ClinicalTrials.gov record NCT07054515

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, GM1 Gangliosidosis or GM2 Gangliosidosis

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Aug 22, 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, France, Germany, India, Italy, Mexico, Pakistan, Portugal, Spain, Sweden, 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 Phase 3, randomized, double-blind, placebo-controlled study compares oral nizubaglustat (AZ-3102) with placebo in people with late‑infantile or juvenile forms of Niemann‑Pick Type C disease, GM1 gangliosidosis, or GM2 gangliosidosis. The blinded treatment period is 18 months. Participants are assigned to a disease-specific subprotocol and randomized 2:1 to drug or placebo. The registry lists the Niemann‑Pick type C subprotocol as recruiting and the GM1/GM2 subprotocol as active but no longer recruiting new participants.

What it is studying. The study is testing the safety and efficacy of oral nizubaglustat (AZ-3102) compared with placebo in people with late‑infantile or juvenile-onset Niemann‑Pick Type C disease, GM1 gangliosidosis, or GM2 gangliosidosis.

What the researchers hope to learn. To evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in the listed diseases and to collect disease-specific data under separate subprotocols to inform future research and development.

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 and in late-infantile and juvenile-onset forms of GM1 gangliosidosis or GM2 gangliosidosis

Interventions being studied

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

Drug
  • AZ-3102
  • Placebo

Eligibility

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

Inclusion criteria

  • Male and female participants, aged 4 years and older with a diagnosis of the late-infantile or juvenile form of NPC disease. Detailed inclusion criteria are presented in the NPC disease-specific subprotocol AZA-001-301-NPC (NCT07082725).
  • Male and female participants, aged 4 years and older with a diagnosis of GM1 or GM2 (Tay-Sachs, Sandhoff, or GM2AB variant disease) gangliosidosis of late-infantile/ juvenile onset. Detailed inclusion criteria are presented in the GM1/GM2 gangliosidosis-specific subprotocol AZA-001-301-GMx (NCT07082543).

Exclusion criteria

  • Detailed exclusion criteria are presented in the NPC disease-specific subprotocol AZA-001-301-NPC
  • Detailed exclusion criteria are presented in the GM1/GM2 gangliosidosis-specific subprotocol AZA-001-301-GMx

Study description and what participation involves

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

Detailed description from the registry

This is a Phase 3 randomized, double-blinded, placebo-controlled study that will evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in multiple disease areas using a Master Protocol Research Program. Participants are randomized to different subprotocols based on disease type: Niemann-Pick type C (NPC), GM1 gangliosidosis or GM2 gangliosidosis. Treatment specific procedures will be described in the disease-specific subprotocol. Individual subprotocols may have additional eligibility requirements, safety and efficacy procedures, or endpoints, which will be described in the corresponding subprotocols. For information specific to each individual subprotocol included in this trial, please refer to the corresponding, separate, clinicaltrials.gov records: Niemann-Pick type C disease NCT07082725 (Recruiting) and GM1 gangliosidosis or GM2 gangliosidosis NCT07082543 (Active, not recruiting - No longer looking for participants) .

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 who meet eligibility are randomized (2:1) to take oral nizubaglustat (AZ-3102) dispersible tablets or a matching placebo in a double‑blind schedule for 18 months. They will attend regular clinic visits for safety and efficacy assessments; the exact tests, visit schedule, and any additional procedures vary by the disease-specific subprotocol. Treatment-specific procedures and full eligibility or safety requirements are described in the NPC subprotocol (NCT07082725) and the GM1/GM2 subprotocol (NCT07082543).

How the study is structured. Participants are placed into one of two disease-specific subprotocols (NPC or GM1/GM2) and randomized in a 2:1 ratio to receive oral nizubaglustat or a matching placebo. The study is double-blind (participants, caregivers, investigators, and outcome assessors are masked), parallel-group, and lasts 18 months. Each disease subprotocol describes its own additional procedures, eligibility details, and endpoints.

Questions you could ask

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

  • The registry lists this study under Niemann-Pick Type C Disease; GM1 Gangliosidosis; GM2 Gangliosidosis. Does it apply to my diagnosis?
  • 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, France, Germany, India, Italy, Mexico, Pakistan, Portugal, Spain, Sweden, 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
147 participants (estimated)
Last updated on registry
Sep 24, 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 (39)

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.
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.
blinded
Who does not know which intervention a participant is receiving. It can apply to participants, the people giving the treatment, those assessing results, or those analysing the data.
endpoints
Something the study measures to judge its results, such as a symptom score, a blood test or survival. Outcomes are grouped as primary, secondary and sometimes exploratory.
Active, not recruiting
The study is ongoing but is not enrolling new participants. People already in it may still be receiving treatment or being followed.

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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