Clinical trials: Inherited Abnormalities of Platelets
Von Willebrand Disease
Study of Biostate® in Children With Von Willebrand Disease
This is an open-label study to investigate the pharmacokinetics (PK), efficacy, and safety of a von Willebrand Factor/Factor VIII (VWF/FVIII), Biostate, in children with Von Willebrand disease (VWD) in whom treatment with a VWF…
- Trial name
- NCT01213446
- Testing
- Biostate
- Where
- 7 sites in Belarus, Georgia and 4 more
- Volunteers
- 17 people
- Sponsor
- CSL Behring
Von Willebrand Disease (VWD)
Post Trial Access Program of TAK-577 for Von Willebrand Disease (VWD)
The post-trial access program allows eligible participants to gain access to unlicensed treatment on compassionate grounds. Recombinant von Willebrand factor (rVWF) also known as TAK-577, is a medicine to help treat Von…
- Trial name
- NCT06173024
- Testing
- TAK-577
- Sponsor
- Takeda
Congenital Bleeding Disorder, Congenital FVII Deficiency
Observational Study on the Efficacy and Safety of NovoSeven® During "Real-life" Usage in Germany
This NON INTERVENTIONAL OBSERVATIONAL STUDY is conducted in Europe. The primary aim is to observe the haemostatic efficacy of NovoSeven® treatment during routine practice in German clinics.
- Conditions
- Congenital Bleeding Disorder, Congenital FVII Deficiency, Glanzmann's Disease, Acquired Bleeding Disorder
- Trial name
- WIRK (NCT00697320)
- Testing
- eptacog alfa (activated)
- Where
- Germany
- Volunteers
- 64 people
- Sponsor
- Novo Nordisk A/S
Von Willebrand Disease (VWD)
A Study of Vonicog Alfa (rVWF) in Children With Severe Von Willebrand Disease (vWD)
The main aim of the study is to evaluate the effectiveness of prophylaxis with vonicog alfa (recombinant von Willebrand factor [rVWF]) in children.
- Trial name
- NCT05582993
- Testing
- Vonicog Alfa, ADVATE
- Where
- 23 sites in United States, France and 4 more
- Volunteers
- 24 people
- Sponsor
- Takeda
Von Willebrand Disease (VWD)
A Study of Recombinant Von Willebrand Factor (rVWF) in Pediatric and Adult Participants With Severe Von Willebrand Disease (VWD)
The main aim of the study is to check effectiveness of rVWF (vonicog alfa) prophylaxis based on the annualized bleeding rate (ABR) of spontaneous (not related to trauma) bleeding episodes in pediatric and adult participants…
- Trial name
- NCT03879135
- Testing
- rVWF, rFVIII
- Where
- 33 sites in United States, Austria and 7 more
- Volunteers
- 38 people
- Sponsor
- Baxalta now part of Shire
Von Willebrand Disease
Study of a pd VWF/FVIII Concentrate, Biostate®, in Subjects With Von Willebrand Disease
The aim of this study is to assess the pharmacokinetics (PK), efficacy, and safety of Biostate® in subjects with Von Willebrand Disease (VWD).
- Trial name
- NCT00941616
- Testing
- Biostate®
- Where
- 5 sites in Bulgaria, Poland and 2 more
- Volunteers
- 22 people
- Sponsor
- CSL Behring
VWD - Von Willebrand's Disease
Effectiveness and Tolerability of Eqwilate in Real-life Conditions
MOdalities of use, effectiveNEss and TOlerability of Eqwilate® a balanced combInatiON of VWF and FVIII in von WillEbrand patients in real-life conditions: the ONE-TO-ONE study
- Trial name
- NCT04106908
- Testing
- Eqwilate
- Where
- 9 sites in France
- Volunteers
- 38 people
- Sponsor
- Octapharma
Von Willebrand Disease
A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD)
The main aim of the study is to check effectiveness, side effects, and tolerability of vonicog alfa (recombinant von Willebrand factor [rVWF]), with or without ADVATE, in the treatment and control of nonsurgical bleeding events…
- Trial name
- NCT02932618
- Testing
- Vonicog alfa, Antihemophilic Factor (Recombinant)
- Where
- 46 sites in United States, Austria and 11 more
- Volunteers
- 47 people
- Sponsor
- Baxalta now part of Shire
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
Von Willebrand Disease (vWD)
An Observational Study of Vonicog Alfa (rVWF) in Pediatric Participants With Von Willebrand Disease (vWD)
This study is conducted in Japan of vonicog alfa (rVWF) used to treat pediatric participants with Von Willebrand Disease (vWD). The main aim of the study is to evaluate adverse drug reaction and effectiveness of vonicog alfa…
- Trial name
- NCT07404644
- Testing
- vonicog alfa (rVWF)
- Where
- Japan
- Volunteers
- 13 people
- Sponsor
- Takeda
Fetal and Neonatal Alloimmune Thrombocytopenia
Study to Assess the Occurrence of HPA-1a Alloimmunization in Women With Higher Risk for Fetal and Neonatal Alloimmune Thrombocytopenia
A prospective, non-interventional, natural history study to assess the occurrence of higher FNAIT risk across a broad population of different racial and ethnic characteristics and the occurrence of HPA-1a alloimmunization in…
- Trial name
- NCT05345561
- Testing
- Clinical data collection
- Where
- 28 sites in United States, Canada and 5 more
- Volunteers
- 14,390 people, healthy volunteers welcome
- Sponsor
- Rallybio
Von Willebrand Disease
Extension Study of Biostate in Subjects With Von Willebrand Disease
The aim of the Von Willebrand Disease (VWD) therapy is to treat and prevent bleeding episodes due to abnormal platelet adhesion and abnormal blood coagulation as a result of low or abnormal Von Willebrand Factor (VWF) and/or…
- Trial name
- NCT01224808
- Testing
- Biostate
- Where
- 6 sites in Bulgaria, Germany and 3 more
- Volunteers
- 20 people
- Sponsor
- CSL Behring
Von Willebrand Disease (VWD)
A Study on Estimated Risks of Certain Adverse Events in Adults With Von Willebrand Disease (VWD) Treated With Veyvondi
The main aim of this study is to estimate the risks of certain adverse events in adults with Von Willebrand Disease treated with VEYVONDI. No study medicines will be provided to participants in this study.
