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Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID

NCT02999984 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2022-08-03

Study results available
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Summary

This is a prospective, non-randomized, single-cohort, longitudinal, single-center, clinical study designed to assess the efficacy and safety of a cryopreserved formulation of OTL-101 (autologous CD34+ hematopoietic stem/progenitor cells transduced ex vivo with EFS (Elongation Factor 1α Short form) Lentiviral Vector (LV) encoding for the human ADA gene) administered to ADA-SCID subjects between the ages of 30 days and 17 years of age, who are not eligible for an Human Leukocyte Antigen (HLA) matched sibling/family donor and meeting the inclusion/exclusion criteria. The OTL-101 product is infused after a minimal interval of at least 24 hours following the completion of reduced intensity conditioning. For subjects who successfully receive the OTL-101 product, pegademase bovine (PEG-ADA) Enzyme Replacement Therapy (ERT) is discontinued at Day+30 (-3/+15) after the transplant. After their discharge from hospital, the subjects will be seen at regular intervals to review their history, perform examinations and draw blood samples to assess immunity and safety.

Conditions

  • Severe Combined Immunodeficiency Due to ADA Deficiency

Interventions

GENETIC

Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101)

autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101) are infused intravenously

DRUG

busulfan

Busulfan is used for non-myeloablative conditioning

DRUG

PEG-ADA ERT

PEG-ADA ERT is discontinued at Day +30 (-3/+15 days) after successful engraftment

Sponsors & Collaborators

Principal Investigators

  • Donald B. Kohn, MD · University of California, Los Angeles

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
30 Days
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-12-16
Primary Completion
2018-10-11
Completion
2019-09-26
FDA Drug
Yes

Countries

  • United States

Study Locations

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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 NCT02999984 on ClinicalTrials.gov