Clinical trials: Transfusion Dependent Cytopenias
Bone Marrow Failure Syndromes, Aplastic Anemias
A Phase 1/2 Study to Evaluate the Safety and Efficacy of Dibotatug (DR-01) in Adults With Bone Marrow Failure Syndromes
This is a multicenter, open-label, Phase 1/2 basket study to evaluate the safety and efficacy of Dibotatug (DR-01) in adults with Bone Marrow Failure syndromes.
- Conditions
- Bone Marrow Failure Syndromes, Aplastic Anemias, Severe Aplastic Anemia (SAA), Severe Aplastic Anemia, Relapse
- Trial name
- BMF (NCT07712562)
- Testing
- Dibotatug (DR-01)
- Where
- 10 sites in United States
- Volunteers
- 60 people
- Sponsor
- Dren Bio
Myelodysplastic Syndrome (MDS)
LBH589 Alone or in Combination With Erythropoietin Stimulating Agents (ESA) in Patients With Low or Int-1 Risk Myelodysplastic Syndromes (MDS)
This study assessed the efficacy and safety of LBH589 as single agent and in combination with ESA in red blood cell transfusion-dependent Low and Int-1 MDS patients being either refractory to ESA or with a low probability of…
- Trial name
- GEPARD (NCT01034657)
- Testing
- LBH589, Epoetin Alfa
- Where
- 12 sites in Germany
- Volunteers
- 34 people
- Sponsor
- Novartis Pharmaceuticals
Hematologic Malignancy, Myelodysplastic Syndromes
Home Blood Transfusions
This is a pilot single arm study to evaluate the feasibility and acceptability of a home blood transfusion program. Patients will be enrolled with hematologic malignancies and other bone marrow failure syndromes who are…
- Conditions
- Hematologic Malignancy, Myelodysplastic Syndromes, Acute Myeloid Leukemia, Transfusion Dependent Cytopenias
- Trial name
- NCT07121140
- Testing
- Home Blood Transfusion
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Thomas Jefferson University
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