ClinicalTrials.gov record NCT07082725

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC)

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Jul 27, 2026

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

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 3A later-stage study, usually with more participants, designed to give stronger evidence about how well a treatment works and how safe it is. It often compares the treatment with standard care or a placebo, and results may support an application for approval.
Age4 years old and older
SexAll
LocationArgentina, Australia, Brazil, Canada, Germany, India, Italy, Mexico, Pakistan, Portugal, Spain, Switzerland, Turkey, United Kingdom, United States
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This is an 18-month, randomized, double-blind, placebo-controlled Phase 3 study of once-daily oral nizubaglustat in people with late‑infantile or juvenile‑onset Niemann‑Pick Type C disease. Participants are randomly assigned to nizubaglustat or matching placebo and monitored with neurological and functional assessments. The study collects safety data, drug levels, and measures of neurologic function over the treatment period.

What it is studying. The study tests whether daily oral nizubaglustat, compared with placebo, affects ataxic manifestations over 18 months in people with late‑infantile or juvenile NPC. It also evaluates other neurologic and functional outcomes, pharmacokinetics and pharmacodynamics, and the safety and tolerability of the drug.

What the researchers hope to learn. Primary scientific goal: compare nizubaglustat versus placebo on ataxic manifestations (measured by SARA) over 18 months. Secondary goals: assess additional ataxic and non‑ataxic clinical outcomes, seizure measures, pharmacokinetics and pharmacodynamics, biomarker changes, and safety/tolerability.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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 Niemann-Pick type C disease

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • Nizubaglustat
  • Placebo

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

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 NPC disease 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 NPC disease 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 NPC disease

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants are randomly assigned, in double‑blind fashion, to take once‑daily oral nizubaglustat or matching placebo for 18 months. They will attend regular clinic visits for neurologic and functional tests (including SARA and other scales at baseline and at months 6, 12, and 18), complete questionnaires and goal assessments, keep a seizure diary, and provide blood samples for safety and drug‑level (PK/PD) testing (PK sampling is planned at baseline and at months 1, 18, and 21). Women of childbearing potential will have pregnancy testing and must follow contraceptive guidance; people on miglustat must complete a 1‑month washout before starting study medication.

How the study is structured. Participants are randomly assigned to one of two parallel groups: once-daily oral nizubaglustat dispersible tablets or matching placebo. The trial is double-blind (quadruply masked), so participants, caregivers, investigators, and outcome assessors do not know the assignment. Study visits include baseline and periodic neurologic and functional assessments (including SARA ataxia scoring), safety testing, and blood sampling for drug levels and biomarkers over the 18-month treatment period.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • Some participants receive a placebo. How is that decided, and what happens for placebo-group participants when the study ends?
  • The listed sites are in Argentina, Australia, Brazil, Canada, Germany, India, Italy, Mexico, Pakistan, Portugal, Spain, Switzerland, Turkey, United Kingdom, United States. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on Niemann-Pick disease type C

Clinical trials15 trials listed for Niemann-Pick disease type CEvery trial the registry lists for Niemann-Pick disease type C, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in Niemann-Pick disease type C researchLatest monthly update: September 2026. Includes: Gene change range. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Jun 30, 2025
Primary completion
May 14, 2028 (estimated)
Study completion
Jun 14, 2028 (estimated)
Target enrollment
72 participants (estimated)
Last updated on registry
Sep 28, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Azafaros B.V.

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (34)

Terms used on this page · explanations by MedicalUpdates.ai

double-blind
Two types of people involved in the study do not know which intervention a participant is receiving. The ClinicalTrials.gov record specifies who is masked, for example participants, care providers, investigators or outcome assessors.
randomized
Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
placebo-controlled
A study design in which one group receives a placebo so that the study treatment's effects can be compared with no active treatment.
informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
Baseline
Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
childbearing potential
Studies often ask participants who could become pregnant, or could father a child, to use contraception during the study and for a period afterwards, because the effect of the treatment on a pregnancy is unknown.
Principal Investigator
The doctor or scientist responsible for running the study at a site or overall.
investigational drug
A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
before Screening
The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
washout period
A period during which a participant stops a previous treatment before starting the study treatment, so the earlier treatment does not affect the results.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
PRIMARY OBJECTIVE
The main result the study is designed to measure and report on. Other results, called secondary outcomes, are also collected.
placebo
A treatment that looks like the study treatment but contains no active ingredient. Some participants receive it instead of the study treatment, usually without knowing which they got.
pharmacokinetic
How the body absorbs, distributes, breaks down and removes a drug. It is studied by measuring drug levels or related substances at set times after a dose.
pharmacodynamic
What a drug does to the body, for example how it changes a blood marker or a symptom, and how that relates to the dose.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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