Gene therapy offers hope for children with SCID-X1

NCT ID NCT03311503

Recruiting now Disease control Sponsor: David Williams Source: ClinicalTrials.gov ↗

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

Summary

This trial tests a gene therapy for children with X-linked severe combined immunodeficiency (SCID-X1), also known as 'bubble boy' disease. Participants receive a low dose of busulfan followed by an infusion of their own blood stem cells that have been genetically corrected with a lentiviral vector. The goal is to restore a working immune system and reduce the need for lifelong treatments. The study is recruiting up to 12 children aged 5 or younger.

What this could mean

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

Active substance: autologous CD34+ cells transduced with G2SCID lentiviral vector
What this could lead to: If successful, this could provide a one-time gene therapy that restores immune function in children with SCID-X1, reducing the need for lifelong treatments.
What could go wrong: This is an early-phase trial with only 12 participants, so results may not apply to all patients. Risks include failure to restore immunity, side effects from busulfan, or need for a stem cell transplant.

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

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for GENE THERAPY are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

T-B+ severe combined immunodeficiency due to gamma chain deficiency X-Linked Combined Immunodeficiency Diseases

As listed by the trial registrant

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

GENE THERAPY SEVERE COMBINED IMMUNODEFICIENCY, X LINKED

Contacts and locations

Locations

Boston Childrens Hospital
RECRUITING

Boston, Massachusetts, 02115, United States
Cincinnati Children's Hospital Medical Center
RECRUITING

Cincinnati, Ohio, 45229, United States
Emory University/Childrens Healthcare of Atlanta
RECRUITING

Atlanta, Georgia, 30322, United States
Mattel Children's Hospital - UCLA
RECRUITING

Los Angeles, California, 90095, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.