Clinical trials: Congenital Bleeding Disorder
Congenital Bleeding Disorder, Haemophilia A
Safety and Efficacy of Turoctocog Alfa in Previously Treated Male Children With Haemophilia A
This trial is conducted in Asia, Europe, and North and South America. The aim of this clinical trial is to investigate the safety and efficacy of turoctocog alfa (recombinant factor VIII, rFVIII (N8)) in male previously treated…
- Trial name
- guardian™ 3 (NCT01138501)
- Testing
- turoctocog alfa
- Where
- 38 sites in United States, Brazil and 11 more
- Volunteers
- 65 people
- Sponsor
- Novo Nordisk A/S
Haemophilia A
Adverse Event Data Collection From the EUHASS Registry on Turoctocog Alfa Pegol
This non-interventional study concerns a safety data collection based on adverse event data from a third-party registry (European Haemophilia Safety Surveillance System, EUHASS) that includes information about adverse events from…
- Trial name
- NCT04682145
- Testing
- Turoctocog alfa pegol
- Where
- Denmark
- Volunteers
- 50 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A
Efficacy and Safety Study of a Recombinant Protein-Free Manufactured Factor VIII (rAHF-PFM) in Previously Untreated Hemophilia A Patients
The purpose of this study is to evaluate whether Antihemophilic factor, recombinant, manufactured protein-free (rAHF-PFM) is effective and safe in the treatment of hemophilia A patients who have not been treated with factor VIII…
- Trial name
- NCT00157157
- Testing
- Recombinant Antihemophilic Factor Manufactured and Formulated without Added Human or Animal Proteins (rAHF-PFM)
- Where
- 35 sites in United States, Austria and 8 more
- Volunteers
- 66 people
- Sponsor
- Baxalta now part of Shire
Acute Heart Failure
Study of Safety, Tolerability and Pharmacokinetics of Serelaxin in Japanese Acute Heart Failure (AHF) Patients
This is a multicenter, randomized, double-blind, placebo-controlled study to assess safety, tolerability and pharmacokinetics and to explore efficacy of IV infusion of 10 µg/kg/day and 30 µg/kg/day serelaxin for 48 hours compared…
- Trial name
- NCT02002702
- Testing
- Serelaxin
- Where
- 15 sites in Japan
- Volunteers
- 46 people
- Sponsor
- Novartis Pharmaceuticals
Hemophilia B
Post Marketing Observational Study of Reformulated BeneFIX
The primary objective of this observational study is to collect safety data on reformulated BeneFIX as prescribed in routine clinical practice conditions in France.
- Trial name
- NCT00835068
- Testing
- No intervention
- Where
- 17 sites in France
- Volunteers
- 58 people
- Sponsor
- Pfizer
Hemophilia B
Ascending Dose Study of Genome Editing by Zinc Finger Nuclease Therapeutic SB-FIX in Subjects With Severe Hemophilia B
The purpose of the study is to evaluate the safety, tolerability and effect on FIX antigen and activity levels of ascending doses of SB-FIX.
- Trial name
- NCT02695160
- Testing
- SB-FIX
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Sangamo Therapeutics
Haemophilia A and B With and Without Inhibitors
A Research Study on How Well Concizumab Works for You if You Have Haemophilia A or B With or Without Inhibitors
This study will test how well a new medicine called concizumab works for participants who have haemophilia A or B with or without inhibitors. The purpose is to show that concizumab can prevent bleeds and is safe to use.
- Trial name
- Explorer10 (NCT05135559)
- Testing
- Concizumab
- Where
- 91 sites in United States, Algeria and 22 more
- Volunteers
- 153 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A
Study to Establish Bioequivalence of ReFacto AF (BDDrFVIII) With Advate (FLrFVIII) in Hemophilia A
The study will consist of two parts: a safety and efficacy period in which all subjects will participate and a pharmacokinetic analysis period, in which 30 eligible subjects will participate to compare ReFacto AF and Advate…
- Trial name
- NCT00141843
- Testing
- ReFacto AF, B-Domain deleted Recombinant Factor VIII, BDDrFVIII
- Where
- 41 sites in United States, Australia and 11 more
- Volunteers
- 100 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
HIV Infections, Hemophilia A
Effect of Indinavir Plus Two Other Anti-HIV Drugs on Blood Clotting in HIV-Positive Males With Hemophilia
The purpose of this study is to see if indinavir plus two other anti-HIV drugs affect blood clotting in HIV-positive patients with hemophilia.
- Trial name
- NCT00002386
- Testing
- Indinavir sulfate, Lamivudine, Stavudine
- Where
- 8 sites in United States, Canada
- Volunteers
- 55 people
- Sponsor
- Merck Sharp & Dohme LLC
Hemophilia A
Study Evaluating Refacto For Pharmacovigilance
The purpose of this study is to investigate the effectiveness and safety of treatment with ReFacto under conditions of routine therapy. Furthermore a continuous benefit/risk assessment will be done.
