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

Adenosine Deaminase Deficiency Clinical Trial

Official title:

An Observational Long-term Follow-up Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency (ADA-SCID)

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT04049084
• Other study ID #: OTL-101-6
• Status: Enrolling by invitation
• Start date: September 26, 2019
• Est. completion date: August 2035

Study information

• Verified date: May 2023
• Source: University of California, Los Angeles
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 70
• Est. completion date: August 2035
• Est. primary completion date: August 2035
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Adenosine Deaminase Deficiency
• Immunologic Deficiency Syndromes
• Severe Combined Immunodeficiency
• Severe Combined Immunodeficiency (SCID)

Intervention

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)

Locations

Country	Name	City	State
United Kingdom	UCL Great Ormond Street Institute of Child Health	London
United States	Mattel Children's Hospital UCLA/Ronald Reagan UCLA Medical Center	Los Angeles	California

Sponsors (2)

Lead Sponsor	Collaborator
University of California, Los Angeles	Great Ormond Street Hospital for Children NHS Foundation Trust

Countries where clinical trial is conducted

United States,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Overall Survival	Establishing efficacy through overall survival	15 years post-treatment
Primary	Event-Free Survival	Establishing efficacy through event-free survival	15 years post-treatment
Primary	Incidence of Adverse Events	Long-Term Safety incidence of AEs and SAEs	15 years post-treatment