Clinical trials: Hemophilia a
Congenital Bleeding Disorder, Haemophilia A
A Single Dose Trial Investigating Safety and Local Tolerability of Ascending Doses of Long Acting Activated Recombinant Human Factor VII in Healthy Male Volunteers
This trial is conducted in Europe. The aim of this clinical trial is to investigate the safety, local tolerability and pharmacokinetic profile (the determination of the concentration of the administered medication in blood over…
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia B, Healthy
- Trial name
- NCT00951873
- Testing
- activated recombinant human factor VII, long acting
- Where
- United Kingdom
- Volunteers
- 38 people, healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S
Hemophilia
The Effect of Functional Activities and Balance Ability With Taping in Subjects of Hemophilia
Background Hemophilia is a sex-linked genetic disorders. When the joint or the muscles is bleeding, it may cause haemarthrosis, synovium, cartilage tissue thickening, joint activity (Range of Motion) decreasing and other…
- Trial name
- NCT02389205
- Testing
- kinesio group
- Volunteers
- 30 people
- Sponsor
- Tri-Service General Hospital (TSGH)
Hemophilia, Rebalancing Agents
Monitoring of Anti-TFPI in Hemophilia
During the development of anti-TFPI antibodies, thrombin generation assay (TGA) was employed using both in vitro measurements (antibodies added to blood samples) and ex vivo approaches (blood samples from patients in phase II and…
- Conditions
- Hemophilia, Rebalancing Agents, Prophylaxis
- Trial name
- EUREKA (NCT07101926)
- Testing
- blood sampling
- Where
- 2 sites in France
- Volunteers
- 11 people
- Sponsor
- Hospices Civils de Lyon
Hemophilia B
A Study to Evaluate the Safety and Efficacy of ZS801 in Adult Hemophilia B Patients
A non-randomized, open-label, dose-escalation, phase I/II study to evaluate the safety, tolerability, kinetics and efficacy of a single intravenous infusion of ZS801 in hemophilia B subjects with endogenous FIX ≤2%.
- Trial name
- NCT05641610
- Testing
- ZS801
- Where
- China
- Volunteers
- 21 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Hemophilia
Effectiveness of an Educational Program for Pain Management in Patients With Hemophilic Arthropathy
Introduction: Hemophilic arthropathy is a common complication of hemophilia, characterized by chronic pain, functional limitation, and impaired quality of life.
- Trial name
- NCT07420387
- Testing
- Educational group
- Where
- Spain
- Volunteers
- 70 people
- Sponsor
- Investigación en Hemofilia y Fisioterapia
Hemophilia A
Study Evaluating The Efficacy And Safety Of Xyntha In Children Less Than 6 Years Of Age
This study will be investigating the safety and efficacy of Xyntha (moroctocog alfa (AF-CC)) in male patients less than 6 years old.
- Trial name
- NCT00759655
- Testing
- Moroctocog alfa
- Volunteers
- 1 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Hemophilia A, Hemophilia B
Patterns of Hemophilia Care in Assiut Children Patients
Hemophilia A and B are congenital, recessive X-linked disorders caused by lack or deficiency of clotting factor VIII (FVIII) or IX (FIX), respectively.
- Trial name
- NCT05980377
- Volunteers
- 60 people
- Sponsor
- Assiut University
Hemophilia A or B
A Study to Describe Patient REal-world FitusiRan ExpeRience
This is a real-world, mixed-methods observational study conducted among adolescent (ages 12-17 years) and adult participants diagnosed with hemophilia A or B, with or without Factor VIII (FVIII) or Factor IX (FIX) inhibitors, who…
- Trial name
- PREFER (NCT07895381)
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Sanofi
Hemophilia
Are There Differences Between Carriers of Haemophilia A and B?
