ClinicalTrials.gov record NCT07399704

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

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

Open-label Study to Evaluate the Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease, With or Without Previous Administration of Miglustat

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 2A study that tests whether a treatment has the intended effect and continues to check safety, usually in a larger group than phase 1.
Age12 years old and older
SexAll
LocationBrazil
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 open-label Phase 2 study follows people with GM2 gangliosidosis or Niemann-Pick Type C (NPC) disease who receive daily oral Nizubaglustat (AZ-3102). It includes participants continuing from a prior Phase 2 RAINBOW study and a group of NPC patients who wish to stop miglustat and transition to nizubaglustat. The study will collect safety, drug-level, seizure, ECG, biomarker, and clinical data over an average follow-up of about four years.

What it is studying. The study is gathering long-term information on daily Nizubaglustat for people with GM2 gangliosidosis or NPC disease: whether the drug is tolerated, what plasma drug levels (pharmacokinetics) look like over time, and how clinical measures and laboratory biomarkers change. It also aims to observe the safety and clinical/biochemical effects when some NPC patients switch from stable, full-dose miglustat to Nizubaglustat.

What the researchers hope to learn. To collect long-term safety and tolerability data for Nizubaglustat; to measure pharmacokinetics (drug concentrations over time); to track seizures and ECG changes; and to measure effects on disease-related biomarkers (for example GlcCer, NfL, GM2-related markers, and PPCS) and clinical outcomes. The study aims to inform understanding of Nizubaglustat's safety profile and biological effects in GM2 and NPC disease.

What this study is about

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

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 stable, full-dose Miglustat (Cohort 2).

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

Eligibility

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

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 months and experienced disease stabilization or worsening with treatment over the 2 previous clinic visits. Patients experiencing clinical improvement with Miglustat over the preceding 3 months should not be considered for this study.
  • Wish to change treatment to Nizubaglustat for their NPC disease.
  • Participants from Phase 2 Study AZA-001-5A2-01 (RAINBOW) who transitioned to Miglustat may be eligible for Cohort 2 if they meet all other criteria.
  • Participation is supported and deemed beneficial by the Principal Investigator. Be willing and able to be evaluated for all protocol assessments. The participant, parent, and/or legal guardian can read, understand, and sign the informed consent form. Where appropriate, assent will also be sought for participants who have not reached the age of majority.

Exclusion criteria

  • A positive serum pregnancy test (only tested for women of childbearing potential).
  • Female planning to breastfeed during the study.
  • Any medical event/condition that prevents participation in the study based on the judgment of the Principal Investigator.
  • Participation in another interventional or non-interventional study or early access program.

Study description and what participation involves

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

Detailed description from the registry

This is a multicenter, open-label study to assess the safety, tolerability, PK, PD, and efficacy of Nizubaglustat in male or female patients with late-infantile or juvenile onset GM2 gangliosidosis or NPC disease in two cohorts:

  • Cohort 1: Patients who previously took part in Phase 2 Study AZA-001-5A2-01 (RAINBOW) and wish to continue in this open-label study
  • Cohort 2: Approximately 10 patients with NPC disease, aged ≥12 years who received full-dose Miglustat for more than 12 months, have stable or worsening disease over the 2 previous clinic visits, and who wish to stop Miglustat treatment and transition to Nizubaglustat.
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 take daily oral Nizubaglustat (AZ-3102) dispersible tablets and attend regular clinic visits over the study (safety and outcomes are followed for an average of about 4 years). Visits include safety monitoring and reporting of adverse events, electrocardiograms (ECGs), maintaining a seizure diary if applicable, and blood draws for drug levels (pharmacokinetics) and biomarker testing. Cohort 2 NPC participants who enroll will stop Miglustat and transition to Nizubaglustat. Participants must be able to complete the required assessments and provide informed consent/assent.

How the study is structured. Interventional, open-label Phase 2 study with a single treatment group (no masking). All participants receive daily oral Nizubaglustat (AZ-3102) as dispersible tablets and are followed with regular visits for safety checks (including ECGs and seizure diaries), drug-level tests, and biomarker and clinical assessments. The study combines two cohorts (prior RAINBOW participants who continue, and NPC patients transitioning from miglustat) and follows participants for several years (primary outcomes report an average of about four years).

Questions you could ask

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

  • The listed sites are in Brazil. 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
Feb 4, 2026
Primary completion
Apr 15, 2030 (estimated)
Study completion
Aug 7, 2030 (estimated)
Target enrollment
21 participants (estimated)
Last updated on registry
Jun 18, 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.

Participating sites (3)

Terms used on this page · explanations by MedicalUpdates.ai

open-label
No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
PK
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.
biomarker
Something measurable in the body, such as a blood test result or an imaging finding, that indicates how a condition or treatment is behaving.
Cohort
A group of people who share a characteristic, such as a diagnosis, and are studied together. 'Prospective' means they are followed forward from enrolment; 'retrospective' means existing records are reviewed.
prior treatment
Treatment a person has already had for the condition. A 'line of therapy' is one course of treatment; studies often require, or limit, the number of prior lines.
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.
Principal Investigator
The doctor or scientist responsible for running the study at a site or overall.
legal guardian
A person who is legally allowed to make decisions, including consent to research, on behalf of someone who cannot consent for themselves.
informed consent form
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.
assent
A child's or dependent adult's agreement to take part, given alongside the formal consent of a parent or legal representative.
women of 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.
non-interventional
A study in which researchers do not assign participants to a treatment or other intervention. They observe or measure what happens, which may include study visits, tests, questionnaires or a review of medical records.
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.

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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