NCT03645460 Gene Therapy for ADA-SCID Using an Improved Lentiviral Vector (Ivlv-ADA)
| NCT ID | NCT03645460 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Shenzhen Geno-Immune Medical Institute |
| Condition | Adenosine DeAminase Severe Combined ImmunoDeficiency (ADA-SCID) |
| Study Type | INTERVENTIONAL |
| Enrollment | 10 participants |
| Start Date | 2024-06-30 |
| Primary Completion | 2026-12-31 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
This trial targets 10 participants in total. It began in 2024-06-30 with a primary completion date of 2026-12-31.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
This is a Phase I/II trial of in vivo lentiviral gene therapy for treating adenosine deaminase severe combined immunodeficiency (ADA-SCID) using a self-inactivating lentiviral vector (LV) ivlv-ADA to functionally correct the genetic defect. The primary objectives are to evaluate the safety and efficacy of the direct intravenous (iv) LV gene therapy protocol.
Eligibility Criteria
Inclusion Criteria: * Diagnosis of classical ADA-SCID based on: * A proven defective adenosine deaminase (ADA) gene as defined by direct sequencing of patient DNA. * T-cell immune deficiency defined as one or more of the following: CD3+ autologous T cells \< 300/ul, or less than 50% of normal value for in vitro mitogen stimulation, or absent proliferation in vitro to antigens. * With severe infections, including but not limited to: pneumonitis; protracted diarrhea requiring total parenteral nutrition; infection with herpes viruses or adenovirus or fungus; disseminated BCG infection. * No cytogenetic abnormalities (medullary karyotype) and no detection of main rearrangements associated with acute leukemia of children. * No prior allogeneic stem cell transplantation. * Life expectancy ≥ 2 months. * Negative for HIV infection. * Written, informed consent obtained prior to any study-specific procedures. Exclusion Criteria: * None
Contact & Investigator
Lung-Ji Chang, Ph.D
PRINCIPAL INVESTIGATOR
Shenzhen Geno-Immune Medical Institute
Frequently Asked Questions
Who can join the NCT03645460 clinical trial?
This trial is open to participants of all sexes, aged 1 Month or older, studying Adenosine DeAminase Severe Combined ImmunoDeficiency (ADA-SCID). Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT03645460 currently recruiting?
Yes, NCT03645460 is actively recruiting participants. Contact the research team at c@szgimi.org for enrollment information.
Where is the NCT03645460 trial being conducted?
This trial is being conducted at Shenzhen, China.
Who is sponsoring the NCT03645460 clinical trial?
NCT03645460 is sponsored by Shenzhen Geno-Immune Medical Institute. The principal investigator is Lung-Ji Chang, Ph.D at Shenzhen Geno-Immune Medical Institute. The trial plans to enroll 10 participants.