Clinical trials: Blood Coagulation Disorders, Inherited
Von Willebrand Disease
Efficacy, PK, Immunogenicity and Safety of Wilate in Severe Von Willebrand Disease (VWD) Patients <6 Years of Age
The WIL-33 study aimed to determine the efficacy, pharmacokinetics, immunogenicity and safety of wilate as routine prophylaxis in up to 12 paediatric patients (eight evaluable) with severe von Willebrand Disease VWD (defined as…
- Trial name
- NCT04953884
- Testing
- Wilate
- Where
- 9 sites in United States, Czechia and 5 more
- Volunteers
- 12 people
- Sponsor
- Octapharma
Hemarthrosis, Hemophilia
Low-Cost Platelet-Rich Plasma for Hemarthropathy
Orthopedic complications can be injuries or diseases that affect the bones, muscles, and joints. Hemarthropathy is due to bleeding disorders, most commonly hemophilia, and can result in severe issues related to chronic amounts of…
- Trial name
- NCT06543368
- Testing
- Intra articular PRP Injection
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- University of Utah
Blood Coagulation Disorders, Hemophilia A
Trial to Evaluate the Efficacy and Safety of a New Full Length Recombinant Human FVIII for Hemophilia A
The study will assess the pharmacokinetics (part A) safety, tolerability, and efficacy of prophylaxis treatment (2 to 3 times a week) (part B) with BAY81-8973 over a one year period (split into two six month treatment periods).
- Trial name
- Leopold I (NCT01029340)
- Testing
- Recombinant Factor VIII (BAY81-8973), Recombinant Factor VIII (Kogenate FS, BAY14-2222)
- Where
- 60 sites in United States, Argentina and 20 more
- Volunteers
- 74 people
- Sponsor
- Bayer
Congenital Bleeding Disorder, Haemophilia A
Safety and Efficacy of Turoctocog Alfa in Prevention and Treatment of Bleeds in Previously Untreated Children With Haemophilia A
This trial is conducted in Asia, Europe and North America. The purpose of the trial is to evaluate the safety and efficacy of turoctocog alfa in prevention and treatment of bleeds in previously untreated children with haemophilia…
- Trial name
- guardian™4 (NCT01493778)
- Testing
- turoctocog alfa
- Where
- 69 sites in United States, Algeria and 16 more
- Volunteers
- 60 people
- Sponsor
- Novo Nordisk A/S
Severe Hemophilia A
Safety and Pharmacokinetics of Subcutaneous Injection of OCTA101 in Adult Patients With Severe Hemophilia A
This Phase 1/2 study will be a dose escalation study in adults in 5 cohorts (named cohorts 1, 2, 3, 5 and 6), with the main purpose to assess the safety of subcutaneous injection of OCTA101 (a human-cl rhFVIII and recombinant…
- Trial name
- NCT04046848
- Testing
- OCTA101
- Where
- Bulgaria
- Volunteers
- 36 people
- Sponsor
- Octapharma
Hemophilia A, Hemophilia B
A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors
The purpose of this study was to determine the frequency of bleeding episodes in participants receiving fitusiran as prophylactic treatment of hemophilia compared to participants who were assigned to continue with their regular…
- Trial name
- ATLAS-INH (NCT03417102)
- Testing
- fitusiran, Bypassing agents
- Where
- 58 sites in United States, Australia and 15 more
- Volunteers
- 60 people
- Sponsor
- Genzyme, a Sanofi Company
Hemophilia A, Hemophilia B
Recombinant Factor VIIa BI (rFVIIa BI) Treatment of Acute Bleeding Episodes Per an On-demand Regimen
The purpose of the study is to determine the efficacy and safety of rFVIIa BI as part of a six-month on-demand treatment regimen in hemophilia A or B subjects with inhibitors.
