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New hope for rare brain diseases: long-term drug trial now recruiting

NCT ID NCT07399704

Disease control Sponsor: Azafaros B.V. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests the long-term safety and effectiveness of a daily medication called nizubaglustat in people with two rare genetic diseases that affect the brain and body. About 21 participants who were in a previous study or who have been on a similar drug (miglustat) will take nizubaglustat for up to several years. Researchers will monitor side effects, seizures, and disease markers to see if the drug helps control the condition over time.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 21 people

The number the study aims to enrol. It can still change while the study runs.

Started

Feb 2026

Expected to finish

Aug 2030

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

12 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

GM2 gangliosidosis Niemann-Pick disease type C

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

GM2 gangliosidosis Niemann-Pick type C disease

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    3 sites. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Associação Hospitalar de Prot à Infância Dr. Raul Carneiro

    RECRUITING

    Água Verde, Curitiba, 80250-060, Brazil

  • Hospital de Clinicas de Porto Alegre

    RECRUITING

    Porto Alegre, Rio Grande do Sul, 90035-903, Brazil

  • Instituto Nacional de Saúde da Mulher, da Criança e do Adolescente Fernandes Figueira

    RECRUITING

    Rio de Janeiro, 22250, Brazil

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