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New pill hopes to slow rare brain disease in kids

NCT ID NCT07082725

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 18, 2026 · Updated 14 times

Summary

This study tests an oral drug called nizubaglustat in children and teens aged 4 and older with Niemann-Pick type C disease, a rare genetic disorder that affects movement and thinking. Participants will take the drug or a placebo for 18 months to see if it slows the disease and improves coordination. The trial is for those who cannot take or are not helped by the current standard treatment.

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 72 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: * Signed informed consent * Confirmed diagnosis of NPC disease * Patient is unable or unwilling to take miglustat, or is, in the opinion of the investigator, unsatisfactorily treated with miglustat * Male and female participants aged 4 years and older at the time of informed consent * Onset of neurological symptoms from 2 to 15 years * Disability level at Baseline: Ataxic disturbances with a total SARA score of ≥3 and ≤30 at Baseline * Female 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 NPC disease 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) * Current treatment with miglustat, provided the patient has been using the recommended dose for most of the past 12 months AND is, in the opinion of the investigator, satisfactorily treated with miglustat. Any participants receiving miglustat are required to undergo a 1-month washout period before starting study medication

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Conditions

The condition(s) this trial relates to.

Niemann-Pick disease type C

As listed by the trial registrant

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

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

    34 sites in 15 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

  • 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 Principe

    NOT_YET_RECRUITING

    Curitiba, Brazil

  • 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 Children's 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: •••••@•••••

  • 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 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

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