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Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) Deficiency

NCT01380990 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 36

Last updated 2021-09-16

Study results available
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Summary

This is a historically controlled, non-randomized Phase I/II clinical trial to assess the safety and efficacy of autologous transplantation of CD34+ hematopoietic stem/progenitor cells (HSPCs), obtained from infants affected by ADA-SCID, following transduction of the HSPCs with a lentiviral vector (LV) carrying the human ADA complementary DNA (cDNA) under the control of the elongation factor 1 alpha shortened (EFS) promoter. Subjects treated in the trial receive the infusion of autologous, transduced cells following marrow cytoreduction with busulfan. The outcomes are compared to those observed in a historical control group of patients who received an allogeneic hematopoietic stem cell transplant (HSCT).

This Phase I/II clinical trial will be performed at Great Ormond Street Hospital (GOSH), London, United Kingdom.

Conditions

  • Adenosine Deaminase Deficiency
  • Severe Combined Immunodeficiencies (SCID)

Interventions

GENETIC

Infusion of autologous EFS-ADA LV CD34+ cells

Autologous EFS-ADA LV CD34+ cells (OTL-101\*) are infused intravenously

OTHER

Haematopoietic Stem Cell Transplantation (HSCT)

Historical data from a database of ADA-SCID patients treated with allogeneic HSCT from GOSH will be collected as comparator group.

DRUG

Busulfan

Busulfan is used for non-myeloablative conditioning

DRUG

Peg-Ada

Peg-Ada enzyme replacement therapy is discontinued at Day +3- (-3/+15 days) after successful engraftment

Sponsors & Collaborators

  • Orchard Therapeutics

    collaborator INDUSTRY

  • Great Ormond Street Hospital for Children NHS Foundation Trust

    lead OTHER

Principal Investigators

  • Claire Booth, Dr · Great Ormond Street Hospital NHS Foundation Trust

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Max Age
15 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2012-11-15
Primary Completion
2019-12-23
Completion
2019-12-23

Countries

  • United Kingdom

Study Locations

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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 NCT01380990 on ClinicalTrials.gov