NCT05758922

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

Randomized, Double Blind, Placebo Controlled, Multicenter, 12 Weeks Phase 2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients with GM2 Gangliosidosis or Niemann-Pick Type C Disease

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 13 (actual)
• Sponsor: Azafaros A.G. · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

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

Type	Name	Description
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

Timeline

• Start date: 2023-04-24
• Primary completion: 2024-04-19
• Completion: 2026-03-01
• First posted: 2023-03-08
• Last updated: 2025-03-20

Locations

3 sites across 1 country: Brazil

Regulatory

• FDA-regulated drug study