NCT07082725 · Azafaros A.G.
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)
What this study is about
An 18-month where neither patients nor doctors know which treatment is given, randomly assigned, compared against an inactive treatment, conducted at multiple hospitals, Phase 3 study to evaluate the safety and effectiveness of taken by mouth nizubaglustat (AZ-3102) in late-infantile and juvenile forms of Niemann-Pick type C disease
View original scientific description
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
Interventions
DRUG
Nizubaglustat
AZ-3102
DRUG
Placebo
Matching placebo
Primary outcome measures
Change from baseline in total Scale for the Assessment and Rating of Ataxia (SARA) score
Time frame: Baseline 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 score
Time frame: Baseline to month 18
Functional SARA uses an abbreviated scale that scores 0 to 16, with higher scores indicating more severe impairment
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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 *
Where
- Oakland, California
- Minneapolis, Minnesota
- Rochester, Minnesota
- Dallas, Texas
- Fairfax, Virginia
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jul 14, 2026 · Source of record for eligibility and locations