SCID-X1 Gene Therapy Via Intravenous Lentiviral (Ivlv-X1) Injection

Recruiting Phase 1 Phase 2 Interventional Study
SCID, X-Linked
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
1 Month – 1
Sex
Male
Trial phase
Phase 1/2
Study type
Interventional
Purpose
Treatment
Participants needed
10 (estimated)
Sponsor
Shenzhen Geno-Immune Medical Institute · Other
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About This Trial

This is a Phase I/II gene therapy trial treating X-linked severe combined immunodeficiency (SCID-X1) using a self-inactivating lentiviral vector (ivlv-X1) to functionally correct the genetic defect. The primary objectives are to evaluate the safety and efficacy of the direct intravenous lentiviral gene transfer protocol.

Trial Locations
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Eligibility Criteria
Inclusion Criteria: 1. Diagnosis of SCID-X1 based on: * A proven mutation in the common interleukin-2 receptor gamma chain 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 …
Contacts

Lung-Ji Chang, Ph.D

+86 0755-86573763

c@szgimi.org

CONTACT