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
Systemic Lupus Erythematous
Pilot Study Related to the Effect of Clopidogrel on Plasmatic Soluble CD40 Ligand During Systemic Lupus Erythematous
CD40 Ligand (CD40L) has been identified as a key feature in systemic lupus erythematosus (SLE) pathogenesis, a systemic autoimmune disease characterized by a multiorgan involvement.
- Trial name
- CLOPUS (NCT02320357)
- Testing
- Treatment by clopidogrel
- Where
- 3 sites in France
- Volunteers
- 18 people
- Sponsor
- University Hospital, Bordeaux
Prader-Willi Syndrome, PWS-like Syndrome
GROWing Up With Rare GENEtic Syndromes
Introduction Rare complex syndromes Patients with complex genetic syndromes, by definition, have combined medical problems affecting multiple organ systems, and intellectual disability is often part of the syndrome.
- Conditions
- Prader-Willi Syndrome, PWS-like Syndrome, Silver Russel Syndrome, Congenital Hypopituitarism
- Trial name
- GROW UR GENES (NCT04463316)
- Testing
- Retrospective file studies
- Where
- Netherlands
- Volunteers
- 600 people
- Sponsor
- dr. Laura C. G. de Graaff-Herder
Von Willebrands Disease
Investigating a Von Willebrand Factor (VWF) Functional Screening Assay for Assigning the Phenotypic Variants of Von Willebrand Disease (VWD)
The purpose of this study is to improve the investigators ability to diagnose von Willebrand Disease (VWD), a common inherited bleeding disorder.
- Trial name
- VWF-phV (NCT02466789)
- Where
- United States
- Volunteers
- 134 people
- Sponsor
- Jonathan Roberts
Cancer
Liposomal Amphotericin B in Treating Granulocytopenia and Persistent Unexplained Fever in Cancer Patients
RATIONALE: Liposomal amphotericin B may be effective in controlling fever and granulocytopenia. It is not yet known which regimen of liposomal amphotericin B is more effective in treating cancer patients who have these…
- Trial name
- NCT00003938
- Testing
- liposomal amphotericin B
- Where
- 24 sites in Belgium, Czechia and 13 more
- Volunteers
- 115 people
- Sponsor
- European Organisation for Research and Treatment of Cancer - EORTC
Sickle Cell Disease
Study of Blood Platelets in Sickle Cell Disease
We are studying if sickle cell disease blood platelets are larger than normal and how they may cause obstruction of blood vessels.
- Trial name
- NCT00226083
- Where
- United States
- Volunteers
- healthy volunteers welcome
- Sponsor
- Rockefeller University
Von Willebrand Disease
Molecular and Clinical Profile of Von Willebrand Disease in Spain
The present Project is a third phase of the previous PCM-EVW-ES Project (Batlle et al. Thromb \& Haemost 2015) with the aim of its extension, further analysis with an innovation development in the field of von Willebrand disease…
- Trial name
- PCM-EVW-ES (NCT02869074)
- Testing
- VWF gene analysis
- Where
- Spain
- Volunteers
- 790 people, healthy volunteers welcome
- Sponsor
- Spanish Society of Thrombosis and Haemostasis
Antiphospholipid Syndrome (APS), Bullous Pemphigoid (BP)
Phase 1b Trial of RAY121 in Immunological Diseases (RAINBOW Trial)
This Phase 1b basket trial will investigate the safety, tolerability, pharmacokinetics, pharmacodynamics, immunogenicity and preliminary efficacy of RAY121, a inhibitor of classical complement pathway, after multiple dose…
- Conditions
- Antiphospholipid Syndrome (APS), Bullous Pemphigoid (BP), Behçet's Syndrome (BS), Dermatomyositis (DM)
- Trial name
- NCT06371417
- Testing
- RAY121
- Where
- 77 sites in United States, Australia and 18 more
- Volunteers
- 144 people
- Sponsor
- Chugai Pharmaceutical
Hypospadias
Comparison Of Outcome Of Different Layers in Hypospadias Surgery
To compare the outcome of applying autologous platelet-rich plasma membrane layer and dartos fascial flap versus only dartos fascial flap in mid and distal penile hypospadias surgery.
- Trial name
- NCT06275646
- Testing
- PRP layer, dartos flap
- Where
- Pakistan
- Volunteers
- 220 people
- Sponsor
- Children Hospital and Institute of Child Health, Lahore
Thrombocytopenia, Neonatal Thrombocytopenia
Neonatal Thrombocytopenia in the NICU
This prospective cohort study aims to determine the frequency, severity, and clinical outcome of thrombocytopenia among neonates admitted to the neonatal intensive care unit.
- Trial name
- NCT07882342
- Volunteers
- 120 people
- Sponsor
- Assiut University
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
Glanzmann Thrombasthenia
The Genetics and Functional Basis of Inherited Platelet, White Blood Cell, Red Blood Cell, and Blood Clotting Disorders.
Blood contains red blood cells, white blood cells, and platelets, as well as a fluid portion termed plasma. We primarily study blood platelets, but sometimes we also analyze the blood of patients with red blood cell disorders…
- Trial name
- NCT00230165
- Where
- United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Rockefeller University
Glanzmann Thrombasthenia
The Experiences of People Who Live With Glanzmanns Thrombasthenia.
To understand the lived experiences of people with Glanzmanns Thrombasthenia
- Trial name
- NCT05315232
- Testing
- survey and interview
- Where
- United Kingdom
- Volunteers
- 122 people
- Sponsor
- Haemnet
Chronic Myeloproliferative Disorders, Leukemia
Alemtuzumab and Combination Chemotherapy Followed By Donor Lymphocytes in Treating Patients Who Are Undergoing Donor Stem Cell Transplant for Hematologic Cancer
RATIONALE: Giving low doses of chemotherapy before a donor peripheral blood stem cell transplant helps stop the growth of cancer cells. It also stops the patient's immune system from rejecting the donor's stem cells.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Myelodysplastic Syndromes
- Trial name
- NCT00104975
- Testing
- alemtuzumab, therapeutic allogeneic lymphocytes, fludarabine phosphate
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Yale University
Chronic Myeloproliferative Disorders, Leukemia
Total-Body Irradiation, Fludarabine, and Alemtuzumab Followed By Stem Cell Transplant in Treating Patients With Myeloproliferative Disorder, MS, AML, or CML
Patients are being asked to participate in this study because they have a malignant blood disease such as Myelodysplastic Syndrome (MDS), Myeloproliferative Disorder (MPD), Acute Myelogenous Leukemia (AML) or Chronic Myelogenous…
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Myelodysplastic Syndromes
- Trial name
- MPDMDSBMT (NCT00069992)
- Testing
- Total Body Irradiation, Fludarabine, Campath 1H
- Where
- 2 sites in United States
- Volunteers
- 7 people
- Sponsor
- Baylor College of Medicine
Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders
Studying Fentanyl in Patients With Cancer
RATIONALE: Studying blood samples from cancer patients undergoing pain treatment in the laboratory may help doctors learn more about how pain drugs work in the body.
- Conditions
- Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders, Leukemia, Lymphoma
- Trial name
- NCT00899951
- Testing
- fentanyl citrate, pharmacological study
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
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
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