18-month Double-blind, Randomized, Placebo-controlled, Multicenter, Phase 3 Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease and in Late-infantile and Juvenile-onset Forms of GM1 Gangliosidosis or GM2 Gangliosidosis

Status: Recruiting
Location: See all (31) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
SUMMARY

An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of GM1 gangliosidosis or GM2 gangliosidosis

Eligibility
Participation Requirements
Sex: All
Minimum Age: 4
Healthy Volunteers: f
View:

• Confirmed GM1 gangliosidosis or Tay-Sachs, Sandhoff, or GM2AB variant

• Male and female participants aged 4 years and older at the time of informed consent

• Onset of neurological symptoms from 1 to 10 years

• Disability level at Baseline: Ataxic disturbances with a total SARA score of ≥3 and ≤30 at Baseline

• Females of childbearing potential who are sexually active willing to follow the contraceptive guidance

• Male participants with a female partner of childbearing potential willing to follow the contraceptive guidance

Locations
United States
California
Children's Hospital and Research Center at Oakland
NOT_YET_RECRUITING
Oakland
Minnesota
Mayo Clinic Children's Center - PIN
NOT_YET_RECRUITING
Rochester
Virginia
Lysosomal Rare Disorders Research and Treatment Center
NOT_YET_RECRUITING
Fairfax
Other Locations
Argentina
Hospital Universitario Austral
NOT_YET_RECRUITING
Ciudad Autónoma Buenos Aires
Hospital de Niños de La Santisima Trinidad
NOT_YET_RECRUITING
Córdoba
Australia
Women's and Children's Hospital
NOT_YET_RECRUITING
North Adelaide
Royal Children's Hospital Melbourne - PIN
NOT_YET_RECRUITING
Parkville
Brazil
Hospital Pequeno Principe
NOT_YET_RECRUITING
Curitiba
Hospital de Clinicas de Porto Alegre (HCPA) - PPDS
NOT_YET_RECRUITING
Porto Alegre
Instituto Fernandes Figueira
NOT_YET_RECRUITING
Rio De Janeiro
Canada
M.A.G.I.C. Clinic Ltd. Metabolics and Genetics in Calgary
NOT_YET_RECRUITING
Calgary
University of Alberta Medical Genetics Clinic
NOT_YET_RECRUITING
Edmonton
Centre Hospitalier de l'Universite de Montreal-1000 rue Saint-Denis
NOT_YET_RECRUITING
Montreal
France
AP-HM- Hôpital de La Timone
NOT_YET_RECRUITING
Marseille
AP-HP - Hôpital Armand Trousseau
NOT_YET_RECRUITING
Paris
India
Amrita Institute of Medical Sciences and Research Centre
NOT_YET_RECRUITING
Ernākulam
JK Lone Hospital
NOT_YET_RECRUITING
Jaipur
Seth G S Medical College and K E M Hospital
NOT_YET_RECRUITING
Mumbai
All India Institute of Medical Sciences (AIIMS) - New Delhi
NOT_YET_RECRUITING
New Delhi
Italy
Fondazione IRCCS Istituto Neurologico Carlo Besta
NOT_YET_RECRUITING
Milan
Portugal
ULS de Santa Maria,EPE - Hospital de Santa Maria - PPDS
NOT_YET_RECRUITING
Lisbon
ULS de Santo António, EPE - Centro Materno Infantil Norte
RECRUITING
Porto
Spain
Hospital Universitario Vall d'Hebron - PPDS
NOT_YET_RECRUITING
Barcelona
Hospital Infantil Universitario Niño Jesus - PIN
RECRUITING
Madrid
Sweden
Sahlgrenska universitetssjukhuset Östra
RECRUITING
Gothenburg
Switzerland
Inselspital - Universitätsspital Bern
NOT_YET_RECRUITING
Bern
Turkey
Balcali Hastanesi Saglik Uygulama ve Arastirma Merkezi
NOT_YET_RECRUITING
Adana
Ege Universitesi Tip Fakultesi
NOT_YET_RECRUITING
Bornova
Gazi Universitesi Saglik Arastirma ve Uygulama Merkezi
NOT_YET_RECRUITING
Çankaya
United Kingdom
Great Ormond Street Hospital NHSFT
NOT_YET_RECRUITING
London
University College London Hospitals (UCLH)
NOT_YET_RECRUITING
London
Contact Information
Primary
Patient Advocacy Representative
info@azafaros.com
Please reach out by email
Backup
Contact for Healthcare Professionals
medinfo@azafaros.com
Please reach out by email
Time Frame
Start Date: 2025-06-30
Estimated Completion Date: 2027-11-04
Participants
Target number of participants: 75
Treatments
Experimental: Nizubaglustat
Once daily oral dispersible tablets
Placebo_comparator: Placebo
Related Therapeutic Areas
GM1 Gangliosidosis
Tay-Sachs Disease
Niemann-Pick Disease
Gangliosidosis
Sponsors
Leads: Azafaros A.G.

This content was sourced from clinicaltrials.gov

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