Clinical trials: Hemophilia B, Severe
Hemophilia B, Hemophilia B, Moderately Severe or Severe
BeCoMe-9: A Clinical Study of BE-101 for the Treatment of Adults With Moderately Severe or Severe Hemophilia B
The BeCoMe-9 Study (BE-101-01) is a Phase 1/2, first in human, multi-center, open-label, dose-escalation study to evaluate the safety and clinical activity of a single intravenous (IV) dose of BE-101 in adults with moderately…
- Trial name
- NCT06611436
- Testing
- BE-101
- Where
- 5 sites in United States
- Volunteers
- 5 people
- Sponsor
- Be Biopharma
Hemophilia A, Hemophilia B
A Phase 1 Study of an Investigational Drug, ALN-AT3SC, in Healthy Volunteers and Hemophilia A or B Patients
The purpose of this study is to determine the safety, tolerability, and pharmacokinetics of ALN-AT3SC in healthy volunteers and Hemophilia A or B patients.
- Trial name
- NCT02035605
- Testing
- ALN-AT3SC, Sterile Normal Saline (0.9% NaCl)
- Where
- 14 sites in United States, Bulgaria and 3 more
- Volunteers
- 51 people, healthy volunteers welcome
- Sponsor
- Sanofi
Hemophilia A; Hemophilia B
A Study to Assess Pharmacokinetics and Pharmacodynamics Following Administration of BAY1093884 in Patients With Severe Hemophilia
The primary objective of this study is to assess the pharmacokinetics in patients with severe hemophilia. The secondary objective is to assess the pharmacodynamics of BAY1093884 based on tissue factor pathway inhibitor activity
- Trial name
- NCT03481946
- Testing
- BAY1093884
- Where
- Israel
- Volunteers
- 6 people
- Sponsor
- Bayer
Traumatic Hemorrhage, Coagulopathy
Factor In the Initial Resuscitation of Severe Trauma 2 Patients
Injury is the leading cause of death for people between the ages of 1-44. This is especially true in trauma patients who have bleeding complications.
- Conditions
- Traumatic Hemorrhage, Coagulopathy, Massive Hemorrhage
- Trial name
- FiiRST-2 (NCT04534751)
- Testing
- Fibrinogen + PCC, Frozen Plasma
- Where
- 6 sites in Canada
- Volunteers
- 142 people
- Sponsor
- University Health Network, Toronto
Hemophilia A, Hemophilia B
Females With Severe or Moderate Hemophilia A or B: an International Multi-center Study
This is a multi-center, international study designed to collect clinical, genetic and quality of life information on females with hemophilia, an inherited bleeding disorder.
- Trial name
- NCT00936312
- Where
- 11 sites in United States, Australia and 7 more
- Volunteers
- 200 people
- Sponsor
- Weill Medical College of Cornell University
Hemophilia
Safety and Efficacy of FVIIa to Prevent Excessive Bleeding in Hemophilia Patients With Inhibitors Undergoing Major Surgery
This is an interventional, prospective, international, multicenter, single-arm, Phase 3, and sequential efficacy and safety study in adolescents and adults with congenital hemophilia A or B with inhibitors to factor VIII (FVIII)…
- Trial name
- SCOPE HIM (NCT05695391)
- Testing
- Coagulation Factor VIIa (Recombinant)
- Where
- 15 sites in United States, Malaysia and 4 more
- Volunteers
- 2 people
- Sponsor
- Laboratoire français de Fractionnement et de Biotechnologies
Hemophilia B
Ascending Dose Study of Genome Editing by Zinc Finger Nuclease Therapeutic SB-FIX in Subjects With Severe Hemophilia B
The purpose of the study is to evaluate the safety, tolerability and effect on FIX antigen and activity levels of ascending doses of SB-FIX.
- Trial name
- NCT02695160
- Testing
- SB-FIX
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Sangamo Therapeutics
Hemophilia B
A Safety and Efficacy Study of a Recombinant Factor IX in Patients With Severe Hemophilia B
This study will examine the safety and efficacy of a Recombinant Coagulation Factor IX Albumin Fusion Protein (rIX-FP) for the control and prevention of bleeding episodes in subjects who have previously received factor…
- Trial name
- NCT01361126
- Testing
- Recombinant Coagulation Factor IX Albumin Fusion Protein
- Where
- 2 sites in Bulgaria, Israel
- Volunteers
- 17 people
- Sponsor
- CSL Behring
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
Study to Test the Safety of an Investigational Drug Given Repeatedly to Adult Men With Severe Hemophilia
The purpose of this study is to investigate the safety of a test drug to treat hemophilia in adult men.
