Clinical trials: Haemophilia
Congenital Bleeding Disorder, Haemophilia A
Epidemiological Study on Haemophilia Care and Orthopaedic Status in Developing Countries
This study is conducted in Africa and Asia. The aim of this study is to evaluate in the participating countries the orthopaedic status and the degree of arthropathy of severe haemophilia patients in general.
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia A With Inhibitors, Haemophilia B
- Trial name
- HAEMOcare (NCT01503567)
- Testing
- No treatment given
- Where
- 4 sites in India, Morocco and 2 more
- Volunteers
- 282 people
- Sponsor
- Novo Nordisk A/S
Hemophilia B
Lead-in Study of VGB-R04 Gene Therapy for Hemophilia B-- An Observational Survey Analysis Study
This is a prospective, open, multicenter, observational lead-in study，to collect prospective efficacy and safety data of current replacement therapy in adult hemophilia B patients.
- Trial name
- NCT05442528
- Volunteers
- 20 people
- Sponsor
- Shanghai Vitalgen BioPharma Co., Ltd.
Hemophilia B
Dose Confirmation Trial of AAV5-hFIXco-Padua
This is an open-label, single-dose, single-arm, multi-center trial, with a screening, a treatment + post-treatment follow-up phase, and a long-term follow-up phase.
- Trial name
- NCT03489291
- Testing
- AAV5-hFIXco-Padua (AMT-061)
- Where
- 4 sites in United States
- Volunteers
- 3 people
- Sponsor
- CSL Behring
Hemophilia
Hemophilia Mobile App Usability Pilot
The purpose of this study is to evaluate the utility and user experience of a smart phone app for people with medical conditions, used in conjunction with an ActiGraph wearable device and a connected scale.
- Trial name
- NCT03168685
- Testing
- SureSource Engage application
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Healthcare Innovation Technology Lab
Haemostasis, Congenital Bleeding Disorder
Bridging Hemophilia B Experiences, Results and Opportunities Into Solutions (B-HERO-S)
This study is conducted in the United States of America (USA). Tha aim of this study is bridging Hemophilia B Experiences, Results and Opportunities into Solutions (B-HERO-S).
- Conditions
- Haemostasis, Congenital Bleeding Disorder, Haemophilia B
- Trial name
- NCT02568202
- Testing
- No treatment given
- Where
- United States
- Volunteers
- 449 people
- Sponsor
- Novo Nordisk A/S
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
BAX 802 in CHA With Inhibitors
The purpose of this study is to evaluate the efficacy and safety of BAX 802 in males with congenital hemophilia A (CHA) with inhibitors who are undergoing major or minor elective surgical, dental, or other invasive procedures.
- Trial name
- CHAWI (NCT02895945)
- Testing
- Antihemophilic Factor (Recombinant), Porcine Sequence (BAX 802)
- Where
- 24 sites in United States, Bulgaria and 10 more
- Volunteers
- 8 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A
Safety, Efficacy and PK of BIVV001 in Pediatric Patients With Hemophilia A
Primary Objective: To evaluate the safety of BIVV001 in previously treated pediatric participants with hemophilia A. Secondary Objectives: To evaluate the efficacy of BIVV001 as a prophylaxis treatment.
- Trial name
- XTEND-Kids (NCT04759131)
- Testing
- efanesoctocog alfa (BIVV001)
- Where
- 40 sites in United States, Australia and 13 more
- Volunteers
- 74 people
- Sponsor
- Bioverativ, a Sanofi company
Hemophilia A
Study of Biostate® in Children With Hemophilia A
The objective of this study is to assess the efficacy and safety of a Von Willebrand Factor/Factor VIII (VWF/FVIII), Biostate, and to investigate the pharmacokinetics of Biostate in children with haemophilia A.
- Trial name
- NCT01229007
- Testing
- Biostate
- Where
- 8 sites in Belarus, Georgia and 4 more
- Volunteers
- 35 people
- Sponsor
- CSL Behring
Hemophilia A
Moroctocog Alfa (AF-CC) for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Hemophilia A Patients
Moroctocog-alfa (AF-CC) is indicated for the control and prevention of hemorrhagic episodes and for routine and surgical prophylaxis in patients with hemophilia A (congenital factor VIII deficiency or classic hemophilia).
