The Safety and Efficacy of Intravenous EXG110 in Patients With Fabry Disease

Sponsor
Guangzhou Jiayin Biotech Ltd
Study ID
NCT06819514
Phase
PHASE1/PHASE2
Status
Not Yet Recruiting

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Conditions

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • EXG110 Injection — DRUG
    EXG110 Injection is gene therapy for Fabry Disease , uses a proprietary AAV capsid with improved liver and muscle specificity.

Study Details

A phase 1/2, multicenter, open-label, Dose-escalation study to evaluate the safety and efficacy of intravenous EXG110 in patients with Fabry disease

Key Dates

Start date
Mar 1, 2025
Status verified
Feb 2025
Primary completion
Mar 15, 2028
Completion
Mar 15, 2028

Study Design

Enrollment
16 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Low dose arm
    EXG110 injection, use once by intravenous
  • Experimental: High dose arm
    EXG110 injection, use once by intravenous

Primary Outcome Measure

Phase 1: To evaluate the safety and tolerability of EXG110 following a single intravenous infusion [ Time Frame: 52 weeks ]

Central Contacts

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