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)
NCT ID: NCT07082725
Last Updated: 2025-11-10
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
RECRUITING
PHASE3
72 participants
INTERVENTIONAL
2025-06-30
2027-11-04
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
PRIMARY OBJECTIVE
The primary objective of this study is to demonstrate superior efficacy on ataxic manifestations with oral nizubaglustat dosing compared with placebo when administered over 18 months in participants with late-infantile and juvenile forms of NPC disease
SECONDARY OBJECTIVES
I. To assess additional efficacy in ataxic and non-ataxic manifestations comparing nizubaglustat dosing with placebo when administered over 18 months in participants with late-infantile and juvenile forms of NPC disease
II. To assess the pharmacokinetic (PK) properties of nizubaglustat after administration of the first dose (Visit 1) and at steady state after multiple once daily doses
III. To assess the pharmacodynamic (PD) effects of nizubaglustat
IV. To assess the safety and tolerability of daily oral nizubaglustat dosing compared with placebo, when administered over 18 months in participants with late-infantile and juvenile forms of NPC disease
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Keywords
Explore important study keywords that can help with search, categorization, and topic discovery.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Nizubaglustat
Once daily oral dispersible tablets
Nizubaglustat
AZ-3102
Placebo
Matching placebo
Placebo
Matching placebo
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
Nizubaglustat
AZ-3102
Placebo
Matching placebo
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* 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
* 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
4 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Azafaros A.G.
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Children's Hospital and Research Center at Oakland
Oakland, California, United States
University of Minnesota Medical School
Minneapolis, Minnesota, United States
Children's Medical Center Dallas
Dallas, Texas, United States
Lysosomal Rare Disorders Research and Treatment Center
Fairfax, Virginia, United States
Hospital Universitario Austral
Ciudad Autónoma Buenos Aires, Buenos Aires, Argentina
Hospital de Niños de La Santisima Trinidad
Córdoba, Córdoba Province, Argentina
Women's and Children's Hospital
North Adelaide, South Australia, Australia
Royal Melbourne Hospital
Parkville, Victoria, Australia
Royal Children's Hospital Melbourne - PIN
Parkville, Victoria, Australia
Instituto Fernandes Figueira
Rio de Janeiro, Rio de Janeiro, Brazil
Hospital de Clinicas de Porto Alegre (HCPA) - PPDS
Porto Alegre, Rio Grande do Sul, Brazil
Hospital Pequeno Principe
Curitiba, , Brazil
M.A.G.I.C. Clinic Ltd. Metabolics and Genetics in Calgary
Calgary, Alberta, Canada
University of Alberta Medical Genetics Clinic
Edmonton, Alberta, Canada
Centre Hospitalier de l'Universite de Montreal-1000 rue Saint-Denis
Montreal, Quebec, Canada
SphinCS GmbH
Höchheim, , Germany
Amrita Institute of Medical Sciences and Research Centre
Ernākulam, Kerala, India
Seth G S Medical College and K E M Hospital
Mumbai, Maharashtra, India
All India Institute of Medical Sciences (AIIMS) - New Delhi
New Delhi, National Capital Territory of Delhi, India
JK Lone Hospital
Jaipur, Rajasthan, India
Fondazione IRCCS Istituto Neurologico Carlo Besta
Milan, , Italy
ULS de Santo António, EPE - Centro Materno Infantil Norte
Porto, Porto District, Portugal
Hospital Universitario Vall d'Hebron - PPDS
Barcelona, Barcelona, Spain
Hospital Infantil Universitario Niño Jesus - PIN
Madrid, Madrid, Spain
Inselspital - Universitätsspital Bern
Bern, Canton of Bern, Switzerland
Balcali Hastanesi Saglik Uygulama ve Arastirma Merkezi
Adana, Adana, Turkey (Türkiye)
Gazi Universitesi Saglik Arastirma ve Uygulama Merkezi
Çankaya, Ankara, Turkey (Türkiye)
Ege Universitesi Tip Fakultesi
Bornova, İzmir, Turkey (Türkiye)
Great Ormond Street Hospital
London, London, United Kingdom
University College London Hospitals (UCLH)
London, Middlesex, United Kingdom
Royal Manchester Children's Hospital
Manchester, , United Kingdom
Countries
Review the countries where the study has at least one active or historical site.
Central Contacts
Reach out to these primary contacts for questions about participation or study logistics.
Patient Advocacy Representative
Role: CONTACT
Phone: Please reach out by email
Email: [email protected]
Contact for Healthcare Professionals
Role: CONTACT
Phone: Please reach out by email
Email: [email protected]
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
2024-515778-28-00
Identifier Type: CTIS
Identifier Source: secondary_id
AZA-001-301-NPC
Identifier Type: -
Identifier Source: org_study_id