Last updated 25 days ago
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
75 patients around the world
Available in Brazil, Argentina, United States
Please see NCT #07054515 for information on the AZA-001-301 Master Protocol
PRIMARY OBJECTIVE
The primary objective of this study is to demonstrate superior efficacy on ataxic
manifestations with oral nizubaglustat dosing compared with placebo when administered
over 18 months in participants with late-infantile and juvenile forms of GM1/GM2
gangliosidosis
SECONDARY OBJECTIVES
I. To assess additional efficacy in ataxic and non-ataxic manifestations comparing
nizubaglustat dosing with placebo when administered over 18 months in participants with
late-infantile and juvenile forms of GM1/GM2 gangliosidosis
II. To assess the pharmacokinetic (PK) properties of nizubaglustat after administration
of the first dose (Visit 1) and at steady state after multiple once daily doses
III. To assess the pharmacodynamic (PD) effects of nizubaglustat
IV. To assess the safety and tolerability of daily oral nizubaglustat dosing compared
with placebo, when administered over 18 months in participants with late-infantile and
juvenile forms of GM1/GM2 gangliosidosis
Azafaros A.G.
5Research sites
75Patients around the world
This study is for people with
Lysosomal Storage Disease
GM1 Gangliosidosis
GM2 Gangliosidosis
Requirements for the patient
From 4 Years
All Gender
Medical requirements
- 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.
- 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 and/or a new anti-epileptic treatment 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.
Sites
Hospital de Niños de la Santísima Trinidad - Córdoba
Hospital Universitario Austral - Pilar
Hospital Pequeno Príncipe
Instituto Fernandes Figueira
Hospital de Clínicas de Porto Alegre
SponsorAzafaros A.G.
Study typeInterventional
Conditions
GM1 GangliosidosisGM2 Gangliosidosis
Requirements
From 4 YearsAll Gender
Unique study IDclinicaltrials.gov
NCT07082543
