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An Exploratory Clinical Trial of VGN-R08b in Patients With Type II Gaucher Disease

NCT06272149 · Status: RECRUITING · Phase: EARLY_PHASE1 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2024-02-22

No results posted yet for this study

Summary

This exploratory trial is to prove the tolerability and safety of VGN-R08b to treat infants with type II Gaucher disease.

Conditions

  • Type II Gaucher Disease

Interventions

DRUG

VGN-R08b

VGN-R08b is a kind of Gene therapy with adeno-associated virus (AAV) serotype 9 (AAV9) driven human GBA1 being injected directly into intracerebroventricular.

Sponsors & Collaborators

  • Shanghai Vitalgen BioPharma Co., Ltd.

    collaborator INDUSTRY

  • Xinhua Hospital, Shanghai Jiao Tong University School of Medicine

    lead OTHER

Principal Investigators

  • Zhang Huiwen, Dr. · Xinhua Hospital, Shanghai Jiao Tong University School of Medicine

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
0 Months
Max Age
24 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-03-01
Primary Completion
2025-01-15
Completion
2029-02-28

Countries

  • China

Study Locations

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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 NCT06272149 on ClinicalTrials.gov