A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis

PHASE3RecruitingINTERVENTIONAL
Enrollment

147

Participants

Timeline

Start Date

June 30, 2025

Primary Completion Date

November 4, 2027

Study Completion Date

November 4, 2027

Conditions
Niemann-Pick Type C DiseaseGM1 GangliosidosisGM2 Gangliosidosis
Interventions
DRUG

AZ-3102

Oral dispersible tablets

DRUG

Placebo

A matching placebo will be administered in the same regimen as the intervention

Trial Locations (32)

1250

NOT_YET_RECRUITING

Balcali Hastanesi Saglik Uygulama ve Arastirma Merkezi, Adana

3010

NOT_YET_RECRUITING

Inselspital - Universitätsspital Bern, Bern

3050

NOT_YET_RECRUITING

Royal Melbourne Hospital, Parkville

3052

NOT_YET_RECRUITING

Royal Children's Hospital Melbourne - PIN, Parkville

5006

NOT_YET_RECRUITING

Women's and Children's Hospital, North Adelaide

6500

NOT_YET_RECRUITING

Gazi Universitesi Saglik Arastirma ve Uygulama Merkezi, Çankaya

8035

RECRUITING

Hospital Universitario Vall d'Hebron - PPDS, Barcelona

13005

NOT_YET_RECRUITING

AP-HM- Hôpital de La Timone, Marseille

20133

RECRUITING

Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan

28009

RECRUITING

Hospital Infantil Universitario Niño Jesus - PIN, Madrid

35100

NOT_YET_RECRUITING

Ege Universitesi Tip Fakultesi, Bornova

55905

NOT_YET_RECRUITING

Mayo Clinic Childrens Center - PIN, Rochester

75012

NOT_YET_RECRUITING

AP-HP - Hôpital Armand Trousseau, Paris

94609

NOT_YET_RECRUITING

Children's Hospital and Research Center at Oakland, Oakland

110029

NOT_YET_RECRUITING

All India Institute of Medical Sciences (AIIMS) - New Delhi, New Delhi

302004

NOT_YET_RECRUITING

JK Lone Hospital, Jaipur

400012

NOT_YET_RECRUITING

Seth G S Medical College and K E M Hospital, Mumbai

682041

NOT_YET_RECRUITING

Amrita Institute of Medical Sciences and Research Centre, Ernākulam

22030-7404

NOT_YET_RECRUITING

Lysosomal Rare Disorders Research and Treatment Center, Fairfax

B1629AHJ

NOT_YET_RECRUITING

Hospital Universitario Austral, Ciudad Autónoma Buenos Aires

X5004 ASL

NOT_YET_RECRUITING

Hospital de Niños de La Santisima Trinidad, Córdoba

22250-020

NOT_YET_RECRUITING

Instituto Fernandes Figueira, Rio de Janeiro

90560-030

NOT_YET_RECRUITING

Hospital de Clinicas de Porto Alegre (HCPA) - PPDS, Porto Alegre

80250-060

NOT_YET_RECRUITING

Hospital Pequeno Príncipe, Curitiba

T3B 6A8

NOT_YET_RECRUITING

M.A.G.I.C. Clinic Ltd. Metabolics and Genetics in Calgary, Calgary

T6G 2B7

NOT_YET_RECRUITING

University of Alberta Medical Genetics Clinic, Edmonton

H2X 0C1

NOT_YET_RECRUITING

Centre Hospitalier de l'Universite de Montreal-1000 rue Saint-Denis, Montreal

4050-651

RECRUITING

ULS de Santo António, EPE - Centro Materno Infantil Norte, Porto

1649-035

RECRUITING

ULS de Santa Maria,EPE - Hospital de Santa Maria - PPDS, Lisbon

416 50

RECRUITING

Sahlgrenska universitetssjukhuset Östra, Gothenburg

WC1N 3JH

NOT_YET_RECRUITING

Great Ormond Street Hospital, London

WC1N 3BG

NOT_YET_RECRUITING

University College London Hospitals (UCLH), London

Sponsors

Lead Sponsor

All Listed Sponsors
lead

Azafaros A.G.

INDUSTRY