Oral nizubaglustat (AZ-3102) for late‑infantile and juvenile Niemann‑Pick Type C

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This Phase 3, double‑blind, randomized, placebo‑controlled multicenter trial enrolls participants aged 4 years and older with confirmed late‑infantile or juvenile NPC and baseline ataxia (SARA score 3–30). Participants receive once‑daily oral nizubaglustat or matching placebo for 18 months, with scheduled visits for clinical outcome measures, safety monitoring, and PK/PD sampling. The primary endpoint is superiority of nizubaglustat versus placebo on ataxic manifestations over 18 months, with secondary endpoints including other neurological features, pharmacokinetics/pharmacodynamics, and tolerability. The protocol is conducted at pediatric specialty centers and follows a master protocol framework referenced to the AZA‑001‑301 master protocol.

Why it matters

Who can join: the full rules

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

Where it's running

• 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

Study contacts

• Study coordinator: Patient Advocacy Representative
• Email: info@azafaros.com
• Phone: Please reach out by email

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Oral nizubaglustat (AZ-3102) for late‑infantile and juvenile Niemann‑Pick C, GM1, and GM2 gangliosidoses

  This 18‑month study will test whether daily oral nizubaglustat (AZ‑3102) helps people age 4 and up with late‑infantile or juvenile Niemann‑Pick C, GM1, or GM2 gangliosidosis compared with placebo.

  Recruiting at 29 sites Phase 3 Azafaros B.V. · Oakland, California and 38 other locations · NCT07054515

• Creating a database for Niemann-Pick Disease, Type C

  This study is collecting blood samples from people with Niemann-Pick Disease, Type C to build a database that helps researchers understand the disease better and find new treatment options.

  Recruiting National Institutes of Health Clinical Center (CC) · Bethesda, Maryland · NCT05588167

• N-acetyl-L-leucine for Niemann-Pick type C

  This trial tests whether N-acetyl-L-leucine helps people aged 4 and older with Niemann-Pick type C by comparing the medicine to a placebo in a randomized, double-blind crossover design.

  Recruiting Phase 3 IntraBio Inc · Rochester, Minnesota and 13 other locations · NCT05163288

• Evaluation of markers in Niemann-Pick Disease, Type C

  This study is trying to find signs in the body that show how Niemann-Pick Disease, Type C is getting worse, to help track progress and see how well new treatments might work for people with this condition.

  Recruiting National Institutes of Health Clinical Center (CC) · Bethesda, Maryland · NCT00344331

• Natural history of acid sphingomyelinase deficiency in adults and children

  This national French registry follows people with ASMD (including Niemann-Pick A/B/B-like types) to see how the disease progresses and how treatments affect daily life over time.

  Recruiting Groupe Hospitalier Diaconesses Croix Saint-Simon · Paris, France · NCT06985212

• Hyperspectral retinal imaging to detect Alzheimer’s and other neurodegenerative changes

  This project will test whether a special retinal camera that scans many wavelengths of light can detect signs of Alzheimer’s and other neurodegenerative diseases in people with dementia or at risk.

  Recruiting Center for Eye Research Australia · Melbourne, Victoria · NCT07545473

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.