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)
Azafaros B.V. · NCT07082725
Opens the official ClinicalTrials.gov record, with the study team’s contacts. Bored Studies doesn’t enroll anyone.
Key facts
- Compensation
- Not posted. Ask the study team before screening.
- Who can join
- Ages 4+ · All sexes
- Healthy volunteers
- Not accepted (a condition is required)
- Study type
- Interventional · Phase 3
- Target enrollment
- 72
- US sites recruiting
- 5
- Study start
- June 30, 2025
- Record last updated
- September 28, 2026
Status re-checked against ClinicalTrials.gov within the last 12 hours.
What the study is about
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
Being studied: Nizubaglustat; Placebo
Conditions: Niemann-Pick Type C Disease
Eligibility criteria
Copied from the official record. The study team makes the final decision at screening.
Show full 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
Recruiting US locations
UCSF Children's Hospital and Research Center at Oakland
Oakland, CA 94609
Site contact: Veronica Cheung, CRC · 510-428-3885 · Veronica.Cheung@ucsf.edu
University of Minnesota Medical School
Minneapolis, MN 55455
Site contact: Melissa Fetterly · 612-672-5151 · melissa.fetterley@fairview.org
Mayo Clinic Children's Center - PIN
Rochester, MN 55905
Site contact: Brad Jorde · 507-293-3609 · RSTCGRESEARCH@mayo.edu
Children's Medical Center Dallas
Dallas, TX 75235
Site contact: Charina Ramirez, MD · 214-456-8000 · charina.ramirez@utsouthwestern.edu
Lysosomal Rare Disorders Research and Treatment Center
Fairfax, VA 22030-7404
Site contact: Lauren Noll · 571-732-4655 · lnoll@ldrtc.org
Study contacts (from the official record)
- Patient Advocacy Representative · Please reach out by email · info@azafaros.com
- Contact for Healthcare Professionals · Please reach out by email · medinfo@azafaros.com