IT and IV Lentiviral Gene Therapy for X-ALD

Sponsor
Shenzhen Geno-Immune Medical Institute
Study ID
NCT03727555
Status
Recruiting
Apply to this trial

Conditions

Eligibility Criteria

Sex
ALL
Age
1 Year - 60 Years
Healthy Volunteers
Not accepted

Interventions

  • Intrathecal and intravenous LV gene therapy — GENETIC
    Direct IT and IV LV gene therapy to deliver high levels of LVs at 1-2×10\^9 multiplicity of infection/ml which carry normal ABCD1 gene

Study Details

This is a Phase I/II clinical trial of gene therapy for treating X-linked adrenoleukodystrophy using a high-safety, high-efficiency, self-inactivating lentiviral vector (LV) TYF-ABCD1 to functionally correct the defective gene. The objectives are to evaluate the safety and efficacy of the intrathecal and intravenous lentiviral gene transfer clinical protocol.

Key Dates

Start date
Aug 31, 2025
Status verified
Sep 2025
Primary completion
Dec 31, 2027
Completion
Dec 31, 2028

Study Design

Enrollment
30 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Lentivirus-mediated delivery of ABCD1 to the CNS and the body
    Intrathecal and intravenous injections with lentiviral TYF-ABCD1 vector carrying the functional gene

Primary Outcome Measure

Safety evaluation of IT and IV injections of lentiviral TYF-ABCD1, determined by number of participants with treatment-related adverse events (AEs), according to scheduled assessments, vital signs, & physical examinations as assessed by CTCAE v4.0. [ Time Frame: Minimum 1 day, maximum 1 year follow up ]

Central Contacts

Related Studies