Clinical Trials in Italy / 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 Italy tracker
Condition(s) studied
Gangliosidoses, GM2Gangliosidosis, GM1
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
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)
- Change from baseline in total Scale for the Assessment and Rating of Ataxia (SARA) score — Baseline to month 18
Total SARA comprises eight categories with a cumulative score ranging from 0 (no ataxia) to 40 (most severe ataxia) - Change from baseline in functional SARA score — Baseline to month 18
Functional SARA uses an abbreviated scale that scores 0 to 16, with higher scores indicating more severe impairment
Trial sites (27)
| Facility | City | Region | Status |
|---|---|---|---|
| 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 | |
| AP-HP - Hôpital de la Pitié Salpétrière | 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 Italy
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 Italy