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NCT01852071 · ClinicalTrials.gov registry record · Phase 1
Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene
A Phase 1 study, sponsored by University of California, Los Angeles.
- Completed
- Registry status
- Phase 1
- Development phase
- 46
- Enrollment target
NCT01852071 is a Phase 1 study that has completed, run by University of California, Los Angeles. The registered enrollment target is 46 participants.
The verdict
NCT01852071, a Phase 1 study, has completed, sponsored by University of California, Los Angeles.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 46 participants
- Enrollment target
Study 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.
Interventions
- DRUG busulfan
- DRUG PEG-ADA ERT
- GENETIC Infusion of autologous EFS-ADA LV CD34+ (OTL-101)
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 46 participants |
| Start Date | 2013-08-02 |
| Est. Completion | 2018-08-27 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT01852071
The ClinicalTrials.gov registry entry for NCT01852071 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 46 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is University of California, Los Angeles, which has 897 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 3 interventions - of which busulfan is the first listed.
NCT01852071 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT01852071 about?
NCT01852071 is a clinical study titled "Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene". 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 bl...
What is the current status of trial NCT01852071?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 46 participants. The study started on 2013-08-02. Estimated completion is 2018-08-27.
What interventions are being tested in trial NCT01852071?
The interventions under investigation include: busulfan (DRUG), PEG-ADA ERT (DRUG), Infusion of autologous EFS-ADA LV CD34+ (OTL-101) (GENETIC).
Who is sponsoring clinical trial NCT01852071?
This trial is sponsored by University of California, Los Angeles, which has 897 total clinical trials registered on ClinicalTrials.gov.
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