- Trial name
- NCT00195442
- Testing
- Moroctocog alfa
- Where
- 26 sites in Austria, Germany
- Volunteers
- 288 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of 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
Congenital Bleeding Disorder, Haemophilia A
Investigating Safety and Pharmacokinetics of NNC 0128-0000-2011 Compared to NNC 0128-0000-2021 in Healthy Male Subjects
This trial is conducted in Europe. The aim of this trial is to investigate the safety and pharmacokinetics (the rate at which the body eliminates the trial drug) of NNC 0128-0000-2011 compared to NNC 0128-0000-2021 when given for…
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia B
- Trial name
- NCT01272206
- Testing
- NNC 0128-0000-2011, NNC 0128-0000-2021
- Where
- United Kingdom
- Volunteers
- 12 people
- Sponsor
- Novo Nordisk A/S
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
BAX 855 Continuation
To continue the evaluation of the safety and efficacy of BAX 855 for prophylaxis and treatment of bleeding episodes in adult and pediatric previously treated patients (PTPs) aged ≤ 75 years of age with severe hemophilia A.
- Trial name
- NCT01945593
- Testing
- BAX855
- Where
- 89 sites in United States, Australia and 21 more
- Volunteers
- 218 people
- Sponsor
- Baxalta now part of Shire
Von Willebrand Disease
Registry of Patients With Von WilLEbrand Disease Treated With Voncento®
Description of the long-term evolution of patients with von Willebrand disease and treated with Voncento® and of the hemostatic efficacy in the prevention and the treatment of non-surgical bleeding episodes and prevention of…
- Trial name
- OPALE (NCT04657887)
- Testing
- Voncento®
- Where
- 17 sites in France
- Volunteers
- 135 people
- Sponsor
- CSL Behring
Von Willebrand's Disease (VWD)
To Assess the Pharmacokinetics and Safety and Tolerability of Efanesoctocog Alfa (BIVV001)in Adults With Type 2N and 3 Von Willebrand Disease (VWD)
Primary Objective: -To characterize the pharmacokinetics (PK) of BIVV001 after a single intravenous (IV) administration, as assessed by factor VIII (FVIII) activity determined by the one-stage activated partial thromboplastin…
- Trial name
- NCT04770935
- Testing
- efanesoctocog alfa (BIVV001)
- Where
- 4 sites in United States, France
- Volunteers
- 6 people
- Sponsor
- Bioverativ, a Sanofi company
Vascular Anomalies, Hemorrhage
Safety and Efficacy Of Amber Peripheral Liquid Embolic System
A prospective, single-arm, multicenter, open-label, First-in-Human \& Pivotal Study to assess the safety and efficacy of amber SEL-P in 70 patients requiring peripheral embolization: vascular anomalies, hemorrhages, aneurysms…
- Conditions
- Vascular Anomalies, Hemorrhage, Aneurysm, Pseudoaneurysm
- Trial name
- OPAL (NCT06456125)
- Testing
- amber SEL-P Peripheral Liquid Embolic System
- Where
- Spain
- Volunteers
- 70 people
- Sponsor
- LVD Biotech S.L
Von Willebrand Disease
A Study of the Pharmacokinetics, Pharmacodynamics, and Safety of ARC1779 Injection in Patients With Von Willebrand Disease Type 2B
To evaluate the effect of ARC1779, a therapeutic oligonucleotide ("aptamer") in patients with Type2B von Willebrand Disease.
- Trial name
- NCT00694785
- Testing
- ARC1779
- Volunteers
- 2 people
- Sponsor
- Archemix Corp.
Hemophilia A
Evaluate Efficacy and Safety of Recombinant Factor VIII （rFVIII）Treatment of Severe or Moderately Severe Hemophilia A
Efficacy, Safety and Pharmacokinetics Study of a rFVIII in Chinese subjects with Hemophilia A.To assess efficacy and safety of rFVIII administered as treatment and as on-demand therapy in adult and adolescent (12-65 years)…
- Trial name
- NCT02930317
- Testing
- Recombinant Factor VIII (50 IU/kg), Recombinant Factor VIII (On-demand treatment)
- Where
- 4 sites in China
- Volunteers
- 60 people
- Sponsor
- Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
Von Willebrand Diseases
Bleeding Incidence in VWD Patients Undergoing On-Demand Treatment
The purpose of this study is to prospectively obtain reliable data on the bleeding and treatment pattern of patients with VWD undergoing on-demand treatment with a VWF-containing product over a period of 6 months.
- Trial name
- NCT04053699
- Testing
- Von Willebrand Factor-Containing Product
- Where
- 15 sites in United States, Belarus and 6 more
- Volunteers
- 56 people
- Sponsor
- Octapharma