Clinical trials: Hemophilia
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 Bleeding Disorder, Haemophilia A With Inhibitors
PROPACT: Retrospective Prophylaxis Patient Case Collection
This study is conducted in Europe and North and South America. The primary aim of this observational study is to evaluate the frequency and pattern of bleeding episodes in haemophilia patients receiving preventative treatment…
- Conditions
- Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
- Trial name
- PROPACT (NCT00882778)
- Testing
- eptacog alfa (activated)
- Where
- 14 sites in United States, Argentina and 12 more
- Volunteers
- 86 people
- Sponsor
- Novo Nordisk A/S
Hemophilia B
Study Evaluating rFIX; BeneFIX® in Hemophilia B
The primary objective of this clinical research study is to assess the safety and efficacy of rFIX for a minimum of 6 months in previously treated patients (PTPs) with hemophilia B (FIX:C ≤2%) during standard-of-care treatment…
- Trial name
- NCT00093171
- Testing
- rFIX
- Where
- 7 sites in United States
- Volunteers
- 23 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Hemophilia
Safety and Efficacy of STSP-0601 in Adult Patients With Hemophilia A or B Without Inhibitor
This study will assess the safety,efficacy,pharmacokinetics and pharmacodynamics of multiple-dose of STSP-0601 at two dose levels for the treatment of bleeding episodes in hemophilia A or B patients without inhibitor.
- Trial name
- NCT05619926
- Testing
- STSP-0601 for Injection
- Where
- 12 sites in China
- Volunteers
- 32 people
- Sponsor
- Staidson (Beijing) Biopharmaceuticals Co., Ltd
Hemophilia A, Hemophilia B
Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively
A study to learn about the long-term safety and efficacy of giroctocogene fitelparvovec or fidanacogene elaparvovec in patients with hemophilia A or hemophilia B respectively, who have received treatment through prior…
- Trial name
- NCT05568719
- Testing
- Testing of hepatic AAV Vector integration
- Where
- 39 sites in United States, Australia and 11 more
- Volunteers
- 173 people
- Sponsor
- Pfizer
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
Moderate or Serious Haemophilia
Objective and Perceived Health Status of Elderly People With Moderate or Severe Haemophilia in France: an Ancillary Study of the FranceCoag Registry
Background: Elderly people with serious haemophilia (PwSH), i.e., with moderate or severe haemophilia, have major comorbidities due to consequences of haemophilia. Also, the comorbidities related to age are added.
- Trial name
- SENIORHEMO (NCT05218772)
- Testing
- Questionnaires
- Where
- 28 sites in France
- Volunteers
- 277 people
- Sponsor
- Assistance Publique Hopitaux De Marseille
Hemophilia A
PHYSICAL ACTIVITY AND FVIII CLEARANCE: RELEVANCE FOR PERSONALIZED THERAPY IN SEVERE HAEMOPHILIA A (PHYSEMO)
In persons with severe haemophilia A (HA) infused factor VIII (FVIII) half-life and other pharmacokinetic parameters can vary according to determinants such as blood group, von Willebrand factor (VWF) level or age.
- Trial name
- PHYSEMO (NCT04303936)
- Testing
- SenseWear® armband device
- Where
- Italy
- Volunteers
- 20 people
- Sponsor
- Catholic University of the Sacred Heart
Prophylaxis, Hemophilia A
Comparison of Prophylaxis and On-demand Treatment in Children With Moderate to Severe Hemophilia A
Comparison of prophylaxis and on-demand treatment in children with moderate to severe hemophilia A. This study determines to compare the efficacy of prophylaxis and on-demand treatment in moderate to severe hemophilia A children…
- Trial name
- NCT02362581
- Testing
- prophylaxis treatment, on demand treatment
- Where
- Thailand
- Volunteers
- 15 people
- Sponsor
- Chulalongkorn University
Hemophilia A
Non-interventional Observation of Practical Implementation, Efficacy and Safety of Continuous Infusion With KOGENATE Bayer in Surgery
The objective of this international post-marketing surveillance study is to collect data on the efficacy and safety of continuous infusion with KOGENATE Bayer in surgery.
- Trial name
- NO PEACKS (NCT00922597)
- Testing
- Recombinant Factor VIII (Kogenate FS, BAY14-2222)
- Where
- 7 sites in Austria, Germany and 5 more
- Volunteers
- 26 people
- Sponsor
- Bayer
Congenital Bleeding Disorder, Haemophilia A
Investigating the Bioequivalence of Eptacog Alfa A 6 mg and NovoSeven® in Healthy Male Subjects
This trial is conducted in Europe. The aim of the trial is to investigate the bioequivalence of eptacog alfa A 6 mg and NovoSeven® in healthy male subjects.
