Clinical trials: Hemophilia A, active
Hemophilia B
A Trial That Evaluates Disease Characteristics in Hemophilia B Adult Male Participants Receiving Prophylaxis With Standard of Care Factor IX Protein (FIX) Replacement Therapy
This study is focused on males who have Hemophilia B and who need regular preventive treatment with factor IX protein (FIX) replacement therapy to prevent and also to control their bleeding events.
- Trial name
- Honey-B (NCT05568459)
- Testing
- Non-Interventional
- Where
- 17 sites in United States, Canada and 2 more
- Volunteers
- 11 people
- Sponsor
- Regeneron Pharmaceuticals
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, Prophylaxis of Bleeding
A Study to Learn More About the Safety of Damoctocog-alfa-pegol When Used in Routine Medical Care in Korean Participants With Hemophilia A
In this study, researchers will observe and study the data from participants with hemophilia A who receive damoctocog alfa pegol as prescribed by their doctors.
- Conditions
- Hemophilia A, Prophylaxis of Bleeding, Treatment of Bleeding
- Trial name
- NCT06222697
- Testing
- Damoctocog-alfa-pegol (Jivi, BAY94-9027)
- Where
- South Korea
- Volunteers
- 20 people
- Sponsor
- Bayer
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
Immune Thrombocytopenia, Thrombotic Thrombocytopenic Purpura
Longitudinal Cohort of Thrombosis and Hemostasis Diseases
This is a multicenter, prospective, longitudinal, observational cohort study to investigate thrombosis and hemostasis diseases in Chinese patients.
- Conditions
- Immune Thrombocytopenia, Thrombotic Thrombocytopenic Purpura, Hemophilia A, Acquired, Disseminated Intravascular Coagulation
- Trial name
- NCT06727669
- Where
- 5 sites in China
- Volunteers
- 3,000 people, healthy volunteers welcome
- Sponsor
- Peking University People's Hospital
Hemophilia A
Lentiviral FVIII Gene Therapy
This study is a Phase I trial using an advanced lentiviral vector to deliver a functional gene for human clotting factor VIII into patients with hemophilia A, to evaluate the safety and efficacy of infusion of lentiviral gene…
- Trial name
- NCT03217032
- Testing
- YUVA-GT-F801
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Shenzhen Geno-Immune Medical Institute
Hemophilia A
Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A
This Phase III clinical study will evaluate the safety and effectiveness of valoctocogene roxaparvovec in Japanese patients with severe hemophilia A.
- Trial name
- GENEr8-JPN (NCT06224907)
- Testing
- Valoctocogene roxaparvovec
- Where
- 4 sites in Japan
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- BioMarin Pharmaceutical
Hemophilia
Feasibility and Safety of Blood-Flow-Restriction Training in Patients With Hemophilia
In the course of a cross-over study design, 12 patients with hemophilic arthropathy will perform a training load on the knee extensor muscles.
- Trial name
- NCT07259356
- Testing
- Control, BFR Exercise (50% of the LOP) while Leg Press Exercise (4 Sets, All out)
- Where
- Germany
- Volunteers
- 12 people
- Sponsor
- University Hospital, Bonn
Hemophilia A
Safety and Efficacy Study of NGGT003 in Hemophilia A Patients
This is an early phase 1, open-label, single-center, dose-escalation pilot trial to evaluate the safety and efficacy of an intravenous infusion of NGGT003 in hemophilia A patients.
- Trial name
- NCT06238908
- Testing
- NGGT003
- Where
- China
- Volunteers
- 6 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
MRI, Hemophilia
MRI Role in Knee Hemophilic Arthopathy
The aim of this study is to assess role of MRI in detecting synovial, cartilaginous , osseous abnormalities ، bleeding inside knee joint and to use a system for assessing HA as support for therapeutic regimes and for monitoring…
- Conditions
- MRI, Hemophilia, Arthropathy Hemophilic, Arthropathy of Knee
- Trial name
- NCT07233122
- Volunteers
- 95 people
- Sponsor
- Assiut University
Hemophilia A
A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)
The BMN 270 clinical development program consists of multiple interventional studies designed to assess the safety and efficacy of a single infusion of BMN 270 for at least 5 years post-infusion.
- Trial name
- GENEr8-LTE (NCT05768386)
- Where
- 41 sites in United States, Australia and 10 more
- Volunteers
- 172 people
- Sponsor
- BioMarin Pharmaceutical
Hemophilia
The World Federation of Hemophilia Gene Therapy Registry
The aim of the WFH GTR is to provide a database in which long-term data on PWH who receive gene therapy from around the world, will be collected and housed.
- Trial name
- WFH GTR (NCT04883710)
- Testing
- Gene therapy
- Volunteers
- 5,000 people
- Sponsor
- World Federation of Hemophilia
Acquired Hemophilia A
A Survey of Susoctocog Alfa (Genetical Recombination) in Participants With Acquired Haemophilia A
This study is a survey in Japan of Susoctocog Alfa (Genetical Recombination) intravenous injection used to treat participants with bleeding events of acquired Haemophilia A (AHA).
- Trial name
- NCT06461533
- Testing
- Susoctocog Alfa (Genetical Recombination)
- Where
- Japan
- Volunteers
- 25 people
- Sponsor
- Takeda
Acquired Hemophilia
An Exploratory Study of Cluster of Differentiation 38 (CD38) Monoclonal Antibody Combined Corticosteroid in Acquired Hemophilia A
To evaluate the time of response, sustained remission rate and relapse rate of CD38 monoclonal antibody (Daratumumab) combined corticosteroid in the treatment of AHA.
- Trial name
- AHA2023 (NCT05849740)
- Testing
- Daratumumab and corticosteroid treatment
- Where
- China
- Volunteers
- 25 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
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)
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 A Patient
Efanesoctocog Prophylaxis Or The Optimization Of Treatment: Real-World Experience Of Taylorizing Clotting Therapy In Hemophilia A
Prophylactic management of severe Hemophilia A (HA) without inhibitors has historically relied on frequent intravenous infusions of standard or EHL FVIII concentrates or on subcutaneous emicizumab administered weekly to monthly…
- Trial name
- E-PROTECT (NCT07830693)
- Testing
- Study questionnaires, Clinical/pharmacological data collection
- Where
- 8 sites in France
- Volunteers
- 553 people
- Sponsor
- Nantes University Hospital
Hemophilia A
Gene Therapy Trial for Platelet Derived Factor VIII Production in Hemophilia A
This is a Phase I study. This research study is being conducted to find new ways to treat severe hemophilia A. This study is a gene therapy study.
- Trial name
- NCT03818763
- Testing
- Auto CD34+PBSC, transduced with a lentiviral vector encoding the B domain deleted from of human coagulation factor VIII
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Medical College of Wisconsin
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
Global Haemostatic Methods Following Administration of Bypassing Agents to Patients With Haemophilia With Inhibitors
Background The treatment of haemophilia A and B has been revolutionized by the use of factor concentrate, both as prophylaxis and to treat bleeding episodes (on-demand treatment).
- Trial name
- NCT02453542
- Where
- Sweden
- Volunteers
- 20 people
- Sponsor
- Karolinska Institutet