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New pill hopes to tame rare childhood brain diseases

NCT ID NCT07054515

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Sep 17, 2026 · Updated 16 times

Summary

This Phase 3 trial tests an oral drug called nizubaglustat (AZ-3102) in people aged 4 and older with Niemann-Pick type C disease, GM1 gangliosidosis, or GM2 gangliosidosis. The study lasts 18 months and compares the drug to a placebo to see if it is safe and can slow disease progression. About 147 participants will be enrolled across multiple centers.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
nizubaglustat (AZ-3102), an oral drug
What this could lead to
If successful, this could provide a treatment option to slow or manage these rare, progressive neurological diseases.
What could go wrong
This is an early-stage Phase 3 trial with only 147 participants, so results may not apply to all patients. The drug may cause side effects or fail to show benefit.

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 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 147 people

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

Started

Jun 2025

Expected to finish

Jun 2028

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

4 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: * 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

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Conditions

The condition(s) this trial relates to.

GM1 gangliosidosis GM2 gangliosidosis Niemann-Pick disease type C

As listed by the trial registrant

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

GM1 gangliosidosis 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 study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    36 sites in 16 countries. The list below names each one and where it is.

  3. 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 ↗

  4. 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

Study contacts

  • Contact

    Email: •••••@•••••

  • Contact

    Email: •••••@•••••

Locations

  • AP-HP - Hôpital Armand Trousseau

    RECRUITING

    Paris, 75012, France

  • All India Institute of Medical Sciences (AIIMS) - New Delhi

    RECRUITING

    New Delhi, National Capital Territory of Delhi, 110029, India

  • Amrita Institute of Medical Sciences and Research Centre

    RECRUITING

    Ernākulam, Kerala, 682041, India

  • Centenario Hospital Miguel Hidalgo

    NOT_YET_RECRUITING

    Aguascalientes, 20000, Mexico

  • Centre Hospitalier de l'Universite de Montreal-1000 rue Saint-Denis

    NOT_YET_RECRUITING

    Montreal, Quebec, H2X 0C1, Canada

  • Children's Medical Center Dallas

    RECRUITING

    Dallas, Texas, 75235, United States

  • Christian Medical College and Hospital

    RECRUITING

    Vellore, Tamil Nadu, 632004, India

  • Cukurova Universitesi Balcali Hastanesi Saglik Uygulama ve Arastirma Merkezi

    RECRUITING

    Adana, Adana, 1250, Turkey (Türkiye)

  • Ege Universitesi Tip Fakultesi

    RECRUITING

    Bornova, İzmir, 35100, Turkey (Türkiye)

  • Fondazione IRCCS Istituto Neurologico Carlo Besta

    RECRUITING

    Milan, 20133, Italy

  • Gazi Universitesi Saglik Arastirma ve Uygulama Merkezi

    RECRUITING

    Çankaya, Ankara, 6500, Turkey (Türkiye)

  • Great Ormond Street Hospital

    RECRUITING

    London, London, WC1N 3JH, United Kingdom

  • Hospital Infantil Universitario Niño Jesus - PIN

    RECRUITING

    Madrid, Madrid, 28009, Spain

  • Hospital Pequeno Príncipe

    NOT_YET_RECRUITING

    Curitiba, 80250-060, Brazil

  • Hospital Universitario Austral

    ACTIVE_NOT_RECRUITING

    Ciudad Autónoma Buenos Aires, Buenos Aires, B1629AHJ, Argentina

  • Hospital Universitario Dr. Jose Eleuterio González

    NOT_YET_RECRUITING

    Monterrey, Nuevo León, 64460, Mexico

  • Hospital Universitario Vall d'Hebron - PPDS

    RECRUITING

    Barcelona, Barcelona, 8035, Spain

  • Hospital de Clinicas de Porto Alegre (HCPA) - PPDS

    RECRUITING

    Porto Alegre, Rio Grande do Sul, 90560-030, Brazil

  • Hospital de Niños de La Santisima Trinidad

    RECRUITING

    Córdoba, Córdoba Province, X5004 ASL, Argentina

  • Inselspital - Universitätsspital Bern

    RECRUITING

    Bern, Canton of Bern, 3010, Switzerland

  • Instituto Fernandes Figueira

    RECRUITING

    Rio de Janeiro, Rio de Janeiro, 22250-020, Brazil

  • JK Lone Hospital

    RECRUITING

    Jaipur, Rajasthan, 302004, India

  • Lysosomal Rare Disorders Research and Treatment Center

    RECRUITING

    Fairfax, Virginia, 22030-7404, United States

  • M.A.G.I.C. Clinic Ltd. Metabolics and Genetics in Calgary

    RECRUITING

    Calgary, Alberta, T3B 6A8, Canada

  • Mayo Clinic Childrens Center - PIN

    RECRUITING

    Rochester, Minnesota, 55905, United States

    Contact Email: •••••@•••••

  • Royal Children's Hospital Melbourne - PIN

    RECRUITING

    Parkville, Victoria, 3052, Australia

    Contact Email: •••••@•••••

  • Royal Manchester Children's Hospital

    NOT_YET_RECRUITING

    Manchester, M13 9WL, United Kingdom

  • Royal Melbourne Hospital

    RECRUITING

    Parkville, Victoria, 3050, Australia

    Contact Email: •••••@•••••

  • Sahlgrenska universitetssjukhuset Östra

    ACTIVE_NOT_RECRUITING

    Gothenburg, Västra Götaland County, 416 50, Sweden

  • SphinCS GmbH

    RECRUITING

    Höchheim, 65239, Germany

  • The Children's Hospital & the University of Child Health Lahore

    NOT_YET_RECRUITING

    Lahore, Punjab Province, Pakistan

  • UCSF Children's Hospital and Research Center at Oakland

    RECRUITING

    Oakland, California, 94609, United States

  • ULS de Santa Maria,EPE - Hospital de Santa Maria - PPDS

    RECRUITING

    Lisbon, 1649-035, Portugal

  • ULS de Santo António, EPE - Centro Materno Infantil Norte

    RECRUITING

    Porto, Porto District, 4050-651, Portugal

  • University College London Hospitals (UCLH)

    NOT_YET_RECRUITING

    London, Middlesex, WC1N 3BG, United Kingdom

  • University of Alberta Medical Genetics Clinic

    NOT_YET_RECRUITING

    Edmonton, Alberta, T6G 2B7, Canada

  • University of Minnesota Medical School

    NOT_YET_RECRUITING

    Minneapolis, Minnesota, 55455, United States

  • Women's and Children's Hospital

    RECRUITING

    North Adelaide, South Australia, 5006, Australia

More trials for these conditions

Other studies related to the condition(s) this trial covers.