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Gene Therapy

Gene Therapy for Severe Combined Immunodeficiency

Phase 1 & 2
Waitlist Available
Research Sponsored by University of California, Los Angeles
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 24 months
Awards & highlights

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.

Eligible Conditions
  • Severe Combined Immunodeficiency

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~24 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 24 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Event-free Survival (EvFS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year)
Overall Survival (OS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year)
Percentage of Participants With Treatment Efficacy After Treatment With OTL 101 (6 Months)
Secondary study objectives
Change From Baseline in CD3+ T Cell Counts (2 Years)
Change From Baseline in Quality of Life Measures (2 Years)
EvFS of Subjects Treated With Investigational Medicinal Product (IMP) (2 Years)
+4 more

Trial Design

1Treatment groups
Experimental Treatment
Group I: Gene TherapyExperimental Treatment3 Interventions
Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101)
2016
Completed Phase 2
~30
busulfan
1999
Completed Phase 3
~3630
PEG-ADA ERT
2013
Completed Phase 2
~60

Find a Location

Who is running the clinical trial?

University of California, Los AngelesLead Sponsor
1,556 Previous Clinical Trials
10,263,803 Total Patients Enrolled
4 Trials studying Severe Combined Immunodeficiency
98 Patients Enrolled for Severe Combined Immunodeficiency
California Institute for Regenerative Medicine (CIRM)OTHER
68 Previous Clinical Trials
3,309 Total Patients Enrolled
1 Trials studying Severe Combined Immunodeficiency
28 Patients Enrolled for Severe Combined Immunodeficiency
Orchard TherapeuticsIndustry Sponsor
22 Previous Clinical Trials
106,436 Total Patients Enrolled
8 Trials studying Severe Combined Immunodeficiency
6,200 Patients Enrolled for Severe Combined Immunodeficiency
~1 spots leftby Sep 2025
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