Clinical trials: Severe Hemophilia A
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
Prevention of Bleeding in Patients With Moderate and Severe Hemophilia A Playing Sports: A Comparison Between Factor VIII and Emicizumab Prophylaxis
Hemophilia A (HA) is a genetic bleeding disorder resulting from a deficiency or absence of factor VIII (FVIII), which is necessary in the clotting process.
- Trial name
- STEP (NCT05022459)
- Testing
- Emicizumab, FVIII
- Where
- 10 sites in United States
- Volunteers
- 72 people
- Sponsor
- Wayne State University
Hemophilia A
Personalized Prediction of Tolerance and Immunogenicity in Hemophilia
This study is designed to accurately identify the pharmacogenetic determinants of risk of Factor VIII (FVIII) inhibitor development by focusing on only a select group of Hemophilia A (HA) patients who have: (i) received a…
- Trial name
- PPTIH (NCT01626105)
- Where
- United States
- Volunteers
- 55 people
- Sponsor
- Victor J Marder, M.D.
Hemophilia A
Efficacy and Safety Study of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Pediatric Patients in Canada With Hemophilia A - A Continuation of Baxter Study 060101
The purpose of this study is to evaluate whether rAHF-PFM is safe and effective in the treatment of children with hemophilia A. The study is open to pediatric patients in Canada who completed Baxter Study 060101.
- Trial name
- NCT00189982
- Testing
- Antihemophilic Factor Manufactured and Formulated without Added Human or Animal Proteins (rAHF-PFM)
- Where
- Canada
- Volunteers
- 4 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A, Factor VIII
Personalized Prophylactic Treatment With Advate® in Severe or Moderate Haemophilia A Patients
Haemophilia A is an inherited bleeding disorder caused by a deficiency of factor VIII (FVIII). Patients with severe hemophilia A have a FVIII plasma concentration less than1 IU/dL and experience spontaneous and trauma-induced…
- Trial name
- NCT02622646
- Testing
- Recombinant VIII factor
- Where
- Spain
- Volunteers
- 25 people
- Sponsor
- Hospital Universitario La Fe
Hemophilia A
Individually Tailored Prophylaxis in Patients With Severe Hemophilia A
Patients with severe haemophilia A lack clotting factor FVIII and suffer from spontaneous and traumatic bleeds. In the absence of treatment, frequent bleeds in joints lead to severe joint destruction.
- Trial name
- OPTIPHASE (NCT00995046)
- Testing
- FVIII
- Where
- France
- Volunteers
- 5 people
- Sponsor
- Hospices Civils de Lyon
Hemophilia A
Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults
C3731003 is a pivotal Phase 3 study to evaluate the clinical efficacy and safety of a single IV infusion of PF-07055480 / giroctocogene fitelparvovec (Recombinant AAV2/6 Human Factor VIII Gene Therapy) in adult male participants…
- Trial name
- AFFINE (NCT04370054)
- Testing
- PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy
- Where
- 45 sites in United States, Australia and 14 more
- Volunteers
- 77 people
- Sponsor
- Pfizer
Hemophilia
A Study to Test a Medicine (Fitusiran) Injected Under the Skin for Preventing Bleeding Episodes in Male Adolescent or Adult Participants With Severe Hemophilia
This is a multicenter, multinational, open-label, one-way cross-over, Phase 3, single-arm study for treatment of hemophilia. The purpose of this study is to measure the frequency of treated bleeding episodes with fitusiran in…
- Trial name
- ATLAS-NEO (NCT05662319)
- Testing
- Fitusiran, Clotting factor concentrates (CFC) or bypassing agents (BPA), Antithrombin concentrate (ATIIIC)
- Where
- 50 sites in United States, Canada and 15 more
- Volunteers
- 91 people
- Sponsor
- Sanofi
Haemophilia A
Efficacy and Safety Evaluation Study of SCT800 in Previously Untreated Hemophilia A Patients.
This is an Open Label, Uncontrolled Study to Evaluate the the Safety and Efficacy of SCT800 for Prophylaxis Treatment in Severe Previously Untreated Hemophilia Patients.
- Trial name
- NCT07437404
- Testing
- SCT800 prophylaxis and treatment for bleeding events
- Where
- China
- Volunteers
- 36 people
- Sponsor
- Sinocelltech Ltd.