- Trial name
- NCT01757405
- Testing
- Recombinant Factor VIIa BI (rFVIIa BI)
- Where
- 16 sites in United States, Japan and 7 more
- Volunteers
- 40 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A
A Study of Emicizumab Administered Subcutaneously (SC) in Pediatric Participants With Hemophilia A and Factor VIII (FVIII) Inhibitors
This non-randomized, multicenter, open-label, Phase III clinical study will evaluate the efficacy, safety, and pharmacokinetics of emicizumab administered subcutaneously initially once weekly (QW) in pediatric participants with…
- Trial name
- HAVEN 2 (NCT02795767)
- Testing
- Emicizumab
- Where
- 28 sites in United States, Costa Rica and 8 more
- Volunteers
- 88 people
- Sponsor
- Hoffmann-La Roche
Severe Hemophilia A
Safety Study of Alphanate in Previously Treated Patients With Severe Hemophilia A
The purpose of this study is to determine the immunologic and overall safety associated with long-term use of Alphanate in subjects diagnosed with severe hemophilia A (Factor VIII:C less than 0.01 IU/ml), who have been previously…
- Trial name
- NCT00323856
- Testing
- Alphanate SD/HT
- Where
- 2 sites in Poland
- Volunteers
- 51 people
- Sponsor
- Grifols Biologicals, LLC
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
Hemophilia A, Von Willebrand Disease
Efficacy and Safety of IL-11 in DDAVP Unresponsive
The purpose of this study is to determine the biologic efficacy and safety of rhIL-11 when given subcutaneously in adults with moderate or mild hemophilia A or Von Willebrand disease unresponsive to DDAVP.
- Trial name
- IL-11DDAVP (NCT00994929)
- Testing
- Neumega (Oprelvekin, Interleukin 11, IL-11)
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- University of Pittsburgh
Hemophilia A and B
Multiple Escalating Dose Study of BAY1093884 in Adults With Hemophilia A or B With or Without Inhibitors
The purpose of this study was to assess the safety and tolerability of multiple doses of a human monoclonal antibody (BAY1093884) given under the skin in subjects with hemophilia A or B.
- Trial name
- NCT03597022
- Testing
- Befovacimab (BAY1093884)
- Where
- 17 sites in Australia, Austria and 9 more
- Volunteers
- 24 people
- Sponsor
- Bayer
Hemophilia A
Phase 1 Safety, Pharmacokinetics And Pharmacodynamics Study Of Recombinant Factor VIIa Variant (813d) In Adult Subjects With Hemophilia
This study hypothesizes that the study drug, PF-05280602 (at the selected doses) will be safe to administer to subjects with severe Hemophilia A or B with or without inhibitors and will demonstrate evidence of hemostatic…
- Trial name
- NCT01439971
- Testing
- PF-05280602
- Where
- 23 sites in United States, Belgium and 6 more
- Volunteers
- 29 people
- Sponsor
- Catalyst Biosciences
Hemophilia A, Hemophilia B
An Open-label Extension Study of an Investigational Drug, Fitusiran, in Patients With Moderate or Severe Hemophilia A or B
Primary Objective: To evaluate the long-term safety and tolerability of fitusiran in male patients with moderate or severe hemophilia A or B Secondary Objectives: To investigate the long-term efficacy of fitusiran To characterize…
- Trial name
- NCT02554773
- Testing
- Fitusiran (SAR439774)
- Where
- 12 sites in United States, Bulgaria and 3 more
- Volunteers
- 34 people
- Sponsor
- Genzyme, a Sanofi Company
Hemophilia A
Study Evaluating Prophylaxis Treatment & Characterizing Efficacy, Safety, & PK Of B-Domain Deleted Recombinant FVIII
The purpose of this research study is to determine the effectiveness, safety, and pharmacokinetics (PK) of moroctocog alfa (AF-CC) in previously treated subjects, who are younger than 6 years of age, with severe or moderately…
- Trial name
- NCT00543439
- Testing
- Moroctocog alfa (AF-CC)
- Where
- 28 sites in United States, Argentina and 11 more
- Volunteers
- 66 people
- Sponsor
- Pfizer
Congenital Bleeding Disorder, Haemophilia A
Efficacy and Safety of Turoctocog Alfa for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Chinese Patients With Haemophilia A
This trial is conducted in China. The aim of this trial is to evaluate the clinical efficacy of turoctocog alfa in treatment of bleeding episodes in Chinese patients with severe haemophilia A (FVIII≤1%).
- Trial name
- guardian TM 7 (NCT02938585)
- Testing
- turoctocog alfa
- Where
- 11 sites in China
- Volunteers
- 68 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A
Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A
This Phase III clinical study will evaluate the safety and effectiveness of valoctocogene roxaparvovec in combination with prophylactic corticosteroids in patients with severe hemophilia A.
- Trial name
- GENEr8-3 (NCT04323098)
- Testing
- valoctocogene roxaparvovec
- Where
- 12 sites in United States, Australia and 2 more
- Volunteers
- 22 people
- Sponsor
- BioMarin Pharmaceutical