MedTrace Logo

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

NCT03645460 · Status: NOT_YET_RECRUITING · Phase: NA · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2026-08-26

No results posted yet for this study

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.

Conditions

  • Adenosine DeAminase Severe Combined ImmunoDeficiency (ADA-SCID)

Interventions

GENETIC

Direct intravenous injection of ivlv-ADA lentiviral vector

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

Sponsors & Collaborators

  • Shenzhen Geno-Immune Medical Institute

    lead OTHER

Principal Investigators

  • Lung-Ji Chang, Ph.D · Shenzhen Geno-Immune Medical Institute

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
1 Month
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2027-06-30
Primary Completion
2028-07-31
Completion
2028-12-31

Countries

  • China

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03645460 on ClinicalTrials.gov