Clinical trials: Severe Hemophilia A, active
Severe Hemophilia A, Moderate Hemophilia A
A Study to Evaluate Overall Health, Physical Activity, and Joint Outcomes in Participants With Severe or Moderate Hemophilia A Without Factor VIII Inhibitors on Emicizumab Prophylaxis
Study MO42623 is a Phase IV, multicenter, open-label, three cohort study designed to evaluate the impact of emicizumab prophylaxis on overall health, physical activity, and joint outcomes in participants aged ≥13 and <70 years…
- Trial name
- Beyond ABR (NCT05181618)
- Testing
- Emicizumab
- Where
- 26 sites in United States, Brazil and 10 more
- Volunteers
- 136 people
- Sponsor
- Hoffmann-La Roche
Severe Hemophilia A
Post Approval Phase 4 Study to Learn More About How Safe Octocog Alfa is, and How Well it Works in Patients With Severe Hemophilia A in India
Hemophilia A is a genetic condition that makes it hard for blood to clot properly. This happens because the body does not have enough of a protein called Factor VIII, which helps stop bleeding.
- Trial name
- HEMOCT INDIA (NCT07446010)
- Testing
- Octocog alfa
- Volunteers
- 30 people
- Sponsor
- Bayer
Haemophilia A (Moderate or Severe)
Study to Provide Continued Access to Treatment for Patients Completing a Previous Trial With Efanesoctocog Alfa
This is a multinational, prospective, open-label, roll-over study in patients with haemophilia A, ≥6 years of age, who have completed participation in any of the parental studies with efanesoctocog alfa; XTEND-ed study…
- Trial name
- LIBERTY (NCT06716814)
- Testing
- Efanesoctocog alfa
- Where
- 20 sites in Bulgaria, France and 5 more
- Volunteers
- 89 people
- Sponsor
- Swedish Orphan Biovitrum
Severe Hemophilia A
A Study to Learn About How Changing Therapy From Emicizumab to Marstacimab Affects People With the Severe Hemophilia A.
The purpose of the study is to learn about safety, how the body processes marstacimab and how it works in patients with severe hemophilia A without inhibitors.
- Trial name
- NCT06703606
- Testing
- MARSTACIMAB
- Where
- 10 sites in United States, India and 1 more
- Volunteers
- 15 people
- Sponsor
- Pfizer
Severe Hemophilia A
A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Emicizumab in Participants From Birth to 12 Months of Age With Hemophilia A Without Inhibitors
This is a Phase IIIb, multicenter, open-label, single-arm study of prophylactic emicizumab in previously untreated and minimally treated patients at study enrollment from birth to ≤12 months of age with severe hemophilia A…
- Trial name
- HAVEN 7 (NCT04431726)
- Testing
- Emicizumab
- Where
- 32 sites in United States, Australia and 12 more
- Volunteers
- 55 people
- Sponsor
- Hoffmann-La Roche
Severe Hemophilia A
Study of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein (FRSW107) On-Demand Treatment
The indication for this product is to control bleeding in patients with hemophilia A (congenital deficiency of factor VIII). The primary objective: Evaluation of the efficacy of recombinant human coagulation factor VIII-Fc fusion…
- Trial name
- NCT07663903
- Testing
- FRSW107
- Where
- 24 sites in China
- Volunteers
- 60 people
- Sponsor
- Hangzhou Gensciences Biopharmaceutical Co., Ltd.
Severe Hemophilia A
Study of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein (FRSW107) as Prophylactic Treatment.
The indication for this product is to control and prophylaxis in patients with Hemophilia A (congenital Factor VIII deficiency): The Primary Objective： To evaluate the efficacy of Recombinant Human Coagulation Factor VIII-Fc…
- Trial name
- NCT07684898
- Testing
- FRSW107
- Where
- 19 sites in China
- Volunteers
- 60 people
- Sponsor
- Hangzhou Gensciences Biopharmaceutical Co., Ltd.
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Severe Hemophilia A
Nuwiq for Perioperative Management Of Patients With Haemophilia A on Emicizumab Regular Prophylaxis Study
Recombinant factor VIII for the prevention of bleeding in patients with severe haemophilia A undergoing major surgery while receiving emicizumab prophylaxis
- Trial name
- NuPOWER (NCT05935358)
- Testing
- Nuwiq
- Where
- 18 sites in United States, Croatia and 9 more
- Volunteers
- 28 people
- Sponsor
- Octapharma
Severe Hemophilia A
To Evaluate the Safety and Efficacy of GS1191-0445 Injection in the Treatment of Severe Hemophilia A
This study is a single-arm, open-label study evaluating the safety and efficacy of GS1191-0445 injection as a single dose in Chinese subjects with severe hemophilia A.
