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Treatment of SCID Due to ADA Deficiency With Autologous Transplantation of Cord Blood or Hematopoietic CD 34+ Cells After Addition of a Normal Human ADA cDNA by the EFS-ADA Lentiviral Vector

NCT02022696 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 1

Last updated 2017-09-27

No results posted yet for this study

Summary

This is a clinical gene transfer study that aims to verify the safety and efficacy of the use of the EFS-ADA lentiviral vector to introduce the human adenosine deaminase (ADA) gene into the hematopoietic progenitors of patients affected with severe combined immunodeficiency due to ADA deficiency. The EFS-ADA vector expresses the human ADA cDNA under the control of the elongation factor alpha short promoter (EFS). In addition, this protocol will examine the effects of the ADA gene transfer on the immune system of treated patients. Patients with ADA deficiency and ineligible for matched sibling allogeneic bone marrow transplantation are eligible to participate in the study. To increase engraftment and selected advantage or gene-corrected cells, busulfan will be used as a cytoreductive agent. Enzyme replacement (PEG-ADA) will be discontinued 30 days after infusion of gene-corrected cells. CD34+ hematopoietic progenitors will be isolated from the patient bone marrow, peripheral blood or cord blood, exposed to lentiviral vector-mediated gene transfer and re-infused into the patient through a peripheral vein. Clinical, immunological and molecular follow-up studies will assess safety, toxicity, and efficacy of the procedure.

Conditions

  • Adenosine Deaminase Deficiency
  • ADA-SCID

Interventions

GENETIC

Lentiviral Gene Transfer

Sponsors & Collaborators

  • UCLA@@@Duke University Medical Center

    collaborator UNKNOWN

  • Duke Univ. Medical Center

    collaborator UNKNOWN

  • National Cancer Institute (NCI)

    collaborator NIH

  • National Institutes of Health Clinical Center (CC)

    collaborator NIH

  • National Human Genome Research Institute (NHGRI)

    lead NIH

Principal Investigators

  • Elizabeth K Garabedian, R.N. · National Human Genome Research Institute (NHGRI)

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
1 Year
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2013-12-16
Primary Completion
2013-12-16
Completion
2017-09-21

Countries

  • United States

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 NCT02022696 on ClinicalTrials.gov