Clinical trials: Moderate Hemophilia A
Hemophilia B, Hemophilia A
A Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FVIII:C≤1%)
To establish baseline prospective efficacy data of current FIX prophylaxis replacement therapy in the usual care setting of hemophilia B subjects, who are negative for nAb to AAV-Spark100, prior to the Phase 3 gene therapy study.
- Trial name
- NCT03587116
- Testing
- Standard of Care FIX Replacement therapy, Standard of Care FVIII Replacement therapy
- Where
- 75 sites in United States, Australia and 16 more
- Volunteers
- 212 people
- Sponsor
- Pfizer
Hemophilia B
BAX 326 Surgery Study in Hemophilia B Patients
The purpose of the study is to assess the hemostatic efficacy and safety of BAX 326 in subjects with severe (FIX level < 1%) or moderately severe (FIX level 1-2%) hemophilia B undergoing major or minor elective or emergency…
- Trial name
- NCT01507896
- Testing
- Recombinant factor IX
- Where
- 14 sites in Argentina, Bulgaria and 8 more
- Volunteers
- 30 people
- Sponsor
- Baxalta now part of Shire
Hemophilia B
Trial of AAV5-hFIX in Severe or Moderately Severe Hemophilia B
This study evaluates how safe gene therapy treatment with AAV5-hFIX is in adult patients with severe or moderately severe hemophilia B and severe bleeding type.
- Trial name
- NCT02396342
- Testing
- AAV5-hFIX
- Where
- 7 sites in Denmark, Germany and 1 more
- Volunteers
- 10 people
- Sponsor
- CSL Behring
Hemophilia A
Prophylaxis Study of Recombinant Factor VIII Manufactured Protein-Free (rAHF-PFM) in Patients With Hemophilia A
The primary purpose of this randomized, two-arm parallel clinical study in 66 previously treated patients with severe or moderately severe hemophilia A is to compare the rate of bleeding episodes for standard prophylaxis (20-40…
- Trial name
- NCT00243386
- Testing
- Antihemophilic factor, recombinant, manufactured protein-free
- Where
- 29 sites in United States, Austria and 8 more
- Volunteers
- 82 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A
Safety & Efficacy of Encapsulated Allogeneic FVIII Cell Therapy in Haemophilia A
SIG-001-121 is a first-in-human (FIH), phase 1/2, multi-centre, open-label, dose escalation study to assess the safety, tolerability, and preliminary efficacy of SIG-001 in adults with severe or moderately severe haemophilia A…
- Trial name
- NCT04541628
- Testing
- SIG-001
- Where
- 6 sites in United States, United Kingdom
- Volunteers
- 3 people
- Sponsor
- Eli Lilly and Company
Severe Hemophilia A
Canadian Hemophilia Prophylaxis Study
Primary prophylaxis given less frequently initially, with the infusion frequency increased if needed (Escalating Dose Prophylaxis), is likely to be less expensive and associated with fewer complications than standard prophylaxis…
- Trial name
- CHPS (NCT01085344)
- Testing
- Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS)
- Where
- Canada
- Volunteers
- 56 people
- Sponsor
- The Hospital for Sick Children
Hemophilia B
Pivotal Study (Pharmacokinetics, Efficacy, Safety) of BAX 326 (rFIX) in Hemophilia B Patients
The purpose of this pivotal Phase 1/3 study is to determine the pharmacokinetic (PK) parameters, the hemostatic efficacy, and the safety of BAX 326, a recombinant factor IX, in previously treated patients (PTPs) with severe and…
- Trial name
- NCT01174446
- Testing
- BAX 326, BeneFIX
- Where
- 25 sites in Argentina, Brazil and 12 more
- Volunteers
- 86 people
- Sponsor
- Baxalta now part of Shire
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, Von Willebrand Disease
Efficacy and Safety of IL-11 in DDAVP Unresponsive
The purpose of this study is to determine the biologic efficacy and safety of rhIL-11 when given subcutaneously in adults with moderate or mild hemophilia A or Von Willebrand disease unresponsive to DDAVP.
- Trial name
- IL-11DDAVP (NCT00994929)
- Testing
- Neumega (Oprelvekin, Interleukin 11, IL-11)
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- University of Pittsburgh
Mild Hereditary Factor VIII Deficiency Disease Without Inhibitor, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor
A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Emicizumab in Participants With Mild or Moderate Hemophilia A Without FVIII Inhibitors
This is a multicenter, open-label, single-arm study designed to evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of emicizumab in participants with mild or moderate hemophilia A without inhibitors against…
- Conditions
- Mild Hereditary Factor VIII Deficiency Disease Without Inhibitor, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor, Hemophilia A
- Trial name
- HAVEN 6 (NCT04158648)
- Testing
- Emicizumab
- Where
- 22 sites in United States, Belgium and 8 more
- Volunteers
- 73 people
- Sponsor
- Hoffmann-La Roche
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
Study To Compare On-Demand Treatment To A Prophylaxis Regimen Of BeneFIX In Subjects With Moderately Severe to Severe Hemophilia B
The purpose of this study will be to determine if a once-weekly prophylaxis regimen of BeneFIX in subjects with moderately severe to severe Hemophilia B is safe and effective.
