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

Part of paid clinical trials in Los Angeles, California.

Sponsor
University of California, Los Angeles
Study ID
NCT04049084
Status
Enrolling By Invitation

Conditions

  • Adenosine Deaminase Deficiency
  • Severe Combined Immunodeficiency (SCID)

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Interventions

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

Study Details

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.

Key Dates

Start date
Sep 26, 2019
Status verified
Dec 2025
Primary completion
Aug 31, 2040
Completion
Aug 31, 2040

Study Design

Enrollment
70 participants (estimated)

Primary Outcome Measure

Overall Survival [ Time Frame: 15 years post-treatment ]

Locations (1)

FacilityCityStateZIPSite coordinators
Mattel Children's Hospital UCLA/Ronald Reagan UCLA Medical CenterLos AngelesCalifornia90095-

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Mattel Children's Hospital UCLA/Ronald Reagan UCLA Medical Center· Los Angeles, CA

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