and 11 more
- Volunteers
- 65 people
- Sponsor
- Novo Nordisk A/S
Severe Hemophilia A
Study to Investigate the Long-term Efficacy and Safety of Human-cl rhFVIII in Previously Treated Patients (PTPs)
The purpose of the study is to study the long-term efficacy, safety and tolerability of Human-cl rhFVIII in previously treated patients with severe hemophilia A.
- Trial name
- NCT01341912
- Testing
- Human-cl rhFVIII
- Where
- 2 sites in United States, Bulgaria
- Volunteers
- 3 people
- Sponsor
- Octapharma
Hemophilia B
Hemophilia B Gene Therapy With AAV8 Vector
Hemophilia B is a bleeding disease in males due to very low levels of coagulation factor IX (FIX) in the blood. The current treatment is intravenous injection of FIX clotting factor concentrates, in response to bleeding.
- Trial name
- NCT01620801
- Testing
- AAV8-hFIX19
- Where
- 3 sites in United States, Australia
- Volunteers
- 4 people
- Sponsor
- Spark Therapeutics, Inc.
Hemophilia A
Efficacy and Safety Study of SCT800 for On-demand Treatment With in Patients With Hemophilia A
In this trial safety and efficacy of SCT800 (B-domain deleted recombinant factor VIII) is being evaluated in 50 subjects, 12 to 65 years of age, with moderate to severe Hemophilia A.
- Trial name
- NCT02921945
- Testing
- SCT800
- Where
- China
- Volunteers
- 50 people
- Sponsor
- Sinocelltech Ltd.
Hemophilia
A Phase 1b Study to Assess the Safety, Tolerability, PK and PD of MG1113 in Hemophilia Patient
The purpose of this study is to assess the safety and tolerability, pharmacokinetics and pharmacodynamics of subcutaneous MG1113 in the multiple ascending dose study in patients with severe hemophilia.
- Trial name
- NCT05493631
- Testing
- MG1113
- Where
- South Korea
- Volunteers
- 15 people
- Sponsor
- GC Biopharma Corp
Hemophilia A With Inhibitor, Hemophilia A
Real-life Clinical FEIBA Samples Measured Using the Version A of the HemA EnzySystem
The goal of this observational study is to assess if the version A of the HemA EnzySystem, a novel portable coagulation testing platform, can be used in patients with hemophilia A treated with Factor VIII Bypassing Agent (FEIBA).
- Trial name
- NCT06357572
- Testing
- Thrombin generation assay (EnzySystem HemA version A - whole blood), Thrombin generation assay (Ceveron s100 (Technoclone) - fresh plasma), Additional coagulation tests
- Where
- Poland
- Volunteers
- 6 people
- Sponsor
- Enzyre B.V.
Hemophilia A, Hemophilia
Prevalence of Inhibitor Development in Hemophilia A Patients Receiving Coageight: A Multicenter Cross-sectional Observational Study
This is a cross-sectional, observational, multi-center study to assess the prevalence of inhibitor development in hemophilia A patients receiving Coageight (rFVIII-Fc, AryoGen Pharmed Co.).
- Trial name
- NCT07791836
- Testing
- efmoroctocog alfa
- Where
- 6 sites in Iran
- Volunteers
- 262 people
- Sponsor
- AryoGen Pharmed Co.
Haemophilia A With or Without Inhibitors
A Research Study Looking at Mim8 in Children With Haemophilia A With or Without Inhibitors
This study is looking at how Mim8 works compared to other medicines in children with haemophilia A, who either have inhibitors or do not have inhibitors. Mim8 is a new medicine that will be used for prevention of bleeds.
- Trial name
- NCT05306418
- Testing
- Mim8
- Where
- 52 sites in United States, Canada and 17 more
- Volunteers
- 70 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A
Safety and Efficacy of BAY94-9027 in Previously Treated Male Children With Haemophilia A
Hemophilia A is an inherited blood disorder in which one protein, Factor VIII, needed to form blood clots is missing or not present in sufficient levels.
- Trial name
- NCT01775618
- Testing
- BAY94-9027
- Where
- 37 sites in United States, Argentina and 15 more
- Volunteers
- 73 people
- Sponsor
- Bayer