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Gene therapy offers new hope for 'bubble boy' disease patients over age 2

NCT ID NCT01306019

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

First seen Oct 01, 2025 · Last updated May 15, 2026 · Updated 27 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 the body unable to fight infections. Participants receive a mild chemotherapy drug to prepare their bone marrow, followed by an infusion of their own blood stem cells that have been corrected with a safer lentiviral vector. The goal is to restore immune function and reduce infections, especially in those who did not fully benefit from a prior bone marrow transplant.

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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.

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Contacts and locations

Study contacts

  • Contact

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

Locations

  • National Institutes of Health Clinical Center

    RECRUITING

    Bethesda, Maryland, 20892, United States

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

Conditions

Explore the condition pages connected to this study.

X-LINKED SEVERE COMBINED IMMUNODEFICIENCY (XSCID) X-LINKED SEVERE COMBINED IMMUNODEFICIENCY (XSCID)