Race against time: new pill aims to slow devastating childhood brain disease

NCT ID NCT07082543

Recruiting now Disease control Sponsor: Azafaros A.G. Source: ClinicalTrials.gov ↗

Summary

This study is testing whether a daily oral medicine called nizubaglustat can slow the progression of two rare, severe genetic brain diseases (GM1 and GM2 gangliosidosis) in children. For 18 months, about 75 participants aged 4 and older will receive either the real medicine or a placebo pill to see if it safely improves movement, speech, and daily function. The goal is to control the disease and preserve quality of life for as long as possible.

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes NO responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

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Study contacts

Contact

Phone: •••-•••-•••• Email: •••••@•••••
Contact

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Locations

AP-HP - Hôpital Armand Trousseau
RECRUITING

Paris, 75012, France

Contact Phone: •••-•••-•••• Email: •••••@•••••
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
NOT_YET_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
NOT_YET_RECRUITING

Dallas, Texas, 75235, United States
Christian Medical College and Hospital
NOT_YET_RECRUITING

Vellore, Tamil Nadu, 632004, India
Ege Universitesi Tip Fakultesi
NOT_YET_RECRUITING

Bornova, İzmir, 35100, Turkey (Türkiye)
Fondazione IRCCS Istituto Neurologico Carlo Besta
RECRUITING

Milan, 20133, Italy

Contact Phone: •••-•••-•••• Email: •••••@•••••
Gazi Universitesi Saglik Arastirma ve Uygulama Merkezi
NOT_YET_RECRUITING

Çankaya, Ankara, 6500, Turkey (Türkiye)
Great Ormond Street Hospital NHSFT
NOT_YET_RECRUITING

London, United Kingdom
Hospital Infantil Universitario Niño Jesus - PIN
RECRUITING

Madrid, Madrid, 28009, Spain

Contact Email: •••••@•••••
Hospital Pequeno Principe
NOT_YET_RECRUITING

Curitiba, Brazil

Contact Phone: •••-•••-•••• Email: •••••@•••••
Hospital Universitario Austral
NOT_YET_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

Contact Phone: •••-•••-•••• Email: •••••@•••••
Hospital de Clinicas de Porto Alegre (HCPA) - PPDS
NOT_YET_RECRUITING

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

Contact Phone: •••-•••-•••• Email: •••••@•••••

Contact
Hospital de Niños de La Santisima Trinidad
NOT_YET_RECRUITING

Córdoba, Córdoba Province, X5004 ASL, Argentina
Inselspital - Universitätsspital Bern
NOT_YET_RECRUITING

Bern, Canton of Bern, 3010, Switzerland

Contact Phone: •••-•••-•••• Email: •••••@•••••
Instituto Fernandes Figueira
NOT_YET_RECRUITING

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

Contact Phone: •••-•••-•••• Email: •••••@•••••
JK Lone Hospital
RECRUITING

Jaipur, Rajasthan, 302004, India
Lysosomal Rare Disorders Research and Treatment Center
RECRUITING

Fairfax, Virginia, 22030-7404, United States

Contact Phone: •••-•••-•••• Email: •••••@•••••
M.A.G.I.C. Clinic Ltd. Metabolics and Genetics in Calgary
NOT_YET_RECRUITING

Calgary, Alberta, T3B 6A8, Canada
Mayo Clinic Children's Center - PIN
NOT_YET_RECRUITING

Rochester, Minnesota, 55905, United States
Royal Children's Hospital Melbourne - PIN
NOT_YET_RECRUITING

Parkville, Victoria, 3052, Australia

Contact Phone: •••-•••-•••• Email: •••••@•••••
Royal Manchester Children's Hospital
NOT_YET_RECRUITING

Manchester, M13 9WL, United Kingdom
Sahlgrenska universitetssjukhuset Östra
RECRUITING

Gothenburg, Västra Götaland County, 416 50, Sweden
SphinCS GmbH
NOT_YET_RECRUITING

Höchheim, 65239, Germany
UCSF Children's Hospital and Research Center at Oakland
RECRUITING

Oakland, California, 94609, United States

Contact

Contact Phone: •••-•••-•••• Email: •••••@•••••
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

Contact Phone: •••-•••-•••• Email: •••••@•••••
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

Contact Phone: •••-•••-•••• Email: •••••@•••••
University of Minnesota Medical School
NOT_YET_RECRUITING

Minneapolis, Minnesota, 55455, United States
Women's and Children's Hospital
NOT_YET_RECRUITING

North Adelaide, South Australia, 5006, Australia

Contact Phone: •••-•••-•••• Email: •••••@•••••

Conditions

Explore the condition pages connected to this study.

GANGLIOSIDOSES, GM2 GANGLIOSIDOSIS, GM1