A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease

Recruiting Phase 2 Interventional Study
GM2 Gangliosidosis Niemann-Pick Type C Disease
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
12 and older
Sex
Any
Trial phase
Phase 2
Study type
Interventional
Purpose
Treatment
Participants needed
21 (estimated)
Sponsor
Azafaros B.V. · Industry
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About This Trial
This open-label study aims to gather long-term safety, tolerability, PK, biomarker, and clinical efficacy data relating to daily administration of Nizubaglustat in participants previously enrolled in the Phase 2 RAINBOW study (Cohort 1). In addition, the study aims to assess safety, clinical, and biochemical impact of transitioning NPC disease patients to Nizubaglustat after prior treatment with s…
Trial Locations
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Eligibility Criteria
Inclusion Criteria: * Cohort 1 (NPC and GM2 patients): * Have been randomized into Phase 2 Study AZA-001-5A2-01. OR Cohort 2 (NPC patients): * Be male or female aged ≥12 years * Have a genetically-confirmed diagnosis of NPC disease * Have received full-dose Miglustat treatment for at least 12…
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