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Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease

NCT05758922 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 13

Last updated 2026-05-14

No results posted yet for this study

Summary

This phase 2 is a randomized, double-blind, placebo controlled, 12 weeks study with daily oral administration of AZ-3102 aiming to evaluate the safety and pharmacokinetic (PK) profile in GM2 Gangliosidosis and Niemann-Pick type C disease (NP-C) patients. After approval by the country health authorities, a double-blind extension period was proposed to the patients who complete the 12-week study.

Conditions

  • GM2 Gangliosidosis
  • Niemann-Pick Disease, Type C

Interventions

DRUG

AZ-3102 (Dose 1)

Pharmaceutical form: capsule Route of administration: oral

DRUG

Placebo

Pharmaceutical form: capsule Route of administration: oral

DRUG

AZ-3102 (Dose 2)

Pharmaceutical form: capsule Route of administration: oral

Sponsors & Collaborators

  • Azafaros A.G.

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
12 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-04-24
Primary Completion
2024-04-19
Completion
2026-05-05
FDA Drug
Yes

Countries

  • Brazil

Study Locations

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Entities

Drugs

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 NCT05758922 on ClinicalTrials.gov