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia A With Inhibitors, Haemophilia B
- Trial name
- NCT02084810
- Testing
- activated recombinant human factor VII, eptacog alfa (activated)
- Volunteers
- healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S
Hemophilia B
Safety and Tolerability of VGB-R04 in Patients With Haemophilia B
An Open-Label, Non-Randomized, uncontrolled, single-dose pilot study of VGB-R04 in subjects with Hemophilia B.
- Trial name
- NCT05152732
- Testing
- VGB-R04
- Where
- China
- Volunteers
- 3 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Hemophilia A Without Inhibitor
The Hemophilia Inhibitor Prevention Trial
This is a multi-center randomized phase III clinical trial, the Inhibitor Prevention Trial, in which Eloctate will be compared with Emicizumab, using adaptive design, to prevent inhibitors in patients with severe hemophilia A.
- Trial name
- NCT04303559
- Testing
- Eloctate Injectable Product, Emicizumab Injection [Hemlibra]
- Where
- 3 sites in United States
- Volunteers
- 1 people
- Sponsor
- Margaret Ragni
Haemophilia A Without Inhibitors, Haemophilia B Without Inhibitors
Research Study to Look at How Well the Drug Concizumab Works in Your Body if You Have Haemophilia Without Inhibitors
This study will test how well a new medicine called concizumab works in the body of people with haemophilia A or B without inhibitors. The purpose is to show that concizumab can prevent bleeds in the body and is safe to use.
- Trial name
- explorer8 (NCT04082429)
- Testing
- Concizumab
- Where
- 113 sites in United States, Algeria and 31 more
- Volunteers
- 156 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A
Pharmacokinetic Study of ADVATE Reconstituted in 2 mL Sterile Water for Injection
The purpose of this study is to determine the pharmacokinetics and safety of Antihemophilic factor, recombinant, manufactured protein-free (rAHF-PFM) reconstituted in 2 mL sterile water for injection (SWFI) and compare with those…
- Trial name
- NCT00952822
- Testing
- Antihemophilic factor, recombinant, manufactured protein-free (rAHF-PFM). (Antihemophilic factor is also known as Factor VIII)
- Where
- 11 sites in United States
- Volunteers
- 52 people
- Sponsor
- Baxalta now part of Shire
Hemophilia B, Blood Coagulation Disorders, Inherited
AAV Gene Therapy Screening/Observational Protocol (ECLIPSE)
Freeline is developing adeno-associated virus (AAV) vector based gene therapies for a number of diseases and is actively advancing a programme in Haemophilia B (HB).
- Conditions
- Hemophilia B, Blood Coagulation Disorders, Inherited, Blood Coagulation Disorder, Haematologic Disease
- Trial name
- ECLIPSE (NCT04272554)
- Where
- 56 sites in United States, Australia and 10 more
- Volunteers
- 37 people
- Sponsor
- Spur Therapeutics
Hemophilia A
Safety and Efficacy Study of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Hemophilia A Patients Undergoing Surgery
The purpose of this study is to evaluate whether rAHF-PFM is safe and effective in the treatment of hemophilia A patients undergoing surgery.
- Trial name
- NCT00157105
- Testing
- Antihemophilic factor, recombinant, manufactured protein-free
- Where
- 10 sites in United States
- Volunteers
- 59 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A
Study of a pd vWF/FVIII, Biostate®, in Subjects With Haemophilia A
The aim of this study are to assess the efficacy of Biostate® [Study Product (SP)] in subjects with Haemophilia A compare the pharmacokinetics of Biostate® [SP] with the previously marketed product Biostate® (here referred to as…
- Trial name
- NCT00879541
- Testing
- Biostate® [SP], Biostate® [RP]
- Where
- 14 sites in Bulgaria, North Macedonia and 2 more
- Volunteers
- 81 people
- Sponsor
- CSL Behring
Carrier of Hemophilia A, Desmopressin
Genetic Factors of the Desmopressin Response in Carriers of Hemophilia A
Hemophilia A (HA) is a rare X-linked bleeding disorder caused by a deficiency in factor VIII (FVIII) affecting 1/5,000 males1. Carriers of HA are females carrying the pathogenic variant responsible for the familial HA at a…
- Conditions
- Carrier of Hemophilia A, Desmopressin, Factor VIII Deficiency
- Trial name
- GIDEHAC (NCT06020456)
- Testing
- Desmopressin
- Where
- 12 sites in France
- Volunteers
- 361 people
- Sponsor
- Groupe Maladies hémorragiques de Bretagne
Haemophilia
A Clinical Phenotype Based Individualized Prophylaxis in Chinese Hemophilia A Children
In the past, due to economic and medical resource constraints, the hemophilia comprehensive care in China was suboptimal. The BCH data of both retrospective and prospective studies reveals that for 4-6y and 6-9y patients with…
- Trial name
- CHIPS (NCT02999308)
- Testing
- Observe the patient's condition and then change the regimen
- Where
- China
- Volunteers
- 40 people
- Sponsor
- Beijing Children's Hospital