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Promising new pill for rare childhood brain diseases enters final testing

NCT ID NCT07082543

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 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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jul 24, 2026 · Updated 6 times

Summary

This study tests an oral drug called nizubaglustat in children and teens with rare genetic disorders (GM1, GM2 gangliosidosis) that damage the brain and nerves. The goal is to see if the drug can slow disease progression and improve movement and coordination. About 75 participants will receive either the drug or a placebo for 18 months.

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 75 people

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

Started

Jun 2025

Expected to finish

Mar 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: * Confirmed GM1 gangliosidosis or Tay-Sachs, Sandhoff, or GM2AB variant * Male and female participants aged 4 years and older at the time of informed consent * Onset of neurological symptoms from 1 to 10 years * Disability level at Baseline: Ataxic disturbances with a total SARA score of ≥3 and ≤30 at Baseline * Females 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 GM1 or GM2 gangliosidosis 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)

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Conditions

The condition(s) this trial relates to.

GM1 gangliosidosis GM2 gangliosidosis Sandhoff disease Tay-Sachs disease

As listed by the trial registrant

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

Gangliosidoses, GM2 Gangliosidosis, GM1

Contacts and locations

Locations

  • AP-HP - Hôpital Armand Trousseau

    Paris, 75012, France

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

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

  • Amrita Institute of Medical Sciences and Research Centre

    Ernākulam, Kerala, 682041, India

  • Balcali Hastanesi Saglik Uygulama ve Arastirma Merkezi

    Adana, Adana, 1250, Turkey (Türkiye)

  • Children's Medical Center Dallas

    Dallas, Texas, 75235, United States

  • Ege Universitesi Tip Fakultesi

    Bornova, İzmir, 35100, Turkey (Türkiye)

  • Fondazione IRCCS Istituto Neurologico Carlo Besta

    Milan, 20133, Italy

  • Gazi Universitesi Saglik Arastirma ve Uygulama Merkezi

    Çankaya, Ankara, 6500, Turkey (Türkiye)

  • Hospital Infantil Universitario Niño Jesus - PIN

    Madrid, Madrid, 28009, Spain

  • Hospital Universitario Austral

    Ciudad Autónoma Buenos Aires, Buenos Aires, B1629AHJ, Argentina

  • Hospital Universitario Vall d'Hebron - PPDS

    Barcelona, Barcelona, 8035, Spain

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

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

  • Hospital de Niños de La Santisima Trinidad

    Córdoba, Córdoba Province, X5004 ASL, Argentina

  • Inselspital - Universitätsspital Bern

    Bern, Canton of Bern, 3010, Switzerland

  • Instituto Fernandes Figueira

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

  • JK Lone Hospital

    Jaipur, Rajasthan, 302004, India

  • Lysosomal Rare Disorders Research and Treatment Center

    Fairfax, Virginia, 22030-7404, United States

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

    Calgary, Alberta, T3B 6A8, Canada

  • Mayo Clinic Children's Center - PIN

    Rochester, Minnesota, 55905, United States

  • Sahlgrenska universitetssjukhuset Östra

    Gothenburg, Västra Götaland County, 416 50, Sweden

  • SphinCS GmbH

    Höchheim, 65239, Germany

  • UCSF Children's Hospital and Research Center at Oakland

    Oakland, California, 94609, United States

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

    Lisbon, 1649-035, Portugal

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

    Porto, Porto District, 4050-651, Portugal

  • University of Minnesota Medical School

    Minneapolis, Minnesota, 55455, United States

  • Women's and Children's Hospital

    North Adelaide, South Australia, 5006, Australia

More trials for these conditions

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