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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)
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)
RecruitingPhase 3
Sponsor: Azafaros B.V.
Conditions: Niemann-Pick Type C Disease
Interventions: Nizubaglustat, Placebo
Countries: United States, Argentina, Australia, Brazil, Canada, Germany, India, Italy
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
Eligibility overview
Sex: ALL
Age: 4 Years to —
Healthy volunteers: No
Study type: INTERVENTIONAL
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
Locations (34)
- Oakland, California, United States
- Minneapolis, Minnesota, United States
- Rochester, Minnesota, United States
- Dallas, Texas, United States
- Fairfax, Virginia, United States
- Córdoba, Córdoba Province, Argentina
- North Adelaide, South Australia, Australia
- Parkville, Victoria, Australia
- Parkville, Victoria, Australia
- Rio de Janeiro, Rio de Janeiro, Brazil
- Porto Alegre, Rio Grande do Sul, Brazil
- Curitiba, Brazil
- Calgary, Alberta, Canada
- Edmonton, Alberta, Canada
- Montreal, Quebec, Canada
- Höchheim, Germany
- Ernākulam, Kerala, India
- New Delhi, National Capital Territory of Delhi, India
- Jaipur, Rajasthan, India
- Vellore, Tamil Nadu, India
- Milan, Italy
- Monterrey, Nuevo León, Mexico
- Aguascalientes, Mexico
- Lahore, Punjab Province, Pakistan
- Porto, Porto District, Portugal
- Barcelona, Barcelona, Spain
- Madrid, Madrid, Spain
- Bern, Canton of Bern, Switzerland
- Adana, Adana, Turkey (Türkiye)
- Çankaya, Ankara, Turkey (Türkiye)
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