NCT05758922View on ClinicalTrials.gov

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

PHASE2Active, not recruitingINTERVENTIONAL
Enrollment

13

Participants

Timeline

Start Date

April 24, 2023

Primary Completion Date

April 19, 2024

Study Completion Date

March 31, 2026

Conditions
GM2 GangliosidosisNiemann-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"

Trial Locations (3)

Unknown

Hospital Pequeno Principe, Curitiba

Hospital de Clinicas de Porto Alegre, Porto Alegre

Instituto Nacional de Saúde da Mulher, da Criança e do Adolescente Fernandes Figueira, Rio de Janeiro

Sponsors

Lead Sponsor

Azafaros A.G.
All Listed Sponsors
lead

Azafaros A.G.

INDUSTRY