CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 46 enrolled
Drug / intervention
Infusion of autologous EFS-ADA LV CD34+ (OTL-101) +2 moregenetic
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT01852071
NCT01852071Phase 2Completed
CompareCT.gov

Autologous Transplantation of Bone Marrow CD34+ Stem/Progenitor Cells After Addition of a Normal Human ADA Complementary DNA (cDNA) by the EFS-ADA Lentiviral Vector for Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency (ADA-SCID)

University of California, Los Angeles·interventional·Posted May 13, 2013·Updated Aug 3, 2022

In Brief

A Phase 2 clinical trial evaluating Infusion of autologous EFS-ADA LV CD34+ (OTL-101), busulfan, and 1 other intervention for ADA-SCID. Completed, enrolled 46 participants across 2 sites.

Detailed 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.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsADA-SCID
CountriesUnited States
CollaboratorsNational Institute of Allergy and Infectious Diseases (NIAID), National Human Genome Research Institute (NHGRI), National Heart, Lung, and Blood Institute (NHLBI), Orchard Therapeutics

Timeline

Phase 2CompletedFinished
20132014201520162017201820192020202120222023202420252026
First PostedMay 13, 2013
Enrollment StartAug 2, 2013
Primary CompletionAug 27, 2018
TodayJul 2, 2026
Enrollment to primary: 5.1 yearsPosted 13.1 years ago

Interventions

Infusion of autologous EFS-ADA LV CD34+ (OTL-101)genetic

autologous EFS-ADA LV CD34+ cells (OTL-101) are infused intravenously

busulfandrug

Busulfan is used for non-myeloablative conditioning

PEG-ADA ERTdrug

PEG-ADA ERT is discontinued at Day +30 (-3/+15 days) after successful engraftment