New transplant approach could help more patients with blood disorders

NCT ID NCT05736419

Recruiting now Disease control Sponsor: Memorial Sloan Kettering Cancer Center Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study is testing a stem cell transplant from half-matched (haploidentical) donors for people with sickle cell disease or beta-thalassemia. Before the transplant, patients receive drugs to lower their immune system, which may reduce the risk of serious side effects like graft failure or graft-versus-host disease. The trial aims to enroll 24 adults and children aged 2 to 50. If it works, this could provide a treatment option for those without a fully matched donor.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: Fludarabine, cyclophosphamide, tacrolimus, mycophenolate mofetil, rabbit ATG, dexamethasone
What this could lead to: If successful, this approach could offer a safer stem cell transplant option for people with sickle cell disease or thalassemia who lack a fully matched donor, potentially controlling the disease long-term.
What could go wrong: This is a small, early-phase trial with only 24 participants. The treatment involves strong immune-suppressing drugs, which carry risks of infection, graft failure, or graft-versus-host disease. Results may not apply to all patients.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

beta thalassemia beta-thalassemia HBB/LCRB sickle cell disease thalassemia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

SICKLE CELL DISEASE THALASSEMIA THALASSEMIA, BETA

Contacts and locations

Locations

Memorial Sloan Kettering Bergen (Consent only)
RECRUITING

Montvale, New Jersey, 07645, United States
Memorial Sloan Kettering Monmouth (Consent only)
RECRUITING

Middletown, New Jersey, 07748, United States
Memorial Sloan Kettering Nassau (All protocol activities)
RECRUITING

Rockville Centre, New York, 11553, United States
Memorial Sloan Kettering Suffolk - Commack (Consent only)
RECRUITING

Commack, New York, 11725, United States
Memorial Sloan Kettering Westchester (Consent only)
RECRUITING

Harrison, New York, 10604, United States
Memorial Sloan Kettering at Basking Ridge (Consent only)
RECRUITING

Basking Ridge, New Jersey, 07920, United States

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