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A Phase I/II Clinical Study to Evaluate the Safety and Efficacy of VGN-R08b in Patients With Type III Gaucher's Disease

NCT07603050 · Status: NOT_YET_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2026-05-22

No results posted yet for this study

Summary

A phase I/II clinical study to evaluate the tolerance, safety and efficacy of VGN-R08b intracerebroventricular injection in patients with type III Gaucher's disease

Conditions

  • Gaucher Disease Type 3

Interventions

DRUG

VGN-R08b

6×10\^10 vg/g

DRUG

VGN-R08b

1.2×10\^11 vg/g

DRUG

VGN-R08b

1.8×10\^11 vg/g

Sponsors & Collaborators

  • Shanghai Vitalgen BioPharma Co., Ltd.

    lead INDUSTRY

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-05-14
Primary Completion
2028-05-29
Completion
2032-08-16

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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 NCT07603050 on ClinicalTrials.gov