RecruitingNot ApplicableNCT03645460

Gene Therapy for ADA-SCID Using an Improved Lentiviral Vector (Ivlv-ADA)

Gene Therapy Via Intravenous Injection of Lentiviral Vector (Ivlv-ADA) for the Treatment of Adenosine Deaminase-severe Combined Immunodeficiency (ADA-SCID)

Sponsor

Shenzhen Geno-Immune Medical Institute

Enrollment

10 participants

Start Date

Jun 30, 2024

Study Type

INTERVENTIONAL

Conditions

Adenosine DeAminase Severe Combined ImmunoDeficiency (ADA-SCID)

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

Min Age: 1 Month

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying Direct intravenous injection of ivlv-ADA lentiviral vector for people with adenosine deaminase severe combined immunodeficiency (ada-scid). The study is currently recruiting participants at 1 location. People eligible for this study include aged 1 Month and older.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

GENETICDirect intravenous injection of ivlv-ADA lentiviral vector

Injection of ivlv-ADA lentiviral vector at \~1x10\^9 per kg body weight

Locations(1)

Shenzhen Geno-immune Medical Institute

Shenzhen, Guangdong, China

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NCT03645460