Clinical trials: Hemophilia a
Chronic Pain, Hemophilia
Chronic Pain and Hemophilia
Hemophilia (A and B) are X-linked hereditary bleeding disorders whose severity depends on the level of coagulation factor (FVIII or FIX respectively). Bleeding is mainly from joints (hemarthrosis) and muscles (hematoma).
- Trial name
- HEMODOL (NCT05202951)
- Testing
- questionnaires
- Where
- 10 sites in France
- Volunteers
- 350 people
- Sponsor
- University Hospital, Clermont-Ferrand
Severe Hemophilia
Study to Evaluate Safety and Tolerability of a Single Dose of PF-06741086 in Chinese Adult Participants With Severe Hemophilia
This Phase 1 study will be a single-arm, open-label, non-randomized, non-controlled investigation of the safety, tolerability, pharmacokinetics, and pharmacodynamics of PF-06741086 in Chinese adult participants with severe…
- Trial name
- NCT04878731
- Testing
- PF-06741086
- Where
- China
- Volunteers
- 6 people
- Sponsor
- Pfizer
Hemophilia A, Gene Therapy
Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5
This study is being conducted by BioMarin Pharmaceutical Inc. as an open label, single dose study to determine the safety of valoctocogene roxaparvovec (an Adenovirus-Associated Virus (AAV) based gene therapy vector) in severe…
- Conditions
- Hemophilia A, Gene Therapy, Clotting Disorders, Blood Disorder
- Trial name
- GENEr8-AAV5+ (NCT03520712)
- Testing
- Valoctocogene Roxaparvovec
- Where
- 9 sites in South Africa, South Korea and 2 more
- Volunteers
- 3 people
- Sponsor
- BioMarin Pharmaceutical
Blood Coagulation Disorder, Blood Coagulation Disorders, Inherited
Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Participants
The aim of this prospective, observational study is to establish a dataset on the frequency of bleeding events, as well as other characteristics of bleeding events and FVIII infusions, in patients with clinically severe…
- Conditions
- Blood Coagulation Disorder, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, Hemophilia A
- Trial name
- NCT03876301
- Testing
- Standard of Care FVIII Replacement therapy
- Where
- 11 sites in United States, Australia and 2 more
- Volunteers
- 25 people
- Sponsor
- Spark Therapeutics, Inc.
Hemophilia
Weight-based Dosing in Hemophilia A
Hemophilia A is an inherited (genetic) disease where a protein, factor VIII (FVIII), which promotes blood clotting is missing or does not work properly. Individuals with hemophilia A are at risk for bleeding.
- Trial name
- NCT02586012
- Testing
- rFVIII
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Craig Seaman
Hemophilia a, Hemophilia B
Measurement Properties in People with Hemophilia
The aim of this project is to determine the measurement properties (namely reliability and validity) of a set of clinical assessments, measuring different aspects of physical function.
- Conditions
- Hemophilia a, Hemophilia B, Musculoskeletal Complication, Measurement Error
- Trial name
- HEMOPRO (NCT06565481)
- Testing
- Measurement Properties (Reliability and Validity) of a Set of Assessments evaluating Physical Function in adult Participants with Hemophilia
- Where
- Switzerland
- Volunteers
- 50 people
- Sponsor
- University of Zurich
Hemophilia
Safety and Efficacy of STSP-0601 in Adult Patients with Hemophilia a or B with Inhibitor
This study will assess the safety and efficacy of multiple-dose of STSP-0601 for the treatment of bleeding episodes in hemophilia A or B patients with inhibitor.
- Trial name
- NCT06289166
- Testing
- STSP-0601 for Injection
- Where
- 20 sites in China
- Volunteers
- 25 people
- Sponsor
- Staidson (Beijing) Biopharmaceuticals Co., Ltd
Hemophilia A, Factor VIII Deficiency
A Study Comparing Factor Level and Inhibitor Titer Testing Results Drawn From Central Venous Lines and Venipuncture
The purpose of this research study is to see if factor levels and inhibitor levels in Hemophilia A and B subjects are accurate when they are drawn from a central venous line (CVL) instead of from a peripheral stick.
- Conditions
- Hemophilia A, Factor VIII Deficiency, Hemophilia, Hemophilia B
- Trial name
- CVL#2 (NCT02402829)
- Testing
- Peripheral Vein Blood draw
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Children's Mercy Hospital Kansas City
Hemophilia, Hemophilia A
ATHN 8: Previously Untreated Patients (PUPs) Matter Study
This is a multi-center cohort study of approximately 250 previously untreated patients (PUPs) with congenital moderate to severe hemophilia A or B in a network of up to 50 US Hemophilia Treatment Centers (HTCs).
- Conditions
- Hemophilia, Hemophilia A, Hemophilia B, Hemophilia A With Inhibitor
- Trial name
- NCT03818529
- Where
- 27 sites in United States
- Volunteers
- 237 people
- Sponsor
- American Thrombosis and Hemostasis Network
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 A
Platelet Function in Patients With Hemophilia A
Abnormalities in the gene encoding Factor VIII (FVIII) results in hemophilia A, an X-linked recessive bleeding disorder with a prevalence of 1 in 5000 males.
