Pre-transplant Immunosuppression and Donor Stem Cell Transplant for the Treatment of Severe Hemoglobinopathies
NCT04776850 · Status: WITHDRAWN · Phase: EARLY_PHASE1 · Type: INTERVENTIONAL
Last updated 2024-10-24
Summary
This clinical trial studies the effect of pre-transplant immunosuppression (PTIS) and donor stem cell transplant in treating patients with severe blood diseases (hemoglobinopathies). PTIS helps prepare the body for the transplant and lowers the risk of developing graft versus host disease (GVHD). Hematopoietic cells are found in the bone marrow and produce blood cells. Hematopoietic cell transplantation (HCT) injects healthy hematopoietic cells into the body to support blood cell production. PTIS and HCT may help to control severe hemoglobinopathies.
Conditions
- Beta Thalassemia Major
- Sickle Beta 0 Thalassemia
- Sickle Beta Plus Thalassemia
- Sickle Beta Thalassemia
- Sickle Cell Disease
- Sickle Cell-SS Disease
Interventions
- DRUG
-
Given IV
- DRUG
-
Busulfan
Given IV
- DRUG
-
Given IV
- DRUG
-
Given IV
- DRUG
-
Fludarabine Phosphate
Given IV
- PROCEDURE
-
Hematopoietic Cell Transplantation
Undergo HCT
- BIOLOGICAL
-
Lapine T-Lymphocyte Immune Globulin
Given IV
- DRUG
-
Mycophenolate Mofetil
Given IV or PO
- PROCEDURE
-
Plasmapheresis
Undergo plasmapheresis
- BIOLOGICAL
-
Given IV
- DRUG
-
Given IV or PO
Sponsors & Collaborators
-
M.D. Anderson Cancer Center
lead OTHER
Principal Investigators
-
Kris M Mahadeo · M.D. Anderson Cancer Center
Study Design
- Allocation
- NA
- Purpose
- TREATMENT
- Masking
- NONE
- Model
- SINGLE_GROUP
Eligibility
- Min Age
- 2 Years
- Max Age
- 30 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2020-12-29
- Primary Completion
- 2022-12-05
- Completion
- 2022-12-05
- FDA Drug
- Yes
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