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Clinical Trials in the USA / NCT07082543
Active, not recruiting Phase 3

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

NCT07082543 · tracked via the Priya Life Science USA tracker
Sponsor
Azafaros B.V.
Phase
Phase 3
Started
2025-06-30
Last updated
2026-07-24

Condition(s) studied

Gangliosidoses, GM2Gangliosidosis, GM1

Investigational drug(s) / intervention(s)

AZ-3102Placebo

AZ-3102: Nizubaglustat

Placebo: Placebo

Study 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

Sex
ALL
Min age
4 Years
Max age
Healthy volunteers
No
Inclusion Criteria: * 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 Exclusion Criteria: * A history of medical conditions other than GM1 or GM2 gangliosidosis that, in the opinion of the Principal Investigator, would confound scientific rigor or the interpretation of results * Body weight of \<10 kg * The presence of another neurologic disease * The presence of moderate or severe hepatic impairment * The presence of moderate or severe renal impairment * Platelet count of \<100x10\^9/L * The dose of any anti-epileptic treatment(s) was not stable (required a change in dose within the previous 3 months) and/or a new anti-epileptic treatment (drug or procedure) was prescribed in the month before Baseline * Prior use of an investigational drug within the 3 months before Screening; or prior participation in a clinical study involving gene therapy or stem cell transplantation within 2 years prior to Screening * A positive serum pregnancy test (for women of childbearing potential)

Primary outcome measure(s)

Trial sites (26)

FacilityCityRegionStatus
UCSF Children's Hospital and Research Center at Oakland Oakland California
University of Minnesota Medical School Minneapolis Minnesota
Mayo Clinic Children's Center - PIN Rochester Minnesota
Children's Medical Center Dallas Dallas Texas
Lysosomal Rare Disorders Research and Treatment Center Fairfax Virginia
Hospital Universitario Austral Ciudad Autónoma Buenos Aires Buenos Aires
Hospital de Niños de La Santisima Trinidad Córdoba Córdoba Province
Women's and Children's Hospital North Adelaide South Australia
Instituto Fernandes Figueira Rio de Janeiro Rio de Janeiro
Hospital de Clinicas de Porto Alegre (HCPA) - PPDS Porto Alegre Rio Grande do Sul
M.A.G.I.C. Clinic Ltd. Metabolics and Genetics in Calgary Calgary Alberta
AP-HP - Hôpital Armand Trousseau Paris France
SphinCS GmbH Höchheim Germany
Amrita Institute of Medical Sciences and Research Centre Ernākulam Kerala
All India Institute of Medical Sciences (AIIMS) - New Delhi New Delhi National Capital Territory of Delhi
JK Lone Hospital Jaipur Rajasthan
Fondazione IRCCS Istituto Neurologico Carlo Besta Milan Italy
ULS de Santo António, EPE - Centro Materno Infantil Norte Porto Porto District
ULS de Santa Maria,EPE - Hospital de Santa Maria - PPDS Lisbon Portugal
Hospital Universitario Vall d'Hebron - PPDS Barcelona Barcelona
Hospital Infantil Universitario Niño Jesus - PIN Madrid Madrid
Sahlgrenska universitetssjukhuset Östra Gothenburg Västra Götaland County
Inselspital - Universitätsspital Bern Bern Canton of Bern
Balcali Hastanesi Saglik Uygulama ve Arastirma Merkezi Adana Adana
Gazi Universitesi Saglik Arastirma ve Uygulama Merkezi Çankaya Ankara
Ege Universitesi Tip Fakultesi Bornova İzmir

More Azafaros B.V. trials in the USA

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07082543 on ClinicalTrials.gov ↗ ← All trials in the USA