- Trial name
- NCT02093065
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- Boston Children's Hospital
Hemophilia A
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg
This Phase III clinical study will assess the efficacy of BMN 270 defined as FVIII activity, during weeks 49-52 following intravenous infusion of BMN 270 and assess the impact of BMN 270 on usage of exogenous FVIII replacement…
- Trial name
- GENEr8-2 (NCT03392974)
- Testing
- Valoctocogene Roxaparvovec
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- BioMarin Pharmaceutical
Hemophilia A
GAS-Hem Feasibility Study
The purpose of this study is to investigate the feasibility and acceptability of the Standardized Goal Attainment Scaling menu for Hemophilia (GAS-Hem) as a patient reported outcome (PRO) measure to monitor clinical progress in…
- Trial name
- NCT02674997
- Testing
- Factor VIII
- Where
- 4 sites in United States, Canada
- Volunteers
- 44 people
- Sponsor
- Baxalta now part of Shire
Acute Heart Failure
Study of Safety, Tolerability and Pharmacokinetics of Serelaxin in Japanese Acute Heart Failure (AHF) Patients
This is a multicenter, randomized, double-blind, placebo-controlled study to assess safety, tolerability and pharmacokinetics and to explore efficacy of IV infusion of 10 µg/kg/day and 30 µg/kg/day serelaxin for 48 hours compared…
- Trial name
- NCT02002702
- Testing
- Serelaxin
- Where
- 15 sites in Japan
- Volunteers
- 46 people
- Sponsor
- Novartis Pharmaceuticals
Hemophilia A With Inhibitors
A Study to Evaluate Efficacy of rFVIIIFc for Immune Tolerance Induction (ITI) in Severe Hemophilia A Participants With Inhibitors Undergoing the First ITI Treatment (verITI-8 Study)
The primary purpose of this study was to describe the time to tolerization (i.e., ITI success) with rFVIIIFc in participants within a maximum of 48 weeks (12 months) of ITI treatment.
- Trial name
- NCT03093480
- Testing
- rFVIIIFc
- Where
- 37 sites in United States, Belgium and 8 more
- Volunteers
- 16 people
- Sponsor
- Bioverativ, a Sanofi company
Hemophilia A With Inhibitor, Hemophilia B With Inhibitor
A Phase I of SS109 in Hemophilia A or and B With Inhibitors
This phase I study aims to evaluate the safety, PK/PD and immunogenicity of SS109 in hemophilia A or and B with inhibitors. Twenty -seven patients are enrolled in study, and divided into five dose cohorts, from 30μg/kg to…
- Conditions
- Hemophilia A With Inhibitor, Hemophilia B With Inhibitor, Factor VII Deficiency
- Trial name
- NCT05651061
- Testing
- SS109
- Where
- 10 sites in China
- Volunteers
- 27 people
- Sponsor
- Hangzhou Gensciences Biopharmaceutical Co., Ltd.
Hemophilia
Physiotherapy to Improve Balance and Walking Capacity in Hemophilia
Introduction: Hemarthrosis is the most common type of bleeding with 70-80% in patient with hemophilia (PwH) and the majority of hemarthrosis occurs in the knee and ankle joints.
- Trial name
- NCT07339410
- Testing
- Physiotherapy
- Where
- Turkey (Türkiye)
- Volunteers
- 35 people
- Sponsor
- Yuzuncu Yil University
Hemophilia A
Clinical Study to Investigate the Pharmacokinetics, Efficacy, Safety and Immunogenicity of a Recombinant FVIII in Patients With Severe Hemophilia A
This is a clinical study to investigate the pharmacokinetics, efficacy, safety and immunogenicity of human-cl rhFVIII, a newly developed human cell-line derived recombinant FVIII concentrate in previously treated patients with…
- Trial name
- NCT00989196
- Testing
- Human-cl rhFVIII, Kogenate FS
- Where
- 9 sites in United States, Bulgaria and 1 more
- Volunteers
- 22 people
- Sponsor
- Octapharma
Hemophilia A With Inhibitor, Hemophilia B With Inhibitor
Study on the Dose-response Relationship of Pharmacodynamic Parameters in Patients With Hemophilia With Inhibitors
Randomized, double-blind, single-dose, 5 ways crossover, exploratory clinical trial evaluating four different doses of AryoSeven (eptacog alfa, activated) and NovoSeven on selected pharmacodynamic parameters in patients with…
- Trial name
- NCT04789954
- Testing
- Eptacog alfa, activated
- Where
- Iran
- Volunteers
- 14 people
- Sponsor
- AryoGen Pharmed Co.
Hemophilia
Gait Examination in Patients With Hemophilia in Austria
Hemophilia is an inherited disease caused by deficiency in varying degrees of clotting factors VIII and IX. Depending on the percentage of clotting factor in the blood the disease is categorized as "severe" (<1%, characterized by…
- Trial name
- JOSEPHA (NCT03541811)
- Testing
- 3-d gait examination (level walking), Anthropometrics and body composition assessment, Biomarkers
- Where
- Austria
- Volunteers
- 42 people, healthy volunteers welcome
- Sponsor
- Hochschule Campus Wien (University of Applied Sciences Campus Vienna)