Clinical trials: Thrombopenia
Shwachman Syndrome, Fanconi's Anemia
Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure Syndromes
OBJECTIVES: I. Assess the efficacy of recombinant human granulocyte colony-stimulating factor (G-CSF) in raising the absolute neutrophil count, platelet count, and hemoglobin level in patients with inherited bone marrow failure…
- Conditions
- Shwachman Syndrome, Fanconi's Anemia, Dyskeratosis Congenita, Thrombocytopenia
- Trial name
- NCT00004787
- Testing
- filgrastim
- Volunteers
- 20 people
- Sponsor
- National Center for Research Resources (NCRR)
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
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)
Gastric/Duodenal Ulcer, Gastric MALT Lymphoma, Idiopathic Thrombocytopenic Purpura, or H. Pylori Gastritis, and Other
Drug Use Surveillance of Takecab for "Supplement to Helicobacter Pylori Eradication"
The purpose of this study is to evaluate the safety and efficacy on participants receiving first-line eradication and second-line eradication including vonoprazan (Takecab) tablets (triple therapy) in the routine clinical setting.
- Trial name
- NCT03219723
- Testing
- Vonoprazan, Amoxicillin hydrate, Clarithromycin
- Where
- Japan
- Volunteers
- 560 people
- Sponsor
- Takeda
Hospitalized Cases of Angioneurotic Oedema, Pneumonia
Drug Utilization and Safety Events Among Children Using Esomeprazole, Other Proton Pump Inhibitors or H2-receptor Antagonists
The purpose of this study is 1. To describe patient characteristics and drug usage among children that are prescribed esomeprazole for the first time and to compare them with patients who are prescribed other proton pump…
- Conditions
- Hospitalized Cases of Angioneurotic Oedema, Pneumonia, Gastroenteritis, Failure to Thrive
- Trial name
- NCT01338363
- Where
- Netherlands
- Volunteers
- 23,470 people
- Sponsor
- AstraZeneca
Kaposiform Hemangioendotheliomas, Tufted Angioma
Safety and Efficacy Study of Sirolimus in Complicated Vascular Anomalies
The purpose of this study is to determine if the use of sirolimus in the treatment of children and young adults with complicated vascular anomalies will prove to be safe and provide objective response resulting in improved…
- Conditions
- Kaposiform Hemangioendotheliomas, Tufted Angioma, Capillary Venous Lymphatic Malformation, Venous Lymphatic Malformation
- Trial name
- NCT00975819
- Testing
- sirolimus
- Where
- 2 sites in United States
- Volunteers
- 61 people
- Sponsor
- Denise Martin Adams
Platelet Function, Thrombocytopenia
Platelet Function During ECMO (Extra Corporeal Membrane Oxygenation)
Platelet transfusions are routinely administered during neonatal ECMO, with an average of 1.3 platelet transfusions per day being administered while a patient is undergoing ECMO treatment.
- Trial name
- NCT00748878
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- University of Utah
Thrombocytopenia
Assessment of Risk Factors and Outcome of Thrombocytopenia in ICU Patients
The many comorbidities in the severely ill patient also make thrombocytopenia very common (∼ 40%) in intensive care unit patients.
- Trial name
- NCT05958511
- Volunteers
- 130 people
- Sponsor
- Assiut University
Congenital Amegakaryocytic Thrombocytopenia, Diamond-blackfan Anemia
Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders
RATIONALE: Monoclonal antibodies, such as alemtuzumab, can block cancer growth in different ways. Some block the ability of cancer cells to grow and spread.
- Conditions
- Congenital Amegakaryocytic Thrombocytopenia, Diamond-blackfan Anemia, Leukemia, Myelodysplastic Syndromes
- Trial name
- NCT00301834
- Testing
- alemtuzumab, busulfan, cyclosporine
- Where
- 2 sites in United States
- Volunteers
- 35 people
- Sponsor
- University of California, San Francisco
Pancytopenia Due to Chemotherapy
Effect of rhIL-11 in Patients With Thrombocytopenia for Childhood ALL
The goal of this clinical research study is to find out if rhIL-11（Interleukin 11） may increase the platelet count in Childhood patients with acute lymphocytic leukemia (ALL) who develop low platelet counts while receiving…
- Trial name
- NCT02314273
- Testing
- rhIL-11
- Where
- 5 sites in China
- Volunteers
- 120 people
- Sponsor
- Shanghai Children's Medical Center
Kaposiform Hemangioendothelioma (KHE), Kasabach-Merritt Syndrome
A Study to Compare Vincristine to Sirolimus for Treatment of High Risk Vascular Tumors
In this research study we want to learn more about which treatment works better for patients diagnosed with a vascular tumor called Kaposiform Hemangioendothelioma (KHE) or other high risk vascular tumors such as Tufted Angioma…
- Conditions
- Kaposiform Hemangioendothelioma (KHE), Kasabach-Merritt Syndrome, Tufted Angioma
- Trial name
- NCT02110069
- Testing
- Vincristine, Sirolimus
- Where
- 7 sites in United States
- Volunteers
- 4 people
- Sponsor
- Boston Children's Hospital
Immune Thrombocytopenia, Treatment
Anti-CD38 Antibody Treating Pediatric Primary Immune Thrombocytopenia (ITP)
To evaluate the safety and efficacy of Anti-CD38 Antibody in the treatment of pediatric primary immune thrombocytopenia in patients who have not responded adequately or relapsed after first-line treatment and at least one…
- Trial name
- NCT06168851
- Testing
- Anti-CD38 antibody Injection
- Where
- China
- Volunteers
- 60 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Immune Thrombocytopenia
A Retrospective Chart Review of Thirty Three Children Who Have Received Clinical Treatment With Either Romiplostim or Eltrombopag From 2009-2013
This is a retrospective analysis of children (≤21years old) who received clinical treatment with either Romiplostim or Eltrombopag at 2 medical centers from 2009-2013.
