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)

Recruiting now · Phase 3 · Has a placebo group

Conditions studied: Niemann-Pick Type C Disease

In brief

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

Key facts

Study ID

NCT07082725

Run by

Azafaros B.V.

People needed

72

Starts

2025-06-30

Expected to finish

2028-06-14

Last updated by the study team

2026-08-04

Who can join

Age: 4 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• UCSF Children's Hospital and Research Center at Oakland — Oakland, California, United States (enrolling)
• All India Institute of Medical Sciences (AIIMS) - New Delhi — New Delhi, National Capital Territory of Delhi, India (enrolling)
• JK Lone Hospital — Jaipur, Rajasthan, India (enrolling)
• Fondazione IRCCS Istituto Neurologico Carlo Besta — Milan, Italy (enrolling)
• ULS de Santo António, EPE - Centro Materno Infantil Norte — Porto, Porto District, Portugal (enrolling)
• Hospital Universitario Vall d'Hebron - PPDS — Barcelona, Barcelona, Spain (enrolling)
• Hospital Infantil Universitario Niño Jesus - PIN — Madrid, Madrid, Spain (enrolling)
• Inselspital - Universitätsspital Bern — Bern, Canton of Bern, Switzerland (enrolling)
• Balcali Hastanesi Saglik Uygulama ve Arastirma Merkezi — Adana, Adana, Turkey (Türkiye) (enrolling)
• Gazi Universitesi Saglik Arastirma ve Uygulama Merkezi — Çankaya, Ankara, Turkey (Türkiye) (enrolling)
• Ege Universitesi Tip Fakultesi — Bornova, İzmir, Turkey (Türkiye) (enrolling)
• Great Ormond Street Hospital — London, London, United Kingdom (enrolling)
• Mayo Clinic Children's Center - PIN — Rochester, Minnesota, United States (enrolling)
• Children's Medical Center Dallas — Dallas, Texas, United States (enrolling)
• Lysosomal Rare Disorders Research and Treatment Center — Fairfax, Virginia, United States (enrolling)
• Hospital de Niños de La Santisima Trinidad — Córdoba, Córdoba Province, Argentina (enrolling)
• Women's and Children's Hospital — North Adelaide, South Australia, Australia (enrolling)
• Royal Melbourne Hospital — Parkville, Victoria, Australia (enrolling)
• Instituto Fernandes Figueira — Rio de Janeiro, Rio de Janeiro, Brazil (enrolling)
• Hospital de Clinicas de Porto Alegre (HCPA) - PPDS — Porto Alegre, Rio Grande do Sul, Brazil (enrolling)
• M.A.G.I.C. Clinic Ltd. Metabolics and Genetics in Calgary — Calgary, Alberta, Canada (enrolling)
• SphinCS GmbH — Höchheim, Germany (enrolling)
• Amrita Institute of Medical Sciences and Research Centre — Ernākulam, Kerala, India (enrolling)
• University of Minnesota Medical School — Minneapolis, Minnesota, United States
• Royal Manchester Children's Hospital — Manchester, United Kingdom

Full record on ClinicalTrials.gov

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