Hemophilia B, Hemophilia A
A Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FVIII:C≤1%)
To establish baseline prospective efficacy data of current FIX prophylaxis replacement therapy in the usual care setting of hemophilia B subjects, who are negative for nAb to AAV-Spark100, prior to the Phase 3 gene therapy study.
- Trial name
- NCT03587116
- Testing
- Standard of Care FIX Replacement therapy, Standard of Care FVIII Replacement therapy
- Where
- 75 sites in United States, Australia and 16 more
- Volunteers
- 212 people
- Sponsor
- Pfizer
Hemophilia A
Safety,Efficacy and Pharmacokinetics Evaluation of SCT800 in Previously Treated Paediatric Patients With Severe Haemophilia A.
This study is a multicenter phase III uncontrolled open-label trial to evaluate the efficacy,safety and pharmacokinetics of SCT800 in regular prophylaxis and perioperative treatment in patients (<12 years old) with severe…
- Trial name
- NCT03947320
- Testing
- Recombinant Human Coagulation FVIII
- Volunteers
- 70 people
- Sponsor
- Sinocelltech Ltd.
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
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
Severe Haemophilia A
Assess the Safety and Efficacy of Individually Tailored Prophylaxis With Human-cl rhFVIII in Patients With Severe Haemophilia A
To compare the number of breakthrough bleeds under tailored prophylaxis with Human cell line recombinant factor FVIII (Human-cl rhFVIII) with the historical bleeding rate from patients who received Human-cl rhFVIII as on demand…
- Trial name
- NCT01863758
- Testing
- Human-cl rhFVIII
- Where
- 20 sites in Austria, Bulgaria and 6 more
- Volunteers
- 66 people
- Sponsor
- Octapharma
Severe Haemophilia B
Dosage and PD Study of Eftrenonacog-alfa
The purpose of this study is to evaluate the performance of different methods for measuring factor IX activity levels in haemophilia B patients treated with eftrenonacog-alfa and assess its pharmacodynamics (PD) in a real-life…
- Trial name
- BIOPAL (NCT04590950)
- Testing
- Non interventional study
- Where
- France
- Volunteers
- 15 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Hemophilia A
An Open Label Study to Determine the Safety and Efficacy of Replacement Factor VIII Protein (Known as rFVIIIFc) in Previously Untreated Males With Severe Hemophilia A
The primary objective of the study was to evaluate the safety of rFVIIIFc (BIIB031) in previously untreated participants (PUPs) with severe hemophilia A.
- Trial name
- NCT02234323
- Testing
- rFVIIIFc
- Where
- 71 sites in United States, Australia and 12 more
- Volunteers
- 108 people
- Sponsor
- Bioverativ, a Sanofi company
Hemophilia A
Study Evaluating B-Domain Deleted Recombinant Factor VIII (BDDrFVIII, ReFacto AF) in Patients With Hemophilia A Undergoing Elective Major Surgery
This study will examine the efficacy and safety of ReFacto AF in patients with severe and moderately severe hemophilia A undergoing elective major surgery when administered by either bolus injections (BI) or continuous infusion…
- Trial name
- NCT00243659
- Testing
- ReFacto AF
- Where
- 17 sites in United States, Australia and 6 more
- Volunteers
- 30 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Severe Hemophilia A
Study of First TIME Immunotolerance Induction in Severe Hemophilia A Patients With Inhibitor at High Risk of Failure: Comparison With FVIII Concentrates With or Without Von Willebrand Factor - RES.I.S.T. Naive
This is a prospective, controlled, randomized, open label study, aimed at comparing FVIII/VWF concentrates with FVIII concentrates at 200 IU/kg daily in their ability to induce immune tolerance in Haemophilia A patients with high…
- Trial name
- RESIST NAIVE (NCT01051544)
- Testing
- FVIII Concentrates, FVIII/VWF concentrates
- Sponsor
- City of Hope Medical Center
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
Severe Hemophilia A
Study of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Subjects With Severe Hemophilia A
The study is to investigate the safety, tolerability, and pharmacokinetics (the determination of the concentration of the administered drug in blood over time) of recombinant Factor VIII Fc fusion protein (rFVIIIFc) in…
- Trial name
- NCT01027377
- Testing
- rFVIIIFc
- Where
- 7 sites in United States, Hong Kong and 1 more
- Volunteers
- 16 people
- Sponsor
- Bioverativ Therapeutics Inc.