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Oral nizubaglustat (AZ-3102) for late‑infantile and juvenile Niemann‑Pick Type C

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 Niemann-Pick Type C Disease and in Late-infantile and Juvenile-onset Forms of GM1 Gangliosidosis or GM2 Gangliosidosis

Phase 3 Interventional Azafaros A.G. · NCT07082725

This 18-month randomized, placebo-controlled study tests whether daily oral nizubaglustat reduces ataxia and improves other symptoms in children and adolescents with late‑infantile or juvenile Niemann‑Pick Type C disease.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment72 (estimated)
Ages4 Years and up
SexAll
SponsorAzafaros A.G. Industry-sponsored
Locations34 sites (Oakland, California and 33 other locations)
Trial IDNCT07082725 on ClinicalTrials.gov

What this trial studies

This Phase 3, double‑blind, randomized, placebo‑controlled multicenter trial enrolls participants aged 4 years and older with confirmed late‑infantile or juvenile NPC and baseline ataxia (SARA score 3–30). Participants receive once‑daily oral nizubaglustat or matching placebo for 18 months, with scheduled visits for clinical outcome measures, safety monitoring, and PK/PD sampling. The primary endpoint is superiority of nizubaglustat versus placebo on ataxic manifestations over 18 months, with secondary endpoints including other neurological features, pharmacokinetics/pharmacodynamics, and tolerability. The protocol is conducted at pediatric specialty centers and follows a master protocol framework referenced to the AZA‑001‑301 master protocol.

Who should consider this trial

Good fit: Ideal candidates are people aged 4 years or older with genetically or biochemically confirmed late‑infantile or juvenile NPC, neurological symptom onset between 2 and 15 years, baseline SARA score between 3 and 30, and who are unable/unwilling or unsatisfactorily treated with miglustat.

Not a fit: Patients with moderate to severe hepatic impairment, another neurologic disease, body weight under 10 kg, or other medical conditions that would confound results are unlikely to benefit from participating in this study.

Why it matters

Potential benefit: If successful, nizubaglustat could slow or reduce ataxia and related neurological decline, improving motor function and daily activities for affected children and adolescents.

How similar studies have performed: Related substrate‑reduction approaches such as miglustat have shown modest benefits in NPC in prior work, but nizubaglustat is a newer agent with limited late‑phase published data so its effectiveness remains unproven.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* Signed informed consent
* Confirmed diagnosis of NPC disease
* Patient is unable or unwilling to take miglustat, or is, in the opinion of the investigator, unsatisfactorily treated with miglustat
* Male and female participants aged 4 years and older at the time of informed consent
* Onset of neurological symptoms from 2 to 15 years
* Disability level at Baseline: Ataxic disturbances with a total SARA score of ≥3 and ≤30 at Baseline
* Female 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 NPC disease 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)
* Current treatment with miglustat, provided the patient has been using the recommended dose for most of the past 12 months AND is, in the opinion of the investigator, satisfactorily treated with miglustat. Any participants receiving miglustat are required to undergo a 1-month washout period before starting study medication

Where this trial is running

Oakland, California and 33 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Niemann-Pick Type C DiseaseNizubaglustat
Last reviewed 2026-06-10 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.