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Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene

NCT01852071 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 46

Last updated 2022-08-03

Study results available
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Summary

The aim of this study is to assess the safety and efficacy of autologous transplantation of hematopoietic stem cells (CD34+ cells) from the bone marrow (BM) of ADA-deficient SCID infants and children following human ADA cDNA transfer by the EFS-ADA lentiviral vector. The level of gene transfer in blood cells and immune function will be measured as endpoints.

Conditions

  • ADA-SCID

Interventions

GENETIC

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

autologous 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

  • National Institute of Allergy and Infectious Diseases (NIAID)

    collaborator NIH

  • National Human Genome Research Institute (NHGRI)

    collaborator NIH

  • National Heart, Lung, and Blood Institute (NHLBI)

    collaborator NIH

  • Orchard Therapeutics

    collaborator INDUSTRY

  • University of California, Los Angeles

    lead OTHER

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
1 Month
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2013-08-02
Primary Completion
2018-08-27
Completion
2018-08-27
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 NCT01852071 on ClinicalTrials.gov