A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC)
18-month Double-blind, Randomized, Placebo-controlled, Multicenter, Phase 3 Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease and in Late-infantile and Juvenile-onset Forms of GM1 Gangliosidosis or GM2 Gangliosidosis
- Niemann-Pick Type C Disease
At a glance
- Phase
- Phase 3
- Study type
- Interventional
- Sponsor
- Azafaros B.V.
- Enrolment target
- 72
- Started
- 30 June 2025
- Main results due
- 14 May 2028
- Study sites
- 34
- Registry updated
- 28 September 2026
Can you take part?
- Aged 4 years and over.
- Open to any sex.
- You need the condition being studied — healthy volunteers are not accepted.
These are the headline rules only. Every study has a longer list, and whether you are eligible is decided by the research team at the site — never by this page.
Read the full eligibility criteria
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
What this study is about
In the sponsor’s own words, from the registry.
An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of Niemann-Pick type C disease
Study sites(34)
UCSF Children's Hospital and Research Center at Oakland
Oakland, California, United States
Recruiting
University of Minnesota Medical School
Minneapolis, Minnesota, United States
Recruiting
Mayo Clinic Children's Center - PIN
Rochester, Minnesota, United States
Recruiting
Children's Medical Center Dallas
Dallas, Texas, United States
Recruiting
Lysosomal Rare Disorders Research and Treatment Center
Fairfax, Virginia, United States
Recruiting
Hospital de Niños de La Santisima Trinidad
Córdoba, Córdoba Province, Argentina
Recruiting
Women's and Children's Hospital
North Adelaide, South Australia, Australia
Recruiting
Royal Melbourne Hospital
Parkville, Victoria, Australia
Recruiting
Royal Children's Hospital Melbourne - PIN
Parkville, Victoria, Australia
Recruiting
Instituto Fernandes Figueira
Rio de Janeiro, Rio de Janeiro, Brazil
Recruiting
Hospital de Clinicas de Porto Alegre (HCPA) - PPDS
Porto Alegre, Rio Grande do Sul, Brazil
Recruiting
Hospital Pequeno Principe
Curitiba, Brazil
Not yet recruiting
M.A.G.I.C. Clinic Ltd. Metabolics and Genetics in Calgary
Calgary, Alberta, Canada
Recruiting
University of Alberta Medical Genetics Clinic
Edmonton, Alberta, Canada
Not yet recruiting
Centre Hospitalier de l'Universite de Montreal-1000 rue Saint-Denis
Montreal, Quebec, Canada
Not yet recruiting
SphinCS GmbH
Höchheim, Germany
Recruiting
Amrita Institute of Medical Sciences and Research Centre
Ernākulam, Kerala, India
Recruiting
All India Institute of Medical Sciences (AIIMS) - New Delhi
New Delhi, National Capital Territory of Delhi, India
Recruiting
JK Lone Hospital
Jaipur, Rajasthan, India
Recruiting
Christian Medical College and Hospital
Vellore, Tamil Nadu, India
Recruiting
Fondazione IRCCS Istituto Neurologico Carlo Besta
Milan, Italy
Recruiting
Hospital Universitario Dr. Jose Eleuterio González
Monterrey, Nuevo León, Mexico
Not yet recruiting
Centenario Hospital Miguel Hidalgo
Aguascalientes, Mexico
Not yet recruiting
The Children's Hospital & the University of Child Health Lahore
Lahore, Punjab Province, Pakistan
Not yet recruiting
ULS de Santo António, EPE - Centro Materno Infantil Norte
Porto, Porto District, Portugal
Recruiting
Hospital Universitario Vall d'Hebron - PPDS
Barcelona, Barcelona, Spain
Recruiting
Hospital Infantil Universitario Niño Jesus - PIN
Madrid, Madrid, Spain
Recruiting
Inselspital - Universitätsspital Bern
Bern, Canton of Bern, Switzerland
Recruiting
Cukurova Universitesi Balcali Hastanesi Saglik Uygulama ve Arastirma Merkezi
Adana, Adana, Turkey (Türkiye)
Recruiting
Gazi Universitesi Saglik Arastirma ve Uygulama Merkezi
Çankaya, Ankara, Turkey (Türkiye)
Recruiting
Ege Universitesi Tip Fakultesi
Bornova, İzmir, Turkey (Türkiye)
Recruiting
Great Ormond Street Hospital
London, London, United Kingdom
Recruiting
University College London Hospitals (UCLH)
London, Middlesex, United Kingdom
Not yet recruiting
Royal Manchester Children's Hospital
Manchester, United Kingdom
Not yet recruiting
Showing 34 of 34 sites. 26 of the 34 sites on this study are recruiting right now — a site can stop enrolling while the study as a whole is still open.
Source: ClinicalTrials.gov record NCT07082725. Trialion does not run this study, is not paid to refer anyone to it, and cannot enrol you. Eligibility is always decided by the research team at the site.
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