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Gene Therapy for Severe Combined Immunodeficiency (SCID)
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.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowTimeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Trial Design
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Who is running the clinical trial?
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- I am older than 30 days.I am a woman of child-bearing age and can provide a negative pregnancy test.I don't have a family member who can donate bone marrow to me.You have a positive test for HIV-1, Hepatitis B, or Parvovirus B19.My kidney function is not normal, or I have very high levels of sodium, potassium, calcium, magnesium, or phosphate.I am not eligible for a stem cell transplant using my own cells.I do not have serious heart problems or a weak heart.I have cancer other than DFSP or my DFSP requires treatment soon or is life-limiting, and I'm not allergic to Busulfan.I have been diagnosed with ADA-SCID due to low ADA enzyme activity or specific gene mutations.I have blood disorders such as anemia or abnormal bleeding/clotting times.My oxygen levels are below 90% without assistance, or I have an active lung problem shown on an X-ray.I have a family history of ADA-SCID and show signs of severe immune deficiency.My legal guardians and I (if required) have signed the consent forms for this study.I have had gene therapy before.I have a significant neurological issue or uncontrolled seizures.My liver tests and blood sugar levels are within normal limits.
- Group 1: Autologous mobilized peripheral blood (mPB) transduced with EFS ADA lentiviral vector
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
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)."
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."
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