Clinical trialNCT04776850WithdrawnEarly phase 1Interventional

Pre-transplant Immunosuppression and Donor Stem Cell Transplant for the Treatment of Severe Hemoglobinopathies

SponsorM.D. Anderson Cancer CenterEnrollment0Beta Thalassemia MajorSickle Beta 0 ThalassemiaSickle Beta Plus ThalassemiaSickle Beta ThalassemiaSickle Cell DiseaseSickle Cell-SS Disease

View the registry record on ClinicalTrials.gov

Summary

The lay summary the sponsor registered on ClinicalTrials.gov.

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.

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Investigators

The overall officials registered on this study. Names that resolve to a findmyKOL profile link to it; the rest are shown as registered.

Kris M Mahadeo
M.D. Anderson Cancer Center
Principal Investigator
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A profile link is shown only when the registered name resolves to a person record with a confident, namesake-checked match; ambiguous names stay unlinked.

Sites

No facility list is in our current data snapshot for this study. The registry record on ClinicalTrials.gov ↗ carries any published locations. Why? →

Drugs studied

The drug and biological interventions registered on this study, normalized to their RxNorm ingredient where possible.

Bortezomib →Busulfan →Cyclophosphamide →Dexamethasone →Fludarabine →Lymphocyte immune globulin, anti-thymocyte globulin →Mycophenolate mofetil →Rituximab →Tacrolimus →

Linked drugs open the directory filtered to KOLs working with them.clinicaltrials.gov ↗

Registration & key dates

Registry attributes as recorded on the ClinicalTrials.gov study record.

Start: 29 Dec 2020
Primary completion: 5 Dec 2022
Completion: 5 Dec 2022
Last update posted: 24 Oct 2024
Registered enrollment: 0
Registry id: NCT04776850

clinicaltrials.gov ↗

Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 24 Aug 2026. Registry facts are shown as registered by the study sponsor; findmyKOL does not interpret them.