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Gene Therapy

Gene Therapy for SCID

Phase 1 & 2
Waitlist Available
Research Sponsored by David Williams
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Age 5 years old or younger
If the patient has previously undergone allogeneic transplant, lack of donor T cell engraftment must be documented
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 year post infusion
Awards & highlights

Study Summary

This trial is a study to test a new treatment for SCID, in which patients receive low doses of targeted busulfan followed by infusion of their own stem cells that have been modified with a virus to correct the SCID gene. The trial will enroll subjects over 3 years and follow them for 2 years afterwards to see how well the treatment works.

Who is the study for?
This trial is for children aged 5 or younger with SCID-X1, a severe immune deficiency known as 'Bubble Boy Disease'. They must lack T cell function and have a specific genetic mutation. Kids without an identical donor match and who haven't had successful previous transplants can join. Participants need to be at least 8 weeks old by busulfan treatment time and commit to long-term follow-up.Check my eligibility
What is being tested?
The study tests gene therapy for SCID-X1 using the patient's own cells modified with G2SCID vector after low dose busulfan conditioning. It's an open-label, multi-center trial where patients are followed for two years post-infusion, then on a separate long-term protocol.See study design
What are the potential side effects?
Potential side effects may include reactions related to low dose busulfan such as digestive issues, mouth sores, fatigue, skin changes, or problems with liver function. The gene therapy could cause immune reactions or influence blood cell counts.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am 5 years old or younger.
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I had an allogeneic transplant and it's confirmed that the donor T cells did not engraft.
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I do not have a family member who is a perfect match for a bone marrow transplant.
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I have SCID-X1 confirmed by a specific gene mutation and poor T cell function.
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I am willing to follow up for 15 years after treatment as required.
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I am at least 8 weeks old at the time of receiving busulfan.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1 year post infusion
This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year post infusion for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
T cell reconstitution
The primary objective is to measure event free survival

Trial Design

1Treatment groups
Experimental Treatment
Group I: Treatment armExperimental Treatment1 Intervention
single infusion of autologous CD34+ cells transduced with the self-inactivating (SIN) lentiviral vector G2SCID

Find a Location

Who is running the clinical trial?

David WilliamsLead Sponsor
4 Previous Clinical Trials
48 Total Patients Enrolled
Sung-Yun Pai, MDStudy ChairNational Institutes of Health (NIH)
1 Previous Clinical Trials
64 Total Patients Enrolled

Media Library

G2SCID vector (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT03311503 — Phase 1 & 2
Bubble Boy Disease Research Study Groups: Treatment arm
Bubble Boy Disease Clinical Trial 2023: G2SCID vector Highlights & Side Effects. Trial Name: NCT03311503 — Phase 1 & 2
G2SCID vector (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03311503 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What is the current level of participant enrollment in this experiment?

"Affirmative. The information hosted on clinicaltrials.gov indicates that this scientific experiment is presently recruiting participants; it was initially posted on January 19th 2018 and has recently been updated on July 19th 2022. Recruitment for 10 patients between 4 sites is necessary to run the trial successfully."

Answered by AI

Are there any Canadian healthcare centers currently conducting this clinical trial?

"This clinical trial is being conducted at four locations, including Mattel Children's Hospital - UCLA in Los Angeles, Emory University/Childrens Healthcare of Atlanta in Atlanta, and Cincinnati Children's Hospital Medical Center."

Answered by AI

Are there any openings available in this clinical trial yet?

"Affirmative. Evidence hosted on clinicaltrials.gov confirms that this research endeavour, which was initially made public on January 19th 2018, is actively seeking out volunteers. At present, 10 participants must be recruited from 4 distinct medical centres."

Answered by AI
~2 spots leftby Oct 2025