- Trial name
- NCT01974232
- Where
- United States
- Volunteers
- 33 people
- Sponsor
- Weill Medical College of Cornell University
Idiopathic Thrombocytopenic Purpura
Study of a New Medication for Childhood Chronic Immune Thrombocytopenia (ITP), a Blood Disorder of Low Platelet Counts That Can Lead to Bruising Easily, Bleeding Gums, and/or Bleeding Inside the Body.
The purpose of this study is to investigate the efficacy, safety and tolerability of eltrombopag in children with previously treated chronic immune thrombocytopenia who are between 1 and 17 years of age. This is a 2 part study.
- Trial name
- PETIT2 (NCT01520909)
- Testing
- Eltrombopag
- Where
- 51 sites in United States, Argentina and 11 more
- Volunteers
- 92 people
- Sponsor
- GlaxoSmithKline
Thrombocytopenia
Efficacy and Safety Study of Platelets Treated for Pathogen Inactivation and Stored for Up to Seven Days
Objective: To determine if platelets treated for pathogen inactivation and stored for 6 to 7 days are safe and effective compared to platelets collected by the same method, stored for the same amount of time and not treated for…
- Trial name
- TESSI (NCT00261924)
- Testing
- Transfusion of Pathogen Inactivated Platelets stored for 6-7 days
- Where
- United Kingdom
- Volunteers
- 211 people
- Sponsor
- Cerus Corporation
Immune Thrombocytopenia With Positive ANA Antibodies
The Efficacy and Safety of HCQ Plus TPO-RA in ANA Positive ITP
The goal of this clinical trial is to learn if hydroxychloroquine (HCQ) plus thrombopoietin receptor agonists (TPO-RA) works to treat primary immune thrombocytopenia with positive anti-nuclear antibodies in adults.
- Trial name
- NCT06479291
- Testing
- Hydroxychloroquine Oral Tablet, Eltrombopag
- Where
- 7 sites in China, Hong Kong and 1 more
- Volunteers
- 126 people
- Sponsor
- Yunfeng Cheng
Immune Thrombocytopenic Purpura ( ITP ), Systemic Lupus Erythematosus (SLE)
Modeling the Early Stages of Autoimmunity Through the Study of Immunological Thrombocytopenic Purpura and Juvenile Lupus
Immunologic thrombocytopenic purpura (ITP) in children is a pre-lupus condition if associated with the presence of anti-nuclear antibodies (ANA), providing a unique model for understanding the natural history of autoimmunity…
- Trial name
- PRELUDE (NCT07680010)
- Testing
- Blood sampling baseline and M3, Monitoring for SLE, Procedure/Surgery: Blood sampling baseline and M3 then each 6 months until 48 months
- Where
- 2 sites in France
- Volunteers
- 105 people, healthy volunteers welcome
- Sponsor
- University Hospital, Bordeaux
Essential Thrombocytopenia
Pegylated Interferon Alfa-2b Versus Interferon Alfa Therapy in Childhood and Adolescent Essential Thrombocythemia
Objectives: To compare the efficacy and safety in childhood and adolescent patients (<20 years) diagnosed as essential thrombocythemia treated with the Pegylated Interferon Alfa-2b vs. Interferon Alfa.
- Trial name
- NCT04226950
- Testing
- Recombinant Interferon Alpha, Pegylated interferon alfa-2b
- Where
- China
- Volunteers
- 40 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Inherited Bone Marrow Failure Syndrome, Fanconi Anemia
Enhancing the Clinical Management of CMF Through a National Pediatric and Adult Network Based on Biological and Genetic Data
A non-profit, translational, prospective and retrospective study. Patients will be enrolled according to the procedures described in the protocol, and samples will be processed and analyzed as specified in the protocol.
- Conditions
- Inherited Bone Marrow Failure Syndrome, Fanconi Anemia, Dyskeratosis Congenita, Telomere Biology Disorder
- Trial name
- 2023-12377081 (NCT07877727)
- Where
- 4 sites in Italy
- Volunteers
- 250 people
- Sponsor
- Istituto Giannina Gaslini
Primary Immune Thrombocytopenia (ITP)
rhTPO Dose Escalation vs Eltrombopag Switch in ITP
This study is a prospective, multicenter, randomized controlled study, planning to enroll 110 ITP patients who failed to respond to conventional-dose rhTPO (300 IU/kg/d) after 14 days of treatment (PLT < 30×10⁹/L).
- Trial name
- NCT07476846
- Testing
- Recombinant Human Thrombopoietin（rhTPO）, Eltrombopag PfOS
- Volunteers
- 112 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China