Clin2
NCT04049084Possibly a fitEnrolling by invitation

Follow-up study for people treated with gene therapy for ADA-SCID

Adenosine Deaminase DeficiencySevere Combined Immunodeficiency (SCID)

Part of Genetic & congenital, Hormones & metabolism, Immune system & allergy clinical trials.

This is a follow-up (no new treatment) study for people who previously received a gene therapy made from their own cells for ADA-SCID. It mainly tracks long-term safety and whether a marker showing the gene change can still be found.

Summary written for real people, not researchers, by Clin2.

Phase
N/A
Enrollment
70 people
Ages
Any age
Study type
Observational

Who can take part

  • You previously received ADA-SCID gene therapy using your own cells (EFS-ADA LV/OTL-101 program).
  • Your study doctor can still detect a lasting “gene marking” signal in you.
  • You (or your parent/legal guardian) can understand and sign informed consent.
  • This is observational, meaning no extra study treatment is added.

View the official record on ClinicalTrials.gov

Quick eligibility check

Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.

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