Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID
Public ClinicalTrials.gov record NCT02999984. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
Efficacy and Safety of Cryopreserved Formulation of Autologous CD34+ Hematopoietic Stem Cells Transduced Ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for Human ADA Gene in Subjects With Severe Combined Immunodeficiency Due to ADA Deficiency
Study identification
- NCT ID
- NCT02999984
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Lead sponsor
- University of California, Los Angeles
- Other
- Enrollment
- 10 participants
Conditions and interventions
Interventions
- Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101) Genetic
- busulfan Drug
- PEG-ADA ERT Drug
Genetic · Drug
Eligibility (public fields only)
- Age range
- 30 Days to 17 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Dec 15, 2016
- Primary completion
- Oct 10, 2018
- Completion
- Sep 25, 2019
- Last update posted
- Aug 2, 2022
2016 – 2019
United States locations
- U.S. sites
- 1
- U.S. states
- 1
- U.S. cities
- 1
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of California, Los Angeles | Los Angeles | California | 90095 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT02999984, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Aug 2, 2022 · Synced Jul 22, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT02999984 live on ClinicalTrials.gov.