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NCT02999984 · ClinicalTrials.gov registry record · Phase 1
Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID
A Phase 1 study of Severe Combined Immunodeficiency Due to ADA Deficiency, sponsored by University of California, Los Angeles.
- Completed
- Registry status
- Phase 1
- Development phase
- 10
- Enrollment target
- 1
- Study location
NCT02999984 is a Phase 1 study of Severe Combined Immunodeficiency Due to ADA Deficiency that has completed, run by University of California, Los Angeles. The registered enrollment target is 10 participants. The trial reports 1 study location across 1 state.
The verdict
NCT02999984, a Phase 1 study of Severe Combined Immunodeficiency Due to ADA Deficiency, has completed, sponsored by University of California, Los Angeles.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 10 participants
- Enrollment target
- 1
- Study location
Study Summary
This is a prospective, non-randomized, single-cohort, longitudinal, single-center, clinical study designed to assess the efficacy and safety of a cryopreserved formulation of OTL-101 (autologous CD34+ hematopoietic stem/progenitor cells transduced ex vivo with EFS (Elongation Factor 1α Short form) Lentiviral Vector (LV) encoding for the human ADA gene) administered to ADA-SCID subjects between the ages of 30 days and 17 years of age, who are not eligible for an Human Leukocyte Antigen (HLA) matched sibling/family donor and meeting the inclusion/exclusion criteria. The OTL-101 product is infused after a minimal interval of at least 24 hours following the completion of reduced intensity conditioning. For subjects who successfully receive the OTL-101 product, pegademase bovine (PEG-ADA) Enzyme Replacement Therapy (ERT) is discontinued at Day+30 (-3/+15) after the transplant. After their discharge from hospital, the subjects will be seen at regular intervals to review their history, perform examinations and draw blood samples to assess immunity and safety.
Conditions Studied
Interventions
- DRUG busulfan
- GENETIC Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101)
- DRUG PEG-ADA ERT
Study Locations (1)
California
- University of California, Los Angeles - Los Angeles
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 10 participants |
| Start Date | 2016-12-16 |
| Est. Completion | 2019-09-26 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT02999984
The ClinicalTrials.gov registry entry for NCT02999984 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 10 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 1 condition, with Severe Combined Immunodeficiency Due to ADA Deficiency appearing as the primary indexed condition, and to 3 interventions - of which busulfan is the first listed.
NCT02999984 reports 1 study location spanning 1 distinct geographic area - top geographies include California.
Frequently Asked Questions
What is clinical trial NCT02999984 about?
NCT02999984 is a clinical study titled "Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID". This is a prospective, non-randomized, single-cohort, longitudinal, single-center, clinical study designed to assess the efficacy and safety of a cryopreserved formulation of OTL-101 (autologous CD34+ hematopoietic stem/progenitor cells transduced ex vivo with EFS (Elongation Factor 1α Short form) L...
What is the current status of trial NCT02999984?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 10 participants. The study started on 2016-12-16. Estimated completion is 2019-09-26.
What conditions does trial NCT02999984 study?
This clinical trial studies the following conditions: Severe Combined Immunodeficiency Due to ADA Deficiency.
What interventions are being tested in trial NCT02999984?
The interventions under investigation include: busulfan (DRUG), Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101) (GENETIC), PEG-ADA ERT (DRUG).
Who is sponsoring clinical trial NCT02999984?
This trial is sponsored by University of California, Los Angeles, which has 897 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT02999984 being conducted?
This trial has 1 study location across California. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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