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Gene Therapy

Gene Therapy for Severe Combined Immunodeficiency (SCID)

Phase 1 & 2
Recruiting
Led By Satiro DeOliveira, MD
Research Sponsored by University of California, Los Angeles
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Subjects ≥30 days of age
Females of child-bearing age will be required to provide a negative pregnancy test 30 days prior to Visit 2
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 24 months
Awards & highlights

Study Summary

This trialwill test how safe & effective it is to use stem cells from the blood of ADA-deficient SCID infants & children after they get gene therapy. Results will measure gene transfer & immune function.

Who is the study for?
This trial is for infants and children with ADA-SCID, a genetic disorder that affects the immune system. Participants must be at least 30 days old, unable to undergo bone marrow transplant from a family donor, and able to follow study procedures. Pregnant individuals or those with certain organ dysfunctions or previous gene therapy are excluded.Check my eligibility
What is being tested?
The trial tests a treatment where patients' own blood stem cells are modified outside the body using a special virus that carries a correct copy of the ADA gene. These modified cells are then returned to the patient's body to help restore their immune function.See study design
What are the potential side effects?
Potential side effects may include reactions related to cell infusion, changes in blood counts, and possible organ inflammation due to busulfan (a medication used before cell infusion). The exact side effects will be monitored closely given this is an innovative therapy.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am older than 30 days.
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I am a woman of child-bearing age and can provide a negative pregnancy test.
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I don't have a family member who can donate bone marrow to me.
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I have been diagnosed with ADA-SCID due to low ADA enzyme activity or specific gene mutations.
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I have a family history of ADA-SCID and show signs of severe immune deficiency.
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My legal guardians and I (if required) have signed the consent forms for this study.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~24 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 24 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Survival
Secondary outcome measures
Cessation of immunoglobulin replacement therapy (IgRT).
Determine incidence of Infection over two years after gene therapy
Evaluate Safety from clinical adverse events.
+6 more
Other outcome measures
Exploratory Study Objectives to measure biological correlates of efficacy (Immune reconstitution - measure serum immunoglobulins)
Exploratory Study Objectives to measure biological correlates of efficacy (Immune reconstitution - quantify T and B cell)
Exploratory Study Objectives to measure biological correlates of efficacy (Immune reconstitution - response to tetanus vaccine)
+5 more

Trial Design

1Treatment groups
Experimental Treatment
Group I: Autologous mobilized peripheral blood (mPB) transduced with EFS ADA lentiviral vectorExperimental Treatment1 Intervention
Evaluate safety and efficacy of this autologous gene therapy

Find a Location

Who is running the clinical trial?

University of California, Los AngelesLead Sponsor
1,525 Previous Clinical Trials
10,279,456 Total Patients Enrolled
Satiro DeOliveira, MDPrincipal InvestigatorAssistant Professor
Satiro De Oliveira, MDPrincipal InvestigatorAssistant Professor

Media Library

EFS-ADA Lentiviral Vector (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05432310 — Phase 1 & 2
Severe Combined Immune Deficiency Research Study Groups: Autologous mobilized peripheral blood (mPB) transduced with EFS ADA lentiviral vector
Severe Combined Immune Deficiency Clinical Trial 2023: EFS-ADA Lentiviral Vector Highlights & Side Effects. Trial Name: NCT05432310 — Phase 1 & 2
EFS-ADA Lentiviral Vector (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05432310 — Phase 1 & 2

Frequently Asked Questions

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

Does this medical trial currently have open enrollment?

"As indicated on clinicaltrials.gov, the trial is currently recruiting volunteers for participation. The initial post was made April 1st 2023 with a subsequent update occurring towards the end of January (24th)."

Answered by AI

What is the current size of the cohort engaged in this investigation?

"Affirmative, according to clinicaltrials.gov, this research is actively recruiting participants. Its initial posting was on April 1st 2023 and the last update was 24th of January in the same year. The study requires a total of 20 volunteers spread among one site."

Answered by AI
~7 spots leftby Dec 2024