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Study of Nizubaglustat for Niemann-Pick Type C Disease

This study is testing a drug called nizubaglustat (AZ-3102) to see if it is safe and effective for people with late-infantile and juvenile forms of Niemann-Pick Type C disease (NPC). Participants will receive either nizubaglustat or a placebo (an inactive substance) for 18 months. The main goal is to see if nizubaglustat can improve ataxia (problems with coordination and balance) compared to the placebo. You may be able to join if you are at least 4 years old, have a confirmed diagnosis of NPC, and started having neurological symptoms between ages 2 and 15. The study is currently recruiting 72 participants.

Study design
This is an 18-month, double-blind (neither you nor your doctor will know if you're getting the drug or placebo), randomized (assigned by chance), placebo-controlled study involving 72 participants.
What's involved
You would take either nizubaglustat or a placebo daily for 18 months. Your ataxia and other symptoms will be assessed from the start of the study to month 18.
Compensation
Not stated in the trial record.
Follow-up
Your progress will be assessed for 18 months after starting treatment.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT07082725

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
PHASE3Ages 4+InterventionalTreatment
Azafaros B.V.
~72 participants
Updated 2026-08-17 on ClinicalTrials.gov
What's tested:NizubaglustatPlacebo

At a glance

Recruiting sites
24 of 34 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Change from baseline in total Scale for the Assessment and Rating of Ataxia (SARA) score
Measured over Baseline to month 18
+1 more outcome measured

Conditions

Niemann-Pick Type C Disease

Explore related trials

Niemann-Pick Disease, Type C

NCT07082725

Where you'd take part

This study runs at 34 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • All India Institute of Medical Sciences (AIIMS) - New Delhi

    New Delhi, National Capital Territory of Delhi, Indiastudy coordinator listed

    Recruiting

  • Amrita Institute of Medical Sciences and Research Centre

    Ernākulam, Kerala, Indiastudy coordinator listed

    Recruiting

  • Centre Hospitalier de l'Universite de Montreal-1000 rue Saint-Denis

    Montreal, Quebec, Canadastudy coordinator listed

    Not yet recruiting

  • Children's Medical Center Dallas

    Dallas, Texasstudy coordinator listed

    Recruiting

  • Christian Medical College and Hospital

    Vellore, Tamil Nadu, Indiastudy coordinator listed

    Recruiting

  • Cukurova Universitesi Balcali Hastanesi Saglik Uygulama ve Arastirma Merkezi

    Adana, Adana, Turkey (Türkiye)study coordinator listed

    Recruiting

  • Ege Universitesi Tip Fakultesi

    Bornova, İzmir, Turkey (Türkiye)study coordinator listed

    Recruiting

  • Fondazione IRCCS Istituto Neurologico Carlo Besta

    Milan, Italystudy coordinator listed

    Recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Who to contact

Patient Advocacy Representative
[email protected]
Please reach out by email
Email the study team

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Eligibility criteria

Inclusion

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

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 trial measures

  • Change from baseline in total Scale for the Assessment and Rating of Ataxia (SARA) scoreBaseline to month 18

    Total SARA comprises eight categories with a cumulative score ranging from 0 (no ataxia) to 40 (most severe ataxia)

  • Change from baseline in functional SARA scoreBaseline to month 18

    Functional SARA uses an abbreviated scale that scores 0 to 16, with higher scores indicating more severe impairment