This study aims to develop a systematic genetic screening strategy for (potential) female carriers of haemophilia by identifying as many female carriers as possible within the families of haemophilia patients regularly followed…
- Trial name
- NCT05217992
- Testing
- Genetic assessment (hemophilia testing)
- Where
- Belgium
- Volunteers
- 900 people, healthy volunteers welcome
- Sponsor
- Cliniques universitaires Saint-Luc- Université Catholique de Louvain
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
Joint Outcome Study Continuation for Children With Severe Factor VIII Deficiency
The original Joint Outcome Study (JOS) enrolled 65 boys with hemophilia from 16 sites nationally. The subjects were randomized to one of two arms (prophylaxis or an enhanced episode-based treatment)and were followed prospectively…
- Trial name
- JOSC (NCT01000844)
- Volunteers
- 11 people
- Sponsor
- University of Colorado, Denver
Congenital Hemophilia A
Pharmacokinetic, Efficacy, and Safety Study of Recombinant Factor VIII Single Chain (rVIII-SingleChain) in Children With Severe Hemophilia A
This is an international, multicenter, open-label study to assess the efficacy, safety, and pharmacokinetic (PK) profile of rVIII-SingleChain in pediatric patients with severe hemophilia A.
- Trial name
- NCT02093897
- Testing
- rVIII-SingleChain
- Where
- 37 sites in United States, Australia and 17 more
- Volunteers
- 84 people
- Sponsor
- CSL Behring
Hemophilia B
LTFU for Gene Transfer Subjects With Hemophilia B
Several subjects enrolled in a multi-site, gene transfer clinical study to evaluate the intrahepatic administration of AAV2-hFIX16 vector for the treatment of severe hemophilia B between 2001 and 2009.
- Trial name
- NCT00515710
- Where
- 2 sites in United States
- Volunteers
- 4 people
- Sponsor
- Spark Therapeutics, Inc.
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Hemophilia, Elbow Joint Contracture
Efficacy of Strength Training in Improving Elbow Range of Motion and Function in Adults With Hemophilia
This is a research study of adults with hemophilia that have limitations in elbow joint motion that is the result of bleeding into the joint.
- Trial name
- NCT03842605
- Testing
- Strength training
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Oregon Health and Science University
Hemophilia A
Study of rFVIIIFc for Immune Tolerance Induction (ITI) in Haemophilia A Patients With Inhibitors Who Have Failed Previous ITI Therapies
The primary purpose of this study is to describe the outcome of Immune Tolerance Induction (ITI) treatment performed with rFVIIIFc within a timeframe of 60 weeks in patients with haemophilia A who have failed previous attempts at…
- Trial name
- ReITIrate (NCT03103542)
- Testing
- Recombinant coagulation factor (rFVIIIFc)
- Where
- 12 sites in United States, Canada and 5 more
- Volunteers
- 16 people
- Sponsor
- Swedish Orphan Biovitrum
Hemophilia
The Effectiveness of Recombinant Coagulation Factor IX With Recombinant Albumin (rIX-FP) in Severe Hemophilia B Patients
The pivotal clinical data support prolonged dosing intervals of up to 14 days for routine prophylaxis in hemophilia B patients. The majority of adult and pediatric patients using Idelvion for routine prophylaxis had a median…
- Trial name
- NCT04108260
- Testing
- Albutrepenonacog Alfa 1 UNT [IDELVION]
- Where
- Taiwan
- Volunteers
- 5 people
- Sponsor
- National Taiwan University Hospital
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 A, Hemophilia B
Psychometric Validation of the Hemophilia Functional Ability Scoring Tool (Hemo-FAST)
The purpose of this study is to validate the newly developed Hemophilia Functional Ability Scoring Tool (Hemo-FAST), which is a fast and simple scoring in haemophilia able to assess patient-reported functional mobility.
- Trial name
- Hemo-FAST (NCT04731701)
- Testing
- Hemophilia Joint Health Score (HJHS)
- Where
- 16 sites in France
- Volunteers
- 180 people
- Sponsor
- Swedish Orphan Biovitrum
Hemophilia A
ADYNOVATE Drug Use-Results Survey
The purpose of this survey is to understand the following items in the actual clinical use of ADYNOVATE in patients: 1. Unexpected adverse drug reactions 2. Occurrence of adverse drug reactions in the actual clinical use 3.
- Trial name
- NCT03169972
- Testing
- ADYNOVATE
- Where
- 52 sites in Japan
- Volunteers
- 135 people
- Sponsor
- Takeda