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NCT02022696 · ClinicalTrials.gov registry record · Phase 1
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
A Phase 1 study, sponsored by National Human Genome Research Institute (NHGRI).
- Completed
- Registry status
- Phase 1
- Development phase
- 1
- Enrollment target
NCT02022696: Completed Phase 1 study, sponsored by National Human Genome Research Institute (NHGRI).
NCT02022696 is a Phase 1 study that has completed, run by National Human Genome Research Institute (NHGRI). The registered enrollment target is 1 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (98% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02022696, a Phase 1 study, has completed, sponsored by National Human Genome Research Institute (NHGRI).
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 1 participants
- Enrollment target
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.
Interventions
- GENETIC Lentiviral Gene Transfer
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 1 participants |
| Start Date | 2013-12-16 |
| Est. Completion | 2017-09-21 |
| Phase | Phase 1 |
What the finished NCT02022696 record still lists
NCT02022696 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 1 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (98% lower).
The record links to 0 conditions, and to 1 intervention - of which Lentiviral Gene Transfer is the first listed.
NCT02022696 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT02022696 about?
NCT02022696 is a clinical study titled "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". 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. Th...
What is the current status of trial NCT02022696?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 1 participants. The study started on 2013-12-16. Estimated completion is 2017-09-21.
What interventions are being tested in trial NCT02022696?
The interventions under investigation include: Lentiviral Gene Transfer (GENETIC).
Who is sponsoring clinical trial NCT02022696?
This trial is sponsored by National Human Genome Research Institute (NHGRI), which has 185 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT02022696's enrollment target sits among peer trials
1 2001st of 2000 the lowest of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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