Gene therapy could end pain crises for sickle cell patients

NCT ID NCT05353647

Ongoing Disease control Sponsor: David Williams Source: ClinicalTrials.gov ↗

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

Summary

This study tests a gene therapy that modifies a patient's own blood stem cells to produce more fetal hemoglobin, a healthy type that prevents sickling. The goal is to reduce or eliminate severe pain crises in people with sickle cell disease. The treatment involves chemotherapy to prepare the body, then infusion of the modified cells. It is for 25 patients aged 13-40 with severe disease who have had at least 4 pain crises in the last 2 years.

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Conditions

The condition(s) this trial relates to.

beta thalassemia sickle cell disease

As listed by the trial registrant

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

SICKLE CELL DISEASE

Contacts and locations

Locations

Boston Children's Hospital

Boston, Massachusetts, 02115, United States
Children's Healthcare of Atlanta/Emory University

Atlanta, Georgia, 30322, United States
Children's Hospital of Los Angeles

Los Angeles, California, 90027, United States
Dana-Farber Cancer Institute/Brigham and Women's Hospital

Boston, Massachusetts, 02115, United States
Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States
Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States
UC Davis Medical Center

Sacramento, California, 95817, United States
UCLA Medical Center

Los Angeles, California, 90095, United States
UCSF Benioff Children's Hospital Oakland

Oakland, California, 94609, United States

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