- Trial name
- NCT01335061
- Testing
- Nonacog alfa
- Where
- 17 sites in Bulgaria, Canada and 7 more
- Volunteers
- 25 people
- Sponsor
- Pfizer
Hemophilia B
BAX 326 Pediatric Study
The purpose of this study is to assess BAX 326 pharmacokinetic parameters, to evaluate its hemostatic efficacy, safety, immunogenicity, and changes in health-related quality of life in pediatric patients.
- Trial name
- NCT01488994
- Testing
- BAX326
- Where
- 11 sites in India, Poland and 4 more
- Volunteers
- 23 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A
Phase 3/4 Study of a Recombinant Protein-Free Factor VIII (rAHF-PFM): Comparison of Continuous Infusion Versus Intermittent Bolus Infusion in Hemophilia A Subjects Undergoing Major Orthopedic Surgery
The purpose of this study is to compare the hemostatic efficacy and safety of continuous infusion versus intermittent bolus infusion in the peri- and post-operative setting, employing rAHF-PFM, a recombinant antihemophilic factor…
- Trial name
- NCT00357656
- Testing
- Recombinant Protein-Free Factor VIII (rAHF-PFM)
- Where
- 36 sites in United States, Austria and 12 more
- Volunteers
- 85 people
- Sponsor
- Baxalta now part of Shire
Congenital Bleeding Disorder, Haemophilia A
Research Study to Look at Side Effects During Regular Injection With Factor VIII Medicine Named Turoctocog Alfa for a 8 Weeks Period
This study will test the well-known medicine turoctocog alfa for any side effects. The purpose is to test turoctocog alfa for any side effects in the Indian population. The participants will get turoctocog alfa.
- Trial name
- guardian 10 (NCT03449342)
- Testing
- turoctocog alfa
- Where
- 9 sites in India
- Volunteers
- 60 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A, Hemophilia B
Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B
Treatment with PF-06741086 is anticipated to demonstrate a clinically relevant advantage and/or a major contribution to patient care in comparison to current methods of treatment for hemophilia A or B because it works differently…
- Trial name
- NCT03938792
- Testing
- PF-06741086
- Where
- 62 sites in United States, Bulgaria and 18 more
- Volunteers
- 188 people
- Sponsor
- Pfizer
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
Severe Hereditary Factor VIII Deficiency Disease Without Inhibitor, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor
An Observational Study to Evaluate Physical Activity, Bleeding Incidence and Health Related Quality of Life, in Participants With Haemophilia A Without Inhibitors Receiving Standard of Care Treatment
This multicenter, non-interventional, prospective study will collect information about activity status, bleeds, health-related quality of life (HRQoL), health status, and safety in participants with moderate or severe haemophilia…
- Conditions
- Severe Hereditary Factor VIII Deficiency Disease Without Inhibitor, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor, Haemophilia A
- Trial name
- POWER (NCT04165135)
- Testing
- Standard of Care for Haemophilia A
- Where
- 17 sites in Italy
- Volunteers
- 103 people
- Sponsor
- Hoffmann-La Roche
Congenital Bleeding Disorder, Haemophilia A With Inhibitors
Observational Registry of NovoSeven® Used as On-demand Treatment of Bleeds in Patients With Haemophilia A and B With Inhibitors
This study was conducted in Africa, Europe, the Middle-East and South America. The primary objective of this registry was to observe the use of single dose and multi-dose use of activated recombinant human factor VII and to…
- Conditions
- Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
- Trial name
- ONE (NCT00703911)
- Testing
- eptacog alfa (activated)
- Where
- 16 sites in Algeria, Austria and 14 more
- Volunteers
- 102 people
- Sponsor
- Novo Nordisk A/S
Hemophilia B
Nonacog Alfa Prophylaxis And Treatment Of Bleeding Episodes In Previously Treated Patients With Hemophilia B
Nonacog alfa is indicated for the control and prevention of hemorrhagic episodes and for routine and surgical prophylaxis in patients with hemophilia B.
- Trial name
- NCT04286412
- Testing
- Nonacog alfa
- Where
- 5 sites in India
- Volunteers
- 25 people
- Sponsor
- Pfizer