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

University of California, Los Angeles (UCLA) logo

University of California, Los Angeles (UCLA)

Status

Invitation-only

Conditions

Adenosine Deaminase Deficiency
Severe Combined Immunodeficiency (SCID)

Treatments

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

Study type

Observational

Funder types

Other

Identifiers

NCT04049084
OTL-101-6

Details and patient eligibility

About

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.

Enrollment

70 estimated patients

Sex

All

Volunteers

No Healthy Volunteers

Inclusion criteria

A patient is eligible for enrollment in the study if all of the following criteria are met:

  1. 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;
  2. the patient displays persistent detectable gene marking, as determined by the Investigator;
  3. the patient or, if applicable, the patient's parent(s)/legal guardian(s), are able and willing to provide informed consent.

Exclusion criteria

  • There are no exclusion criteria for participation in this observational LTFU study.

Trial contacts and locations

2

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Data sourced from clinicaltrials.gov

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