A Phase 3 interventional study of Nizubaglustat and Placebo in Niemann-Pick Type C Disease, sponsored by Azafaros B.V.. Recruiting at 34 sites in 15 countries. Open to participants aged 4 Years and older. Per ClinicalTrials.gov, last updated 2026-10-28.
Sponsored by Azafaros B.V. · Phase 3, Interventional, and Treatment
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
Please see NCT #07054515 for information on the AZA-001-301 Master Protocol
PRIMARY OBJECTIVE
The primary objective of this study is to demonstrate superior efficacy on ataxic manifestations with oral nizubaglustat dosing compared with placebo when administered over 18 months in participants with late-infantile and juvenile forms of NPC disease
SECONDARY OBJECTIVES
I. To assess additional efficacy in ataxic and non-ataxic manifestations comparing nizubaglustat dosing with placebo when administered over 18 months in participants with late-infantile and juvenile forms of NPC disease
II. To assess the pharmacokinetic (PK) properties of nizubaglustat after administration of the first dose (Visit 1) and at steady state after multiple once daily doses
III. To assess the pharmacodynamic (PD) effects of nizubaglustat
IV. To assess the safety and tolerability of daily oral nizubaglustat dosing compared with placebo, when administered over 18 months in participants with late-infantile and juvenile forms of NPC disease
37 studies on the registry are indexed under Niemann-Pick Disease, Type C; 8 are open to participants now.
This study's planned enrollment of 72 is above the median of 21 across 27 interventional studies indexed under Niemann-Pick Disease, Type C.
Browse Niemann-Pick Disease, Type C studies →Azafaros B.V. is the lead sponsor of 8 studies on the registry; 4 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
A planned gap after a treatment, so that its effects can lessen before the study treatment or a study assessment. Ask the study team and the doctor who prescribed it before changing any medicine.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Washout period
A planned gap after a treatment, so that its effects can lessen before the study treatment or a study assessment. Ask the study team and the doctor who prescribed it before changing any medicine.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Once daily oral dispersible tablets
Drug: Nizubaglustat
Matching placebo
Drug: Placebo
AZ-3102
Matching placebo
Change from baseline in total Scale for the Assessment and Rating of Ataxia (SARA) score
Total SARA comprises eight categories with a cumulative score ranging from 0 (no ataxia) to 40 (most severe ataxia)
Time frame: Baseline to month 18
Change from baseline in functional SARA score
Functional SARA uses an abbreviated scale that scores 0 to 16, with higher scores indicating more severe impairment
Time frame: Baseline to month 18
Change from baseline in SARA score for gait/posture
Assessed on a 9-point scale with higher scores indicating inability to complete the task
Time frame: Baseline to months 6, 12, and 18
Change from baseline in SARA score for speech
Assessed on a 7-point scale with higher scores indicating unintelligible speech/anarthria
Time frame: Baseline to months 6, 12, and 18
Change from baseline in SARA score for kinetics
Assessed on a 5-point scale with higher scores indicating more severe impairment
Time frame: Baseline to months 6, 12, and 18
Change from baseline in Vineland Adaptive Behavior Scale (VABS)
Assesses four domains of function: communication, daily living skills, socialization, and motor skills
Time frame: Baseline to months 6, 12, and 18
Change from baseline in Penetration-Aspiration Scale (PAS)
Assessed on an 8-point ordinal scale, with 1 representing the lowest and 8 the highest/most severe score
Time frame: Baseline to months 6, 12, and 18
Change from baseline in 9-Hole Peg Test (9-HPT-D)
Assessed by the number of pegs per second placed in a 50-second, 9-HPT using the dominant hand
Time frame: Baseline to months 6, 12, and 18
Change from baseline in NPC-Clinical Severity Scale (NPC-CSS)
Scores can range from 0 to 61, with a higher score indicating more severe clinical impairment
Time frame: Baseline to months 6, 12, and 18
Change from baseline in Goal Attainment scale (GAS)
Assesses individual goals achieved during the study
Time frame: Baseline to months 6, 12, and 18
Change from baseline in Clinician Global Impression of Change (CGI-C)
Assessed on a 7-point scale with higher scores indicating lower improvement
Time frame: Baseline to months 6, 12, and 18
Change from baseline in Participant/Caregiver Global Impression of Change (PGI-C)
Assessed on a 7-point scale with higher scores indicating lower improvement
Time frame: Baseline to months 6, 12, and 18
Change from baseline in Seizure frequency and duration, as per the seizure diary
Time frame: Baseline to months 6, 12, and 18
Time-to-event comparison between nizubaglustat and placebo over the study duration for pre-defined detrimental events of an increase in total SARA score of ≥2 points
Time frame: Baseline to month 18
Time-to-event comparison between nizubaglustat and placebo over the study duration for pre-defined detrimental events of an increase in functional SARA of ≥1.5 points
Time frame: Baseline to month 18
Time-to-event comparison between nizubaglustat and placebo over the study duration for pre-defined detrimental events of a decrease in PAS of ≥1 point
Time frame: Baseline to month 18
Time-to-event comparison between nizubaglustat and placebo over the study duration for pre-defined detrimental events of participants leaving the study due to participant/caregiver perception of disease progression/lack of efficacy
Time frame: Baseline to month 18
Maximum observed plasma concentration (Cmax)
Time frame: Baseline and months 1,18, and 21
Time to Cmax (Tmax)
Time frame: Baseline and months 1,18, and 21
Plasma trough concentration (Ctrough)
Time frame: Baseline and month 1
Area under the plasma concentration-time curve from time of dosing (zero) to 24 hours post-dose (AUC0-24) at baseline
Time frame: Baseline (Day 1)
Accumulation ratio for Cmax
Time frame: Baseline and months 1,18, and 21
Change from baseline in plasma concentration of glucosylceramide (GlcCer) C16:0; C18:0
Time frame: Baseline and months 1, 6, 12, and 18
Plan to share: No
No publications or documents are linked to this record.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
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Studies of the drug tested here: Nizubaglustat
Niemann-Pick Disease, Type C →
Azafaros B.V.