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
Running, not enrolling · Phase 3 · Has a placebo group
Conditions studied: Gangliosidoses, GM2, Gangliosidosis, GM1
In brief
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
Key facts
- Study ID
- NCT07082543
- Run by
- Azafaros B.V.
- People needed
- 75
- Starts
- 2025-06-30
- Expected to finish
- 2028-03-06
- Last updated by the study team
- 2026-07-24
Who can join
Age: 4 and older. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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
You may not qualify if…
- 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)
Where it is running
- UCSF Children's Hospital and Research Center at Oakland — Oakland, California, United States
- University of Minnesota Medical School — Minneapolis, Minnesota, United States
- Mayo Clinic Children's Center - PIN — Rochester, Minnesota, United States
- Children's Medical Center Dallas — Dallas, Texas, United States
- Lysosomal Rare Disorders Research and Treatment Center — Fairfax, Virginia, United States
- Hospital Universitario Austral — Ciudad Autónoma Buenos Aires, Buenos Aires, Argentina
- Hospital de Niños de La Santisima Trinidad — Córdoba, Córdoba Province, Argentina
- Women's and Children's Hospital — North Adelaide, South Australia, Australia
- Instituto Fernandes Figueira — Rio de Janeiro, Rio de Janeiro, Brazil
- Hospital de Clinicas de Porto Alegre (HCPA) - PPDS — Porto Alegre, Rio Grande do Sul, Brazil
- M.A.G.I.C. Clinic Ltd. Metabolics and Genetics in Calgary — Calgary, Alberta, Canada
- AP-HP - Hôpital Armand Trousseau — Paris, France
- SphinCS GmbH — Höchheim, Germany
- Amrita Institute of Medical Sciences and Research Centre — Ernākulam, Kerala, India
- All India Institute of Medical Sciences (AIIMS) - New Delhi — New Delhi, National Capital Territory of Delhi, India
- JK Lone Hospital — Jaipur, Rajasthan, India
- Fondazione IRCCS Istituto Neurologico Carlo Besta — Milan, Italy
- ULS de Santo António, EPE - Centro Materno Infantil Norte — Porto, Porto District, Portugal
- ULS de Santa Maria,EPE - Hospital de Santa Maria - PPDS — Lisbon, Portugal
- Hospital Universitario Vall d'Hebron - PPDS — Barcelona, Barcelona, Spain
- Hospital Infantil Universitario Niño Jesus - PIN — Madrid, Madrid, Spain
- Sahlgrenska universitetssjukhuset Östra — Gothenburg, Västra Götaland County, Sweden
- Inselspital - Universitätsspital Bern — Bern, Canton of Bern, Switzerland
- Balcali Hastanesi Saglik Uygulama ve Arastirma Merkezi — Adana, Adana, Turkey (Türkiye)
- Gazi Universitesi Saglik Arastirma ve Uygulama Merkezi — Çankaya, Ankara, Turkey (Türkiye)
Full record on ClinicalTrials.gov
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