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Gene Therapy for ADA-SCID Using EFS-ADA Lentiviral Vector

This study is testing a gene therapy for infants and children with Adenosine Deaminase Severe Combined Immune Deficiency (ADA-SCID), a rare genetic disorder that severely weakens the immune system. The therapy involves taking your own blood stem cells, modifying them in the lab with a special virus called EFS-ADA lentiviral vector to carry the healthy ADA gene, and then giving them back to you. Before receiving the modified cells, you will get a chemotherapy medicine called busulfan. Researchers want to see if this treatment is safe and effective in helping your body produce the necessary ADA enzyme and rebuild your immune system. They will measure your survival at 24 months to determine success. This study plans to enroll 20 participants and its current status is unclear.

Study design
This is an interventional study planning to enroll 20 infants and children with ADA-SCID who do not have an HLA-matched sibling donor.
What's involved
You will receive your own gene-modified cells after a reduced intensity conditioning with busulfan. After the transplant, PEG-ADA enzyme replacement therapy will be withheld starting on Day +30.
Compensation
Not stated in the trial record.
Follow-up
The primary endpoint measures survival at 24 months. Patients will be asked to enroll in a long-term follow-up study for a total of 15 years after gene therapy.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT05432310

Gene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and EFS ADA Vector

Active, Not Recruiting
PHASE1Ages 1+InterventionalTreatment
University of California, Los Angeles
~20 participants
Updated 2026-08-20 on ClinicalTrials.gov
What's tested:A cryopreserved formulation of autologous mPB CD34+ hematopoietic stem and progenitor cells transduced ex vivo with the EFS-ADA lentiviral vector encoding the human ADA enzyme

At a glance

Recruiting sites
0 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Survival
Measured over 24 months

Conditions

Adenosine Deaminase Severe Combined Immune Deficiency

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NCT05432310

Where you'd take part

This study runs at 1 site. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • University of California, Los Angeles (UCLA)

    Los Angeles, Californiano site contact published

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Study leadership

  • Satiro De Oliveira, MD · PRINCIPAL_INVESTIGATOR · Assistant Professor

Who to contact

This trial hasn't published a contact. View it on ClinicalTrials.gov

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Eligibility criteria

Exclusion

Anemia (Hb \<8.0 g/dl).
Neutropenia (ANC \<500/mm3). Note: ANC \<500 with absence of myelodysplastic syndrome on bone marrow aspirate and biopsy and normal marrow cytogenetics are acceptable for eligibility.
Thrombocytopenia (platelet count \<50,000/mm3, at any age).
Prothrombin time or international normalized ratio (INR) and partial thromboplastin time (PTT) \>2 x upper limit of normal (ULN) (subjects with a correctable deficiency controlled on medication will not be excluded).
Cytogenetic abnormalities on peripheral blood or bone marrow or amniotic fluid (if available).
Prior allogeneic HSCT with cytoreductive conditioning. 4. Pulmonary abnormality, defined as:
Resting O2 saturation by pulse oximetry \<90% on room air.
Chest X-ray indicating active or progressive pulmonary disease. Note: Chest X ray indicating residual signs of treated pneumonitis is acceptable for eligibility. 5. Cardiac abnormality, defined as:
Abnormal ECG indicating cardiac pathology.
Uncorrected congenital cardiac malformation with clinical symptoms.
Active cardiac disease, including clinical evidence of congestive heart failure, cyanosis, hypotension.
Poor cardiac function as evidenced by left ventricular ejection fraction \<40% on echocardiogram. 6. Neurologic abnormality, defined as:
Significant neurologic abnormality revealed by examination.
Uncontrolled seizure disorder. 7. Renal abnormality, defined as:
Renal insufficiency: serum creatinine ≥1.2 mg/dl (106 µmol/L), or ≥3+ proteinuria.
Abnormal serum sodium, potassium, calcium, magnesium or phosphate levels at \>2 x ULN. 8. Hepatic/gastrointestinal abnormality, defined as:
Serum transaminases \>5 x ULN.
Serum bilirubin \>2 x ULN.
Serum glucose \>1.5 x ULN. 9. Oncologic disease, defined as:
Evidence of active malignant disease other than dermatofibrosarcoma protuberans (DFSP).
Evidence of DFSP expected to require anti-neoplastic therapy within the 5 years following the infusion of genetically corrected cells (if anti-neoplastic therapy has been completed, a subject with a history of DFSP can be included).
Evidence of DFSP expected to be life limiting within the 5 years following the infusion of genetically corrected cells. 10. Known sensitivity to Busulfan. 11. Confirmation of an infectious disease by deoxyribonucleic acid (DNA) polymerase chain reaction (PCR) positive at time of assessment for the following:
HIV-1,
Hepatitis B,
Parvovirus B19. 12. The subject is pregnant or has a major congenital anomaly. 13. Is likely to require treatment during the study with drugs that are not permitted by the study protocol. 14. The subject has previously received another form of gene therapy.

What this trial measures

  • Survival24 months

    The primary study outcome will be to determine survival for all subjects 2 years after gene therapy