- Trial name
- NCT07548411
- Testing
- GS1191-0445 injection
- Where
- 2 sites in China
- Volunteers
- 7 people
- Sponsor
- Gritgen Therapeutics Co., Ltd.
Haemophilia A (Moderate or Severe), Hemophilia, Classic
A Study to Investigate the Course of Synovial Hypertrophy in Patients With Haemophilia A on Efanesoctocog Alfa Prophylaxis
The rationale for conducting this open-label phase 4 study is to assess whether once-weekly prophylaxis with efanesoctocog alfa (50 IU/kg) improves the disease course of existing synovial hypertrophy and prevents the risk of…
- Conditions
- Haemophilia A (Moderate or Severe), Hemophilia, Classic, Haemophilia, Hemophilia
- Trial name
- NCT06752850
- Testing
- Efanesoctocog alfa
- Where
- 10 sites in Italy, Norway and 2 more
- Volunteers
- 37 people
- Sponsor
- Swedish Orphan Biovitrum
Hemophilia A, Hemophilia A, Severe
Hemophilia A Research Program
This study longitudinally observes the intergenerational (mother-child) continuum in hemophilia A from pregnancy through early childhood.
- Conditions
- Hemophilia A, Hemophilia A, Severe, Factor VIII (FVIII), FVIII Deficiency
- Trial name
- HARP (NCT07414511)
- Where
- United States
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- University of Washington
Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia
Palliative Care Needs of Children With Rare Diseases and Their Families
The palliative care needs of family caregivers of children with rare diseases and their children are largely unmet, including the need for support to prepare for future medical decision making.
- Conditions
- Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia, Asparagine Synthetase Deficiency, CHARGE Syndrome
- Trial name
- FACE-Rare (NCT06938542)
- Testing
- Family Centered pediatric palliative care for family caregivers of children with rare diseases.
- Where
- United States
- Volunteers
- 480 people, healthy volunteers welcome
- Sponsor
- Children's National Research Institute
Hemophilia B, Severe, Hemophilia A, Severe
Liver Biopsy In Haemophilia Gene Therapy
To perform a liver biopsy in haemophilia A and B patients with endogenous FVIII:C/FIX:C expression at >1% any time after gene transfer following AAV mediated gene transfer.
- Trial name
- NCT04817462
- Testing
- Liver biopsy
- Where
- United Kingdom
- Volunteers
- 10 people
- Sponsor
- University College, London
Hemophilia A, Severe
An Open-label Study Evaluating the Efficacy, Safety, Pharmacokinetics, and Immunogenicity of SKP-0141 for the Treatment and Prophylaxis in Severe Hemophilia a Patients
This is a prospective, multicenter, open-label study to assess efficacy, safety, pharmacokinetics (PK), and immunogenicity of human plasma-derived Factor VIII (FVIII) in previously treated patients (PTPs) with severe hemophilia…
- Trial name
- NCT06738901
- Testing
- SKP-0141
- Volunteers
- 55 people
- Sponsor
- SK Plasma Co., Ltd.
Severe Hemophilia A
Study of Efficacy and Safety of FRSW107 in Pediatric Patients With Severe Hemophilia A
This study was divided into four stages: screening period, main trial period, extension period and follow-up period. In the main trial, both groups received FRSW107 prophylactic therapy.
- Trial name
- NCT06136507
- Testing
- FRSW107
- Where
- 13 sites in China
- Volunteers
- 76 people
- Sponsor
- Hangzhou Gensciences Biopharmaceutical Co., Ltd.
Severe Hemophilia A Without Inhibitor
A Study on the Bone-health Effectiveness of Applying Recombinant Factor VIII Fc (rFVIIIFc) to Patients With Hemophilia A (Prototype A)
Study Objectives* 1. Provide a systematic evaluation of the treatment outcomes in patients with hemophilia A 2. Emphasize the importance and clinical benefits of rFVIII-Fc in joint and bone health. 3.
- Trial name
- NCT05981274
- Testing
- serum biomarker.
- Where
- Taiwan
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- Tri-Service General Hospital (TSGH)
Severe Hemophilia A, Severe Hemophilia A With Inhibitor
Dynamics of the Anti-factor VIII Antibody Signature During Treatment With Emicizumab
The goal of this observational study is to learn about the changes of antibodies and inhibitors against the coagulation factor VIII in patients with severe hemophilia A receiving emicizumab therapy.
- Conditions
- Severe Hemophilia A, Severe Hemophilia A With Inhibitor, Severe Hemophilia A Without Inhibitor
- Trial name
- NAVIGATE (NCT05802836)
- Testing
- no interventions
- Where
- Germany
- Volunteers
- 100 people
- Sponsor
- Christoph Königs