Recruiting
Phase 3

Nizubaglustat

Sponsor:

Azafaros B.V.

Code:

NCT07082725

Conditions

Niemann-Pick Type C Disease

Eligibility Criteria

Sex: All

Age: 4+

Healthy Volunteers: Not accepted

Interventions

Nizubaglustat

Placebo

Study Details

Brief summary:

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

Conditions

Niemann-Pick Type C Disease

Study ID

NCT07082725

Start date

Jun 30, 2025

Status verified date

Jun, 2026

Completion date

Jun 14, 2028

Anticipated

Primary completion date

May 14, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 4+

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

72 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Nizubaglustat

Once daily oral dispersible tablets

placebo comparator: Placebo

Matching placebo

Interventions

Nizubaglustat

AZ-3102

Placebo

Matching placebo

Primary outcome measure

  • Change from baseline in total Scale for the Assessment and Rating of Ataxia (SARA) score [ Time Frame: Baseline to month 18 ]
  • Change from baseline in functional SARA score [ Time Frame: Baseline to month 18 ]

Central Contacts and Locations

Central contacts

Patient Advocacy Representative

Please reach out by emailinfo@azafaros.com

Contact for Healthcare Professionals

Please reach out by emailmedinfo@azafaros.com

Locations

UCSF Children's Hospital and Research Center at Oakland

Recruiting

Oakland, California, United States, 94609

Contacts

Veronica Cheung, CRC

510-428-3885Veronica.Cheung@ucsf.edu

Principal Investigator:

Caroline Hastings, MD

Mayo Clinic Children's Center - PIN

Recruiting

Rochester, Minnesota, United States, 55905

Contacts

Brad Jorde

507-293-3609RSTCGRESEARCH@mayo.edu

Children's Medical Center Dallas

Recruiting

Dallas, Texas, United States, 75235

Contacts

Charina Ramirez, MD

214-456-8000charina.ramirez@utsouthwestern.edu

Lysosomal Rare Disorders Research and Treatment Center

Recruiting

Fairfax, Virginia, United States, 22030-7404

Contacts

Lauren Noll

571-732-4655lnoll@ldrtc.org

M.A.G.I.C. Clinic Ltd. Metabolics and Genetics in Calgary

Recruiting

Calgary, Alberta, Canada, T3B 6A8

Contacts

Olugbenga Olunaike

+1 587 885 3158olugbenga.olunaike@magiccalgany.ca

More Information

Sponsor

Azafaros B.V.

Last update posted

Aug 17, 2026

Last verified

Jun, 2026

Keywords

  • Nizubaglustat

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-04. This information was provided to ClinicalTrials.gov by Azafaros B.V. on 2026-08-17.