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

NCT04049084 · Status: ENROLLING_BY_INVITATION · Type: OBSERVATIONAL · Enrollment: 70

Last updated 2025-12-18

No results posted yet for this study

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.

Conditions

  • Adenosine Deaminase Deficiency
  • Severe Combined Immunodeficiency (SCID)

Interventions

BIOLOGICAL

autologous ex vivo gene therapy products based on the EFS LV encoding for the human adenosine deaminase (ADA) gene (EFS-ADA LV)

ADA-SCID patients previously treated with autologous ex vivo gene therapy products based on the EFS LV encoding for the human adenosine deaminase (ADA) gene (EFS-ADA LV)

Sponsors & Collaborators

Principal Investigators

  • Donald B. Kohn, M.D. · University of Califorina, Los Angeles

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-09-26
Primary Completion
2040-08-31
Completion
2040-08-31
FDA Drug
Yes

Countries

  • United States
  • United Kingdom

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04049084 on ClinicalTrials.gov