- Trial name
- NCT05265078
- Where
- Germany
- Volunteers
- 102 people
- Sponsor
- Takeda
Von Willebrand Disease (VWD), Heavy Menstrual Bleeding (HMB)
HMBeacon: A Phase 2 Study to Evaluate ALN-6400 in Adult and Adolescent Female Patients With VWD and HMB
The purpose of this study is to evaluate the safety, tolerability, efficacy, and pharmacodynamics (PD) of multiple doses of ALN-6400 in adult and adolescent patients with VWD and HMB
- Trial name
- HMBeacon (NCT07575308)
- Testing
- ALN-6400
- Where
- 21 sites in United States, Netherlands and 3 more
- Volunteers
- 24 people
- Sponsor
- Alnylam Pharmaceuticals
Von Willebrand Disease (VWD), Von Willebrand Disease (VWD), Type 1
A Study of Bleeding and Treatment in Participants With Von Willebrand Disease
The purpose of this screening study is to accumulate information regarding bleeding events, quality of life, and the social and clinical impact of bleeds in participants with Von Willebrand Disease (VWD).
- Conditions
- Von Willebrand Disease (VWD), Von Willebrand Disease (VWD), Type 1, Von Willebrand Disease (VWD), Type 2, Von Willebrand Disease (VWD), Type 3
- Trial name
- NCT06610201
- Testing
- Clinical outcomes of patients with VWD, Type 1, Clinical outcomes of patients with VWD, Type 2A, Type 2M, Type 2N, or Type 3
- Where
- 17 sites in United States, Australia and 1 more
- Volunteers
- 200 people
- Sponsor
- Hemab ApS
Thrombocytopenia, Neonatal Alloimmune
A Study of Nipocalimab or Intravenous Immunoglobulin (IVIG) in Pregnancies At Risk of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT)
The purpose of this study is to assess the efficacy and safety of nipocalimab in reducing the risk of severe fetal and neonatal alloimmune thrombocytopenia (FNAIT).
- Trial name
- FREESIA-3 (NCT06533098)
- Testing
- Nipocalimab, Intravenous immunoglobulins (IVIG), Prednisone
- Where
- 32 sites in United States, Austria and 7 more
- Volunteers
- 50 people
- Sponsor
- Janssen Research & Development, LLC
Thrombocytopenia, Neonatal Alloimmune
A Study of Nipocalimab in Reducing the Risk of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT)
The purpose of this study is to evaluate the effectiveness of nipocalimab compared with placebo in reducing the risk of severe fetal and neonatal alloimmune thrombocytopenia (FNAIT).
- Trial name
- FREESIA-1 (NCT06449651)
- Testing
- Nipocalimab
- Where
- 21 sites in Belgium, Brazil and 10 more
- Volunteers
- 39 people
- Sponsor
- Janssen Research & Development, LLC
Congenital Bleeding Disorder, Glanzmann's Disease
A Study to Evaluate Safety and Efficacy of NovoSeven® in Patients With Glanzmann's Thrombasthenia in Japan
This study is conducted in Asia. The aim of the study is to evaluate the safety and efficacy of NovoSeven® (activated recombinant factor VII, eptacog alfa (activated)) during bleeding episodes and for the prevention of bleeding…
- Trial name
- NCT01876745
- Testing
- eptacog alfa (activated)
- Where
- Japan
- Volunteers
- 4 people
- Sponsor
- Novo Nordisk A/S
Fetal and Neonatal Alloimmune Thrombocytopenia
Phase 2 Study on the Pharmacokinetics and Safety of RLYB212 in Pregnant Women at Higher Risk for HPA-1a Alloimmunization
The purpose of this Phase 2 study is to assess the pharmacokinetics (PK) and safety of RLYB212 in HPA-1b/b pregnant women at higher risk for HPA-1a alloimmunization and FNAIT.
- Trial name
- NCT06435845
- Testing
- Anti-(integrin beta-3) human monoclonal antibody
- Where
- 3 sites in Netherlands, Norway and 1 more
- Volunteers
- 1 people
- Sponsor
- Rallybio
Von Willebrand Disease
Efficacy of Alphanate FVIII/VWF Concentrate in Type 3 Von Willebrand Patients
To assess the efficacy of FVIII/VWF Complex (Human), Alphanate® as replacement therapy in preventing excessive bleeding in subjects with congenital Type 3 von Willebrand Disease (VWD) who undergo surgical procedures.
- Trial name
- NCT00555555
- Testing
- Alphanate SD/HT
- Volunteers
- 4 people
- Sponsor
- Grifols Biologicals, LLC