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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: Completed Phase 1 study of Severe Combined Immunodeficiency Due to ADA Deficiency, sponsored by University of California, Los Angeles.
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, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (83% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
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.
Primary Outcome
Efficacy of OTL-101 treatment at 6 months post OTL-101 infusion based on the following parameters and thresholds: 1. ADA enzyme activity in erythrocytes above baseline/pretreatment level (i.e., \>0 units). ADA enzyme activity is measured to assess the amount of functional gene product produced from the normal ADA transgene delivered by EFS-ADA LV. 2. Absolute CD3+ T cell counts ≥200 cells/μL. Increase in CD3+ T cell counts is a marker of immune reconstitution. 3. Granulocyte samples positive fo
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 |
What the finished NCT02999984 record still lists
NCT02999984 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 10 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (83% lower).
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 a single indexed study location in 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.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT02999984's enrollment target sits among peer trials
10 1913th of 2000 higher than 46 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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