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Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors

NCT07599176 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 90

Last updated 2026-05-20

No results posted yet for this study

Summary

This is a non-ablative (partial) stem cell transplant for patients with severe sickle cell disease or beta-thalassemia requiring red cell transfusions. The intensity of the transplant is slightly increased from our previous transplant regimens. The goal is to aim for higher percentage of donor cells to stably remain in the recipients long term.

Conditions

Interventions

RADIATION

Total Body Irradiation

Total Body Irradiation 400 cGy

DRUG

Alemtuzumab

1 mg/kg

DRUG

Abatacept

10 mg/kg x 6

OTHER

Research blood draw

About 5 tablespoons of blood will be collected from donors for research purposes.

Sponsors & Collaborators

  • National Heart, Lung, and Blood Institute (NHLBI)

    lead NIH

Principal Investigators

  • John F Tisdale, M.D. · National Heart, Lung, and Blood Institute (NHLBI)

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
4 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-06-01
Primary Completion
2032-06-30
Completion
2035-06-30

Countries

  • United States

Study Locations

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Entities

Drugs
Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07599176 on ClinicalTrials.gov