Evaluate the Safety and Preliminary Efficacy of EXG110 in Subjects With Fabry Disease

Sponsor
The Children's Hospital of Zhejiang University School of Medicine
Study ID
NCT06539624
Status
Recruiting
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Conditions

Eligibility Criteria

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

Interventions

  • EXG110 injection — GENETIC
    EXG110 is a recombinant adeno-associated virus (rAAV) that not only significantly increases plasma AGA activity, but is also highly expressed in target organs such as the heart and kidneys.EXG110 will be administered in a single dose by intravenous infusion.

Study Details

Objective: To explore the safety and tolerability of different doses of EXG110 with Fabre disease

Key Dates

Start date
Oct 16, 2024
Status verified
Feb 2026
Primary completion
Apr 9, 2027
Completion
Apr 9, 2027

Study Design

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

Arms

  • Experimental: Dose escalation-Cohort 1
    Genetic : EXG110
  • Experimental: Dose escalation-Cohort 2
    Genetic : EXG110
  • Experimental: Dose escalation-Cohort 3
    Genetic : EXG110
  • Experimental: Dose escalation-Cohort 4
    Genetic : EXG110

Primary Outcome Measure

Incidence and severity of adverse events [ Time Frame: 52 weeks following EXG110 administration ]

Central Contacts

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