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Gene Therapy for SCID-X1 with Low Dose Busulfan

This study is testing a gene therapy called G2SCID for boys aged 0-5 years old with Severe Combined Immunodeficiency, X-Linked (SCID-X1). SCID-X1 is a serious immune system disorder. Participants will first receive a low dose of busulfan (a chemotherapy drug) and then a single infusion of their own blood cells, which have been modified with the G2SCID gene therapy. The main goals are to see how many patients are free from serious events and how well their T-cells (a type of immune cell) recover one year after the treatment. This is a Phase I/II study, meaning it's looking at both safety and how well the treatment works. The study plans to enroll 12 participants, but the current enrollment status is unclear.

Study design
This is an open-label, multi-center Phase I/II study involving a single infusion of the gene therapy. It plans to enroll 12 male participants aged 0-5 years.
What's involved
Participants will receive a low dose of busulfan and then a single infusion of their modified cells. They will be followed for 2 years after the infusion on this protocol.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for 2 years after infusion on this protocol, with long-term monitoring for a total of 15 years on a separate protocol.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT03311503

Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning

Recruiting
PHASE1Ages 0–5InterventionalTreatment
David Williams
~12 participants
Updated 2025-12-11 on ClinicalTrials.gov
What's tested:autologous CD34+ cell transduced with G2SCID vector

At a glance

Recruiting sites
4 of 4 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
The primary objective is to measure event free survival
Measured over 1 year post infusion
+1 more outcome measured

Conditions

Severe Combined Immunodeficiency, X Linked
Gene Therapy

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NCT03311503

Where you'd take part

This study runs at 4 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Boston Childrens Hospital

    Boston, Massachusettsstudy coordinator listed

    Recruiting

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohiostudy coordinator listed

    Recruiting

  • Emory University/Childrens Healthcare of Atlanta

    Atlanta, Georgiastudy coordinator listed

    Recruiting

  • Mattel Children's Hospital - UCLA

    Los Angeles, Californiastudy coordinator listed

    Recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Study leadership

  • Sung-Yun Pai, MD · STUDY_CHAIR · National Institutes of Health (NIH)

Who to contact

Colleen Dansereau
[email protected]
6179197008
Email the study team

Opens a ready-to-send draft in your own email app — review before sending.

What this trial measures

  • The primary objective is to measure event free survival1 year post infusion

    Events will include death, infusion of unmanipulated back-up product for failure of hematopoietic recovery, and allogeneic transplant performed for poor immune reconstitution

  • T cell reconstitution1 year post infusion

    * CD3+ T cell count ≥300 cells/microliter in peripheral blood * Gene marking ≥0.1 copies/cell in sorted CD3+ T cells