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

NCT04370795 · Status: WITHDRAWN · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL

Last updated 2025-12-01

No results posted yet for this study

Summary

Background:

Severe combined immune deficiency (SCID) is a group of conditions where the immune system does not work properly. The only cure for most SCIDs is a stem cell transplant (getting cells from a donor). These transplants can have serious complications. Before the transplant, people often get high doses of drugs and radiation to prepare the body to accept the cells from the donor. Researchers want to see if low doses of drugs alone without radiation work just as well as low doses of drugs with radiation for SCID patients getting stem cell transplants.

Objective:

To test a set of drugs with or without radiation given before a stem cell transplant.

Eligibility:

People ages 3-40 who have SCID and who have a stem cell donor - either related or unrelated.

Design:

Participants will be admitted to the hospital 10 days before transplant. They will undergo:

medical history

medication review

physical exam

blood and urine tests (may include a 24-hour urine collection)

heart, lung, and breathing tests

imaging scans

bone marrow sample

nutrition assessment

dental exam

eye exam

meeting with a social worker.

Participants will get a plastic port called a central line. It is a hollow tube that is placed in the upper chest. It will be used to give medicines and take blood.

All participants will take chemotherapy drugs. Some will get radiation.

Participants will have a stem cell transplant. They will get the cells as an infusion through their central line. They will stay in the hospital for 30 days after transplant.

Participants must stay within 1 hour of NIH for 3 months after transplant. During this time, they will have follow-up visits at NIH at least once a week. Then they will have follow-up visits once or twice a year for 5-6 years.

Conditions

  • Severe Combined Immune Deficiency (SCID)

Interventions

RADIATION

Total Body Irradiation (TBI)

Conditioning (only patients in Group 1 will receive TBI)

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

Sponsors & Collaborators

  • National Institute of Allergy and Infectious Diseases (NIAID)

    lead NIH

Principal Investigators

  • Elizabeth M Kang, M.D. · National Institute of Allergy and Infectious Diseases (NIAID)

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
3 Years
Max Age
40 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-11-26
Primary Completion
2025-11-26
Completion
2025-11-26
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

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 NCT04370795 on ClinicalTrials.gov