- Trial name
- NCT03996486
- Testing
- BAY1093884
- Sponsor
- Bayer
Hemophilia A, Hemophilia B
A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors
The purpose of this study was to determine the frequency of bleeding episodes in participants receiving fitusiran as prophylactic treatment of hemophilia compared to participants who were assigned to continue with their regular…
- Trial name
- ATLAS-INH (NCT03417102)
- Testing
- fitusiran, Bypassing agents
- Where
- 58 sites in United States, Australia and 15 more
- Volunteers
- 60 people
- Sponsor
- Genzyme, a Sanofi Company
Hemophilia B
Study of Recombinant Factor IX Product, IB1001, in Previously Treated Subjects With Hemophilia B
To evaluate the safety (acute adverse effects associated with infusions, and inhibitor development), pharmacokinetics (PK), and efficacy with respect to breakthrough bleeding and control of hemorrhaging during prophylaxis of…
- Trial name
- NCT02048111
- Testing
- IB1001
- Where
- 2 sites in United States, United Kingdom
- Sponsor
- Cangene Corporation
Hemophilia
KN057 Multiple Dose Study in Moderately Severe to Severe Hemophilia
This study is designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of multiple subcutaneous doses of KN057 in subjects with hemophilia A or B, with or without inhibitors to Factor VIII…
- Trial name
- NCT05421429
- Testing
- KN057 doseⅠ, KN057 dose Ⅱ, KN057 dose Ⅲ
- Where
- 2 sites in China
- Volunteers
- 24 people
- Sponsor
- Suzhou Alphamab Co., Ltd.
Hemophilia B Without Inhibitor
Safety and Efficacy Clinical Study of Severe Hemophilia B Treatment With KL001
This is an open- label, non- randomized, uncontrolled, dose-escalation pilot study to evaluate the safety and efficacy of KL001 injection solution in hemophilia B subjects with ≤2 IU/dl residual FIX levels.
- Trial name
- NCT06125756
- Testing
- Low dose KL001, Middle dose KL001, High dose KL001
- Volunteers
- 9 people
- Sponsor
- Affiliated Hospital of Guangdong Medical University
Hemophilia A, Hemophilia B
An Open-label Extension Study of an Investigational Drug, Fitusiran, in Patients With Moderate or Severe Hemophilia A or B
Primary Objective: To evaluate the long-term safety and tolerability of fitusiran in male patients with moderate or severe hemophilia A or B Secondary Objectives: To investigate the long-term efficacy of fitusiran To characterize…
- Trial name
- NCT02554773
- Testing
- Fitusiran (SAR439774)
- Where
- 12 sites in United States, Bulgaria and 3 more
- Volunteers
- 34 people
- Sponsor
- Genzyme, a Sanofi Company
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 B
Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B
This is a phase 3, prospective, open-label, single-arm, single-dose, multicenter study investigating the efficacy, safety, and tolerability of CSL222 (AAV5-hFIXco-Padua) in adolescent male participants with severe or moderately…
- Trial name
- IX-TEND 3004 (NCT07080905)
- Testing
- CSL222 (Adeno-associated viral vector serotype 5 [AAV5]-hFIXco-Padua)
- Where
- 17 sites in United States, Australia and 6 more
- Volunteers
- 20 people
- Sponsor
- CSL Behring
Hemophilia A
Study to Test the Safety and How Well Patients With Severe Hemophilia A Respond to Treatment With BAY 2599023 (DTX 201), a Drug Therapy That Delivers a Healthy Version of the Defective Factor VIII Gene Into the Nucleus of Liver Cells Using an Altered, Non-infectious Virus (AAV) as a "Shuttle"
In this study researchers want to gather more information about safety and effectiveness of BAY 2599023 (DTX201), a drug therapy that delivers the human factor VIII gene into the human body by use of a viral vector to treat the…
- Trial name
- NCT03588299
- Testing
- BAY2599023 (DTX201)
- Where
- 13 sites in United States, Bulgaria and 4 more
- Volunteers
- 11 people
- Sponsor
- Bayer
Hemophilia B
A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B
Participants in this study have a genetic mutation, specifically in the coagulation (blood clotting) Factor 9 gene that causes severe or moderately severe hemophilia B.
- Trial name
- BEYOND-9 (NCT06379789)
- Testing
- REGV131, LNP1265
- Where
- 52 sites in United States, Australia and 7 more
- Volunteers
- 130 people
- Sponsor
- Regeneron Pharmaceuticals