NCT04370795View on ClinicalTrials.gov

Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and Sirolimus

PHASE1/PHASE2Enrolling by invitationINTERVENTIONAL
Enrollment

30

Participants

Timeline

Start Date

October 28, 2025

Primary Completion Date

September 9, 2026

Study Completion Date

September 9, 2026

Conditions
Severe Combined Immune Deficiency (SCID)
Interventions
DRUG

Sirolimus

Post transplant immunosuppressant drug

DRUG

Busulfan

Conditioning drug

DRUG

Horse -Anti-thymocyte

Immune suppression conditioning drug

DRUG

G-CSF

Used to prevent infection and neutropenic fevers caused by chemotherapy

RADIATION

Total Body Irradiation (TBI)

Conditioning (only patients in Group 1 will receive TBI)

Trial Locations (1)

20892

National Institutes of Health Clinical Center, Bethesda

All Listed Sponsors
lead

National Institute of Allergy and Infectious Diseases (NIAID)

NIH

NCT04370795 - Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and Sirolimus | Biotech Hunter | Biotech Hunter