Actively Recruiting
Gene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and EFS ADA Vector
Led by University of California, Los Angeles · Updated on 2026-04-07
20
Participants Needed
1
Research Sites
260 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The aim of this study is to assess the safety and efficacy of autologous transplantation of hematopoietic stem cells (CD34+ cells) from mobilized peripheral blood (mPB) of ADA-deficient SCID infants and children following human ADA gene transfer by the EFS-ADA lentiviral vector. The level of gene transfer in blood cells and immune function will be measured as endpoints.
CONDITIONS
Official Title
Gene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and EFS ADA Vector
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Written informed consent provided by parents/legal guardians and assent from the child when applicable
- Child is at least 30 days old
- Diagnosis of ADA-SCID confirmed by decreased ADA enzyme activity or mutations in ADA alleles
- Evidence of severe immunologic deficiency based on family history or clinical laboratory tests
- Ineligible for matched family allogeneic bone marrow transplantation due to absence of a suitable donor
- Negative pregnancy test for females of child-bearing age 30 days before study procedures
- Willingness and ability of child and parents/legal guardians to comply with study procedures and follow-up visits
You will not qualify if you...
- Ineligibility for autologous hematopoietic stem cell transplantation by clinical site criteria
- Conditions contraindicating mobilization, leukapheresis, busulfan administration, cell infusion, or protocol compliance
- Hematologic abnormalities including anemia (Hb <8.0 g/dl), neutropenia (ANC <500/mm3), thrombocytopenia (platelets <50,000/mm3), or coagulation abnormalities
- Cytogenetic abnormalities in blood, bone marrow, or amniotic fluid
- Prior allogeneic hematopoietic stem cell transplant with cytoreductive conditioning
- Pulmonary abnormalities such as resting oxygen saturation <90% or active lung disease
- Cardiac abnormalities including abnormal ECG, uncorrected congenital defects, active heart disease, or poor heart function
- Neurologic abnormalities including significant findings or uncontrolled seizures
- Renal abnormalities including elevated creatinine or proteinuria and abnormal electrolyte levels
- Hepatic/gastrointestinal abnormalities including elevated liver enzymes, bilirubin, or glucose
- Active oncologic disease other than dermatofibrosarcoma protuberans (DFSP) or DFSP requiring treatment or life limiting within 5 years
- Known sensitivity to busulfan
- Positive DNA PCR for HIV-1, Hepatitis B, or Parvovirus B19 at screening
- Pregnancy or major congenital anomalies
- Likely need for prohibited medications during the study
- Previous receipt of other gene therapy
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
University of California, Los Angeles (UCLA)
Los Angeles, California, United States, 90095
Actively Recruiting
Research Team
S
Satiro De Oliveira, MD
CONTACT
A
Augustine Fernandes, PhD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here