Clinical trials: Hemophilia B, Severe
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
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
Severe Hemophilia B
Long-Term Safety and Efficacy of rFIXFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia B
The primary objective of the study is to evaluate the long-term safety of rFIXFc in participants with hemophilia B. The secondary objective of this study is to evaluate the efficacy of rFIXFc in the prevention and treatment of…
- Trial name
- B-YOND (NCT01425723)
- Testing
- rFIXFc
- Where
- 49 sites in United States, Australia and 16 more
- Volunteers
- 120 people
- Sponsor
- Bioverativ Therapeutics Inc.
Hemophilia A
Study Evaluating ReFacto® in Hemophilia A Undergoing Major Surgery
The primary objective of this clinical research study is to evaluate the safety and efficacy of ReFacto in subjects with hemophilia A undergoing major surgery monitored using the chromogenic substrate assay at the local…
- Trial name
- NCT00092976
- Testing
- ReFacto
- Where
- 4 sites in United States
- Volunteers
- 15 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Hemophilia B
Phase I/IIa Study of FIXFc in Hemophilia B Patients
The primary objective of the study is to assess safety of FIXFc at doses ranging from 1 to 100 IU/kg.
- Trial name
- NCT00716716
- Testing
- rFIXFc
- Where
- 6 sites in United States
- Volunteers
- 10 people
- Sponsor
- Bioverativ Therapeutics Inc.
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, Hemophilia A, Severe
Reliability and Validity of the Turkish Version of the PedHAL
This study aimed to establish the validity and reliability of the Turkish version of the Pediatric Haemophilia Activities List (short - PedHALshort) questionnaire.
- Conditions
- Hemophilia, Hemophilia A, Severe, Hemophilia B
- Trial name
- NCT05889754
- Where
- Turkey (Türkiye)
- Volunteers
- 50 people
- Sponsor
- Hasan Kalyoncu University
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 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
- NCT06119659
- Testing
- Low dose KL001, Middle dose KL001, High dose KL001
- Volunteers
- 9 people
- Sponsor
- Zhejiang University
Congenital Bleeding Disorder, Haemophilia A With Inhibitors
Safety and Efficacy of Activated Recombinant Human Factor VII in Haemophilia Patients With Inhibitors During and After Major Surgery
This trial is conducted in North America. The aim of this trial is to compare the safety and efficacy of activated recombinant human factor VII in patients with haemophilia A or B undergoing major surgical procedures.
- Conditions
- Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
- Trial name
- NCT01561391
- Testing
- activated recombinant human factor VII, factor IX, factor VIII
- Where
- 14 sites in United States
- Volunteers
- 36 people
- Sponsor
- Novo Nordisk A/S
Hemophilia B
Study Evaluating of Recombinant Human Factor IX (BeneFIX) and a New Formulation of BeneFIX (rFIX-R) in Moderate to Severe Hemophilia B
The primary objective of this clinical research study is to establish the bioequivalence of 2 treatments, rFIX and rFIX-R, when given as a 10-minute intravenous bolus infusion.
- Trial name
- NCT00093210
- Testing
- rFIX, rFIX-R
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Hemophilia B
Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B
A long-term follow-up study to evaluate the safety, tolerability, and efficacy of DTX101 in adult males with moderate/severe to severe hemophilia B.
- Trial name
- NCT02971969
- Where
- 5 sites in United States, United Kingdom
- Volunteers
- 6 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Hemophilia A, Hemophilia B
A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B
The purpose of this clinical trial is to learn about the safety and effects of the study medicine (called marstacimab) for the potential treatment of hemophilia in pediatric patients.
- Trial name
- BASIS KIDS (NCT05611801)
- Testing
- marstacimab
- Where
- 63 sites in United States, Argentina and 21 more
- Volunteers
- 100 people
- Sponsor
- Pfizer
Hemophilia B
BAX 326 (rFIX) Continuation Study
The purpose of this BAX 326 Continuation Study is to further investigate incremental recovery over time, the hemostatic efficacy, the safety, immunogenicity, and health-related quality of life (HR QoL) of BAX 326 in previously…
- Trial name
- NCT01286779
- Testing
- BAX 326 (Recombinant factor IX)
- Where
- 42 sites in Argentina, Brazil and 15 more
- Volunteers
- 117 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A, Hemophilia B
Females With Severe or Moderate Hemophilia A or B: A Multi-Center Study
To collect and analyze data on females with hemophilia so as to better define the difference between the study population and the male population with hemophilia.
- Trial name
- NCT00936845
- Where
- 19 sites in United States, Puerto Rico
- Volunteers
- 22 people
- Sponsor
- Weill Medical College of Cornell University
Hemophilia a, Hemophilia B
The Safety and Tolerability of SerpinPC in Healthy Men and in Men with Severe Blood Disorders (haemophilia a and B)
The purpose of this study is to investigate the safety and activity in the body of a new drug called SerpinPC. The study will be split into 7 parts: Part 1a will be conducted in healthy male volunteers in the UK (up to 15) and…
- Trial name
- NCT04073498
- Testing
- SerpinPC
- Where
- 3 sites in Georgia, Moldova and 1 more
- Volunteers
- 39 people, healthy volunteers welcome
- Sponsor
- ApcinteX Ltd
Haemophilia B
A Study to Investigate the Safety and Efficacy of Replenine®-VF in Haemophilia B Patients Undergoing Major Surgery.
The main objective of the study is to investigate the safety and efficacy of Replenine®-VF administered by continuous infusion in appropriate dosage to prevent bleeding and achieve haemostasis in patients with haemophilia B…
- Trial name
- NCT02250560
- Testing
- Replenine®-VF (High Purity Factor IX)
- Where
- 5 sites in United Kingdom
- Sponsor
- Bio Products Laboratory
Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1
Baby Detect : Genomic Newborn Screening
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions.
- Conditions
- Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1, Phosphoglucomutase 1 Deficiency, Maturity Onset Diabetes of the Young
- Trial name
- BabyDetect (NCT05687474)
- Where
- Belgium
- Volunteers
- 6,824 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
Hemophilia A or B
PF-06741086 Multiple Dose Study in Severe Hemophilia
This study is designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of multiple subcutaneous and/or intravenous doses of PF-06741086 in subjects with severe hemophilia.
- Trial name
- NCT02974855
- Testing
- PF-06741086
- Where
- 10 sites in United States, Chile and 4 more
- Volunteers
- 27 people
- Sponsor
- Pfizer
Hemophilia
A Study of Fitusiran in Severe Hemophilia A and B Patients Previously Receiving Factor or Bypassing Agent Prophylaxis
Primary Objective: To characterize the frequency of bleeding episodes (BE) while receiving fitusiran treatment, relative to the frequency of bleeding episodes while receiving factor concentrate or bypassing agent (BPA)…
- Trial name
- ATLAS-PPX (NCT03549871)
- Testing
- Fitusiran, BPA prophylaxis, Factor (FVIII or FIX) prophylaxis
- Where
- 35 sites in United States, Australia and 13 more
- Volunteers
- 80 people
- Sponsor
- Genzyme, a Sanofi Company