New gene therapy offers hope for older kids and adults with 'bubble boy' disease

NCT ID NCT01306019

Recruiting now Disease control Sponsor: National Institute of Allergy and Infectious Diseases (NIAID) Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 30, 2026 · Updated 2 times

Summary

This study tests a gene therapy for people aged 2 to 50 with X-linked severe combined immunodeficiency (SCID-X1), a condition that leaves them without a working immune system. Participants receive their own blood stem cells that are genetically corrected in a lab, then infused back after mild chemotherapy. The goal is to restore immune function and reduce infections, especially for those who cannot get a perfect bone marrow match.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for X-LINKED SEVERE COMBINED IMMUNODEFICIENCY (XSCID) 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.

X-LINKED SEVERE COMBINED IMMUNODEFICIENCY (XSCID)

Contacts and locations

Show contact details

Study contacts

Contact

Phone: •••-•••-•••• Email: •••••@•••••

Locations

National Institutes of Health Clinical Center
RECRUITING

Bethesda, Maryland, 20892, United States

Contact Phone: •••-•••-•••• Email: •••••@•••••