Clinical trials: Severe Haemophilia A, active
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
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 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 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
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 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 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, 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
Hemophilia A
Prevention of Bleeding in Patients With Moderate and Severe Hemophilia A Playing Sports: A Comparison Between Factor VIII and Emicizumab Prophylaxis
Hemophilia A (HA) is a genetic bleeding disorder resulting from a deficiency or absence of factor VIII (FVIII), which is necessary in the clotting process.
- Trial name
- STEP (NCT05022459)
- Testing
- Emicizumab, FVIII
- Where
- 10 sites in United States
- Volunteers
- 72 people
- Sponsor
- Wayne State University
Hemophilia A
Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults
C3731003 is a pivotal Phase 3 study to evaluate the clinical efficacy and safety of a single IV infusion of PF-07055480 / giroctocogene fitelparvovec (Recombinant AAV2/6 Human Factor VIII Gene Therapy) in adult male participants…
- Trial name
- AFFINE (NCT04370054)
- Testing
- PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy
- Where
- 45 sites in United States, Australia and 14 more
- Volunteers
- 77 people
- Sponsor
- Pfizer
Hemophilia
A Study to Test a Medicine (Fitusiran) Injected Under the Skin for Preventing Bleeding Episodes in Male Adolescent or Adult Participants With Severe Hemophilia
This is a multicenter, multinational, open-label, one-way cross-over, Phase 3, single-arm study for treatment of hemophilia. The purpose of this study is to measure the frequency of treated bleeding episodes with fitusiran in…
- Trial name
- ATLAS-NEO (NCT05662319)
- Testing
- Fitusiran, Clotting factor concentrates (CFC) or bypassing agents (BPA), Antithrombin concentrate (ATIIIC)
- Where
- 50 sites in United States, Canada and 15 more
- Volunteers
- 91 people
- Sponsor
- Sanofi
Haemophilia A
Efficacy and Safety Evaluation Study of SCT800 in Previously Untreated Hemophilia A Patients.
This is an Open Label, Uncontrolled Study to Evaluate the the Safety and Efficacy of SCT800 for Prophylaxis Treatment in Severe Previously Untreated Hemophilia Patients.
- Trial name
- NCT07437404
- Testing
- SCT800 prophylaxis and treatment for bleeding events
- Where
- China
- Volunteers
- 36 people
- Sponsor
- Sinocelltech Ltd.
Hemophilia A With Inhibitors
A Study of Elranatamab for Inhibitor Eradication in Patients With Hemophilia A
This is a prospective, single-arm, open-label, exploratory clinical study designed to evaluate the safety and efficacy of Elranatamab for inhibitor eradication in adults with moderate-to-severe hemophilia A with inhibitors.
- Trial name
- NCT07788469
- Testing
- Elranatamab
- Where
- China
- Volunteers
- 20 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
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
Hemophilia A
A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A
This study will assess the safety and tolerability of SPK-8011QQ in adult males with moderately severe to severe hemophilia A.
- Trial name
- NCT07226206
- Testing
- SPK-8011QQ
- Where
- 6 sites in United States
- Volunteers
- 5 people
- Sponsor
- Hoffmann-La Roche
Hemophilia A, Hemophilia B
Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors
Study B7841007 is an open-label extension study to assess the long-term safety, tolerability, and efficacy of prophylaxis treatment with marstacimab in participants who did not require "Early Termination" from the Phase 3 Study…
- Trial name
- NCT05145127
- Testing
- PF-06741086
- Where
- 78 sites in United States, Argentina and 22 more
- Volunteers
- 245 people
- Sponsor
- Pfizer
Haemophilia
Determinants and Consequences of the Transition to Adulthood for Adolescents With Severe Haemophilia: TRANSHEMO 2, an Ancillary Study to the TRANSHEMO Project
Haemophilia is a rare genetic disorder which, in its severe form and in the absence of treatment, can be life-threatening. Since the 1960s and the introduction of coagulation factor concentrates, the life expectancy of people…
- Trial name
- TRANSHEMO2 (NCT07418827)
- Testing
- questionnaires
- Where
- 25 sites in France
- Volunteers
- 75 people
- Sponsor
- Assistance Publique Hopitaux De Marseille
Hemophilia A
ASC618 Gene Therapy in Hemophilia A Patients
Currently, hemophilia A patients are managed with prophylactic or on-demand replacement therapy with recombinant FVIII or alternative therapeutics.
- Trial name
- NCT04676048
- Testing
- ASC618
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- ASC Therapeutics
Hemophilia A
Long-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A
Primary Objective: To evaluate the long-term safety of BIVV001 in previously treated subjects with hemophilia A Secondary Objectives: To evaluate the efficacy of BIVV001 as a prophylaxis treatment.
- Trial name
- XTEND-ed (NCT04644575)
- Testing
- efanesoctocog alfa (BIVV001)
- Where
- 85 sites in United States, Argentina and 21 more
- Volunteers
- 261 people
- Sponsor
- Bioverativ, a Sanofi company
Hemophilia A
Long-term Anticoagulation in a Patient With Severe Hemophilia A
To date, and to our knowledge, no case of severe hemophilia A patients receiving long-term anticoagulation has been published. Severe hemophilia A is a hereditary bleeding disorder characterized by a factor VIII (FVIII)…
- Trial name
- HAAC (NCT07314983)
- Where
- France
- Volunteers
- 1 people
- Sponsor
- University Hospital, Strasbourg, France