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Study to Evaluate the Efficacy and Safety of Nizubaglustat (AZ-3102) in Patients With Gaucher Disease Type 3 (GD3)

NCT07715084 · Status: NOT_YET_RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 20

Last updated 2026-07-20

No results posted yet for this study

Summary

A 96-week, open-label, Phase 2 study to evaluate the efficacy and safety of Nizubaglustat (AZ-3102) in patients with Gaucher disease type 3 (GD3).

Conditions

  • Gaucher Disease Type 3

Interventions

DRUG

AZ-3102

Daily oral intake of AZ-3102 orodispersible tablets

Sponsors & Collaborators

  • Azafaros B.V.

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
4 Years
Max Age
30 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-09-28
Primary Completion
2028-01-31
Completion
2029-07-31

Countries

  • India
  • Pakistan
  • Turkey (Türkiye)

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 NCT07715084 on ClinicalTrials.gov