Clinical trials: Inherited Abnormalities of Platelets
Congenital Heart Defects, Blood Platelet Disorders
Platelet Aggregation Inhibition in Children on Clopidogrel (PICOLO)
PICOLO is a double blind placebo controlled phase II dose ranging, dose escalating study in patients of Blalock-Taussig age categories (neonates and infants/toddlers), to determine the dose providing inhibition of platelet…
- Trial name
- NCT00115375
- Testing
- Clopidogrel (SR25990)
- Where
- 6 sites in United States, Belgium and 4 more
- Volunteers
- 92 people
- Sponsor
- Sanofi
Von Willebrand Diseases
Clinical Study to Investigate the Efficacy and Safety of Wilate During Prophylaxis in Previously Treated Patients With VWD
This is a prospective, non-controlled, international, multi-center phase 3 study investigating the efficacy and safety of Wilate in previously treated adult patients with VWD, to obtain additional data on the safety and efficacy…
- Trial name
- NCT04052698
- Testing
- Wilate
- Where
- 14 sites in United States, Belarus and 6 more
- Volunteers
- 43 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
A Canadian Study to Assess the Safety of Humate-P® Ivr (Infusion Volume Reduced)
As part of CSL Behring Canada's continued commitment to ensuring the safety of the new low volume preparation of Humate-P®, CSL Behring Canada proposes to conduct a prospective, multi-center structured data collection of routine…
- Trial name
- NCT00701545
- Where
- Canada
- Volunteers
- 21 people
- Sponsor
- CSL Behring
Autoimmune Lymphoproliferative Syndrome, Autoimmune Cytopenia
Conception of a Diagnosis, Prognosis and Therapeutic Decision Tool for Patients With Autoimmunity and Inflammation
The main objective of this study is to generate diagnosis and therapeutic-decision tools through the identification of molecular causes of PIDs with autoimmunity/inflammation and the variability in disease outcome at the…
- Conditions
- Autoimmune Lymphoproliferative Syndrome, Autoimmune Cytopenia, Autoimmune Diseases, Autoimmune Anemia
- Trial name
- ATRACTion (NCT04902807)
- Testing
- Collection of samples
- Where
- France
- Volunteers
- 325 people
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Von Willebrand Disease
A Study With OPTIVATE® in People With Von Willebrand Disease
The main objective of the study is to assess the pharmacokinetics of OPTIVATE® after a single dose of 80 IU/kg VWF:RCo. The secondary objectives of the study are to assess efficacy and safety of OPTIVATE® in long-term use over at…
- Trial name
- NCT00387192
- Testing
- Optivate
- Where
- 4 sites in Israel, United Kingdom
- Volunteers
- 26 people
- Sponsor
- Bio Products Laboratory
Anemia, Chronic Myeloproliferative Disorders
Busulfan and Fludarabine Before Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer
RATIONALE: Drugs used in chemotherapy, such as busulfan and fludarabine, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing.
- Conditions
- Anemia, Chronic Myeloproliferative Disorders, Leukemia, Lymphoma
- Trial name
- NCT00301912
- Testing
- filgrastim, busulfan, fludarabine phosphate
- Where
- United States
- Sponsor
- University of California, San Francisco
Chronic Myeloproliferative Disorders, Leukemia
Combination Chemotherapy Followed By Umbilical Cord Blood Transplantation in Treating Patients With Hematologic Cancer or Severe Aplastic Anemia
RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Umbilical cord blood transplantation may be able to replace cells destroyed by chemotherapy.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00054236
- Testing
- anti-thymocyte globulin, filgrastim, cyclophosphamide
- Where
- United States
- Volunteers
- 55 people
- Sponsor
- Case Comprehensive Cancer Center
Thrombocytopenia, Metachromatic Leukodystrophy
Stem Cell Transplantation (SCT) for Genetic Diseases
OBJECTIVES: I. Ascertain whether stem cell transplantation (SCT) is an effective method by which missing or dysfunctional enzymes can be replaced in patients with various inborn errors of metabolism. II.
- Conditions
- Thrombocytopenia, Metachromatic Leukodystrophy, Fanconi's Anemia, Thalassemia Major
- Trial name
- NCT00004378
- Testing
- Stem Cell Transplantation
- Where
- United States
- Sponsor
- National Center for Research Resources (NCRR)
Pulmonary Hypertension
Anticoagulation For Pulmonary Hypertension in Sickle Cell Disease
Sickle cell disease (SCD) is often referred to as a hypercoagulable state. However, the contribution of coagulation activation to the pathogenesis of SCD remains uncertain.
- Trial name
- NCT01036802
- Testing
- Warfarin
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- University of North Carolina, Chapel Hill
Chronic Myeloproliferative Disorders, Leukemia
Donor Bone Marrow Transplant in Treating Patients With Leukemia, Lymphoma, or Nonmalignant Hematologic Disorders
RATIONALE: Giving chemotherapy and total-body irradiation before a donor bone marrow transplant helps stop the growth of cancer cells. It also helps stop the patient's immune system from rejecting the donor's stem cells.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Multiple Myeloma and Malignant Plasma Cell Neoplasms, Myelodysplastic Syndromes
- Trial name
- NCT00005622
- Testing
- cyclophosphamide, TBI
- Where
- United States
- Volunteers
- 72 people
- Sponsor
- H. Lee Moffitt Cancer Center and Research Institute
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
Hermansky Pudlak Syndrome
A Longitudinal Study of Hermansky-Pudlak Syndrome Pulmonary Fibrosis
Hermansky-Pudlak Syndrome (HPS) is a rare genetic disease that is associated with oculocutaneous albinism, bleeding, granulomatous colitis, and pulmonary fibrosis in some subtypes, including HPS-1, HPS-2, and HPS-4.
- Trial name
- NCT02368340
- Testing
- Pulmonary function test, Chest CT, Sample collection
- Where
- 5 sites in United States
- Volunteers
- 55 people
- Sponsor
- Vanderbilt University
Von Willebrand Diseases
The Lived Experience of People With Von Willebrand Disease
von Willebrand disease (vWD) is reported to be the most common bleeding disorder, with prevalence estimated at 1% of the general population.
- Trial name
- VWD360 (NCT06064643)
- Testing
- Online Survey, Qualitative Interview, Bleed Diary
- Where
- United Kingdom
- Volunteers
- 611 people
- Sponsor
- Haemnet