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

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)

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.

Study Overview

• Status: Enrolling by invitation
• Conditions: Adenosine Deaminase Deficiency; Severe Combined Immunodeficiency (SCID)
• Intervention / Treatment: 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
• Enrollment (Anticipated): 70

Contacts and Locations

Study Locations

• United Kingdom
  • London, United Kingdom, WC1N 1EH
    • UCL Great Ormond Street Institute of Child Health
• United States
  • California
    • Los Angeles, California, United States, 90095
      • Mattel Children's Hospital UCLA/Ronald Reagan UCLA Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

This observational LTFU study is open for enrollment to ADA-SCID patients previously treated with autologous ex vivo gene therapy products based on the EFS-ADA lentiviral vector as part of the OTL-101 clinical development program. Patients treated in clinical trials as part of IND #15440, study OTL-101-5 (EudraCT No 2017-001275-23) or study 10-MI-29 (EudraCT No 2010-024253-36), as well as in Expanded Access or Compassionate Use Programs, will be eligible for enrollment.

Description

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.

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

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

Collaborators and Investigators

• Sponsor: University of California, Los Angeles
• Collaborators: Great Ormond Street Hospital for Children NHS Foundation Trust

Study record dates

Study Major Dates

• Study Start (Actual): September 26, 2019
• Primary Completion (Anticipated): August 1, 2035
• Study Completion (Anticipated): August 1, 2035

Study Registration Dates

• First Submitted: August 6, 2019
• First Submitted That Met QC Criteria: August 6, 2019
• First Posted (Actual): August 7, 2019

Study Record Updates

• Last Update Posted (Estimate): May 5, 2023
• Last Update Submitted That Met QC Criteria: May 3, 2023
• Last Verified: May 1, 2023

More Information

Terms related to this study

• Keywords: OTL-101
• Additional Relevant MeSH Terms: Metabolic Diseases; Immune System Diseases; Infant, Newborn, Diseases; Genetic Diseases, Inborn; DNA Repair-Deficiency Disorders; Primary Immunodeficiency Diseases; Immunologic Deficiency Syndromes; Severe Combined Immunodeficiency; Physiological Effects of Drugs; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Anti-Arrhythmia Agents; Vasodilator Agents; Peripheral Nervous System Agents; Analgesics; Sensory System Agents; Purinergic Agents; Purinergic P1 Receptor Agonists; Purinergic Agonists; Adenosine
• Other Study ID Numbers: OTL-101-6

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No