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

NCT06539624 · Status: RECRUITING · Phase: NA · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2026-02-27

No results posted yet for this study

Summary

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

Conditions

Interventions

GENETIC

EXG110 injection

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.

Sponsors & Collaborators

  • The Children's Hospital of Zhejiang University School of Medicine

    lead OTHER

Principal Investigators

  • Jianhua Mao, PhD · Children's Hospital, Zhejiang University School of Medicine

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
7 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-10-16
Primary Completion
2027-04-09
Completion
2027-04-09

Countries

  • China

Study Locations

More Related Trials

Entities

Diseases

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 NCT06539624 on ClinicalTrials.gov