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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
Must not have
Mechanical ventilation including continuous positive airway pressure
Patients with an active, therapy-resistant infection. Infections that are known to be highly morbid in SCID patients will be considered active and therapy-resistant if the infectious agent is repeatedly isolated despite a minimum of 2 weeks of appropriate therapy and is associated with significant organ dysfunction
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 year post infusion
Awards & highlights
No Placebo-Only Group

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.
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.
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.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I am on a mechanical ventilator or use CPAP.
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I have an infection that hasn't improved after 2 weeks of treatment.
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My liver tests are much higher than normal.
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I have a condition that affects how my body repairs DNA.
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I have a cancer diagnosis that is not related to EBV.
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I am HIV-1 positive.
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I do not have severe birth defects that threaten my life.
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I have seizures that are not controlled by medication.
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My kidney function is very low or I am on dialysis.
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I have been diagnosed with encephalopathy.

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

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

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

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
~1 spots leftby Oct 2025
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