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)

Launched by AZAFAROS A.G. · Jul 16, 2025

Keywords

ClinConnect Summary

This clinical trial is studying a new oral medicine called nizubaglustat (AZ-3102) to see if it is safe and helpful for people with Niemann-Pick Type C disease, a rare condition that affects the brain and nervous system. The study focuses on children and young people who started showing symptoms between the ages of 2 and 15 and who have difficulties with movement, such as problems with balance and coordination. The trial will last about 18 months and compares the new medicine to a placebo (a pill with no active drug) without the participants or doctors knowing who is receiving which treatment.

To take part, patients need to be at least 4 years old, have a confirmed diagnosis of Niemann-Pick Type C disease, and either cannot take or are not responding well to the current treatment called miglustat. Participants must have some level of movement difficulties but not be severely disabled. Women who can have children and men with partners who can have children will need to use birth control during the study. People with other serious health problems or certain medical conditions won’t be eligible. If accepted, participants will receive the study medication or placebo and be closely monitored throughout the trial to check for safety and any improvements in their symptoms. This study is currently recruiting patients at multiple locations.

Gender

ALL

Eligibility criteria

• 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