- Trial name
- NCT04396639
- Testing
- Moroctocog-alfa (AF-CC)
- Where
- 5 sites in India
- Volunteers
- 50 people
- Sponsor
- Pfizer
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
Assessment of the Risk of Inhibitor Formation in Previously Treated Patients With Severe Hemophilia A
Most transient inhibitor formation, if any, will develop within the first 4 weeks. The study is to further monitor whether participants with severe Hemophilia A will develop inhibitors or antibodies at the later stage when…
- Trial name
- NCT00621673
- Testing
- Kogenate (BAY 14-2222)
- Where
- 2 sites in United States
- Volunteers
- 1 people
- Sponsor
- Bayer
Haemophilia B
A Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B in France
Alprolix (rFIXFc) is a recombinant extended half-life coagulation factor product. The purpose of this non-interventional study is to describe the real-world usage and effectiveness of Alprolix in the on-demand and prophylactic…
- Trial name
- B-SURE (NCT03655340)
- Testing
- Alprolix
- Where
- 18 sites in France
- Volunteers
- 91 people
- Sponsor
- Swedish Orphan Biovitrum
Severe Hereditary Factor VIII Deficiency Disease Without Inhibitor, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor
An Observational Study to Evaluate Physical Activity, Bleeding Incidence and Health Related Quality of Life, in Participants With Haemophilia A Without Inhibitors Receiving Standard of Care Treatment
This multicenter, non-interventional, prospective study will collect information about activity status, bleeds, health-related quality of life (HRQoL), health status, and safety in participants with moderate or severe haemophilia…
- Conditions
- Severe Hereditary Factor VIII Deficiency Disease Without Inhibitor, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor, Haemophilia A
- Trial name
- POWER (NCT04165135)
- Testing
- Standard of Care for Haemophilia A
- Where
- 17 sites in Italy
- Volunteers
- 103 people
- Sponsor
- Hoffmann-La Roche
Hemophilia
Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX
Primary Objective: To characterize the long-term safety and tolerability of fitusiran Secondary Objectives: To characterize the efficacy and long-term efficacy of fitusiran as assessed by the frequency of: Bleeding episodes…
- Trial name
- ATLAS-OLE (NCT03754790)
- Testing
- Fitusiran
- Where
- 79 sites in United States, Australia and 18 more
- Volunteers
- 281 people
- Sponsor
- Genzyme, a Sanofi Company
Hemophilia A With Inhibitor, Hemophilia A With Anti Factor VIII
Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors
This Phase I/II clinical study will evaluate the safety and efficacy of valoctocogene roxaparvovec in patients with severe haemophilia A and inhibitors to FVIII.
- Trial name
- GENEr8-INH (NCT04684940)
- Testing
- Valoctocogene roxaparvovec
- Where
- 9 sites in United States, Brazil and 5 more
- Volunteers
- 10 people
- Sponsor
- BioMarin Pharmaceutical
Hemophilia A
Study Comparing Blood Levels of ReFacto and Advante in Hemophilia A
The purpose of the study is to compare the pharmacokinetic parameters of ReFacto and Advate, using the chromogenetic substrate assay to measure plasma Factor VIII activity in plasma.
- Trial name
- NCT00168051
- Testing
- ReFacto, Advante
- Where
- 13 sites in United States, Belgium and 6 more
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Hemophilia B
Phase 1/2 Dose Confirmation Study of FLT180a in Hemophilia B
Study of FLT180a gene therapy in adults with Hemophilia B. Up to 9 patients will be enrolled to receive a single dose of FLT180a and be followed for 52 weeks. Results will confirm the dose for a future Phase 3 study.
- Trial name
- NCT05164471
- Testing
- verbrinacogene setparvovec
- Where
- 7 sites in United States, United Kingdom
- Volunteers
- 6 people
- Sponsor
- Spur Therapeutics
Severe Hemophilia A
Efficacy and Safety Study of Human-cl rhFVIII in PTPs With Severe Hemophilia A
This study will determine the efficacy of human-cl rhFVIII in previously treated patients with severe hemophilia A during prophylactic treatment, treatment of bleeding episodes and in surgical prophylaxis.
- Trial name
- NCT01125813
- Testing
- recombinant Factor VIII
- Where
- 11 sites in Austria, Bulgaria and 2 more
- Volunteers
- 32 people
- Sponsor
- Octapharma
Hemophilia A
Pharmacokinetic Assessment of Standard Half-Life (SHL) FVIII, Extended Half-Life (EHL) FVIII, and Efanesoctocog Alfa (BIVV001) in Severe Hemophilia A
Primary objective To assess the half-life of BIVV001, Standard Half-Life (SHL) rFVIII and Extended Half-Life (EHL) rFVIII after a single intravenous (IV) injection Secondary objectives To characterize additional pharmacokinetic…
- Trial name
- NCT05042440
- Testing
- Efanesoctocog alfa, Octocog alfa, Rurioctocog alfa pegol
- Where
- Bulgaria
- Volunteers
- 13 people
- Sponsor
- Sanofi