Clinical trials: Primary Immune Thrombocytopenia, open to volunteers
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
Primary Immune Thrombocytopenia (ITP)
A Study of Ianalumab in Addition to Eltrombopag in Pediatric Patients With Primary ITP Who Failed Corticosteroids.
The purpose of this study is to assess the efficacy, safety and pharmacokinetics (PK) of ianalumab (VAY736) in addition to eltrombopag treatment; and to inform the dose of ianalumab in pediatric patients (5 to <18 years of age)…
- Trial name
- VAYHIT-P (NCT07660172)
- Testing
- Ianalumab, Eltrombopag
- Volunteers
- 36 people
- Sponsor
- Novartis Pharmaceuticals
Primary Immune Thrombocytopenia
Longitudinal Cohort of Pediatric Primary Immune Thrombocytopenia (ITP)
Immune thrombocytopenic purpura (ITP) is a kind of rare childhood disease that involve autoimmune destruction of platelets.The current Pediatric ITP cohorts are mostly based on single-center or multi-center cases, or cohorts with…
- Trial name
- NCT06107582
- Testing
- data collection
- Where
- China
- Volunteers
- 500 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes
Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
This is a data collection study that will examine the general diagnostic and treatment data associated with the reduced-intensity chemotherapy-based regimen paired with simple alemtuzumab dosing strata designed to prevented graft…
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias
- Trial name
- PRO-RIC (NCT04528355)
- Testing
- data collection
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Paul Szabolcs
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes
Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
The objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), double cord UCBT, matched unrelated donor (MUD) bone marrow transplant…
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias
- Trial name
- HSCT+RIC (NCT01962415)
- Testing
- Hydroxyurea, Alemtuzumab, Fludarabine
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Paul Szabolcs
Primary Immune Thrombocytopenia (ITP)
Sequential rhTPO and Eltrombopag Following Glucocorticoids for Severe Adult ITP
To address the clinical need for improving early response rates, maintaining sustained responses, reducing relapse rates, and minimizing adverse events in the treatment of immune thrombocytopenia (ITP), the investigators…
- Trial name
- NCT07189910
- Testing
- Eltrombopag
- Where
- China
- Volunteers
- 150 people
- Sponsor
- Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
ITP - Immune Thrombocytopenia, Primary Immune Thrombocytopenia (ITP)
RESTI Registry: Spanish Registry of Primary Immune Thrombocytopenia and Other Immune Thrombocytopenia
Primary immune thrombocytopenia (PIT), previously referred to as idiopathic thrombocytopenic purpura, is an acquired disease characterized by accelerated platelet destruction and deficient platelet production by megakaryocytes.
- Trial name
- RESTI (NCT06919341)
- Where
- 36 sites in Spain
- Volunteers
- 1,000 people
- Sponsor
- Fundación Española de Hematología y Hemoterapía