New pill hopes to slow rare brain disease in kids

NCT ID NCT07082725

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

First seen Jun 27, 2026 · Last updated Aug 12, 2026 · Updated 11 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.

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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

Contacts and locations

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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
Balcali Hastanesi Saglik Uygulama ve Arastirma Merkezi
RECRUITING

Adana, Adana, 1250, Turkey (Türkiye)
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
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
Royal Children's Hospital Melbourne - PIN
NOT_YET_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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