By InvitationNot Applicable

An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID

United States, United Kingdom70 participantsStarted 2019-09-26

Plain-language summary

This observational long-term follow-up study is designed to collect safety and efficacy data from ADA-SCID patients previously treated with autologous ex vivo gene therapy products based on the EFS-ADA LV encoding for human adenosine deaminase (ADA) gene (EFS-ADA LV), as part of the OTL-101 clinical development program. No investigational medicinal product will be administered to these patients as part of the OTL-101-6 study.

Who can participate

SexALL

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Inclusion criteria

✓. the patient has been treated with an autologous ex vivo gene therapy product based on the EFS-ADA LV, as part of the OTL-101 clinical development program;
✓. the patient displays persistent detectable gene marking, as determined by the Investigator;
✓. the patient or, if applicable, the patient's parent(s)/legal guardian(s), are able and willing to provide informed consent.

What they're measuring

Overall Survival

Timeframe: 15 years post-treatment

Event-Free Survival

Timeframe: 15 years post-treatment

Incidence of Adverse Events

Timeframe: 15 years post-treatment

Trial details

NCT IDNCT04049084

SponsorUniversity of California, Los Angeles

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2040-08

View on ClinicalTrials.gov More Adenosine Deaminase Deficiency trials