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)

NCT07082725 | Recruiting Phase 3 DMC FDA Drug

• 2 other identifiers: AZA-001-301-NPC2024-515778-28-00
• Study Type: interventional
• Target: 72
• Locations: 15 countries
• Sites: 34

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

Keywords

Nizubaglustat

Outcome Measures

Primary Outcomes (2)

• Change from baseline in total Scale for the Assessment and Rating of Ataxia (SARA) score

  Total SARA comprises eight categories with a cumulative score ranging from 0 (no ataxia) to 40 (most severe ataxia)

  Baseline to month 18

• Change from baseline in functional SARA score

  Functional SARA uses an abbreviated scale that scores 0 to 16, with higher scores indicating more severe impairment

  Baseline to month 18

Secondary Outcomes (21)

• Change from baseline in SARA score for gait/posture

  Baseline to months 6, 12, and 18

• Change from baseline in SARA score for speech

  Baseline to months 6, 12, and 18

• Change from baseline in SARA score for kinetics

  Baseline to months 6, 12, and 18

• Change from baseline in Vineland Adaptive Behavior Scale (VABS)

  Baseline to months 6, 12, and 18

• Change from baseline in Penetration-Aspiration Scale (PAS)

  Baseline to months 6, 12, and 18

Study Arms (2)

Nizubaglustat

EXPERIMENTAL

Once daily oral dispersible tablets

Drug: Nizubaglustat

Placebo

PLACEBO COMPARATOR

Matching placebo

Drug: Placebo

Interventions

Placebo DRUG

Matching placebo

Placebo

Nizubaglustat DRUG

AZ-3102

Nizubaglustat

Eligibility Criteria

• Age: 4 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Azafaros B.V.: lead

Study Sites (34)

UCSF Children's Hospital and Research Center at Oakland

Oakland, California, 94609, United States

RECRUITING

University of Minnesota Medical School

Minneapolis, Minnesota, 55455, United States

NOT YET RECRUITING

Mayo Clinic Children's Center - PIN

Rochester, Minnesota, 55905, United States

RECRUITING

Children's Medical Center Dallas

Dallas, Texas, 75235, United States

RECRUITING

Lysosomal Rare Disorders Research and Treatment Center

Fairfax, Virginia, 22030-7404, United States

RECRUITING

Hospital de Niños de La Santisima Trinidad

Córdoba, Córdoba Province, X5004 ASL, Argentina

RECRUITING

Women's and Children's Hospital

North Adelaide, South Australia, 5006, Australia

RECRUITING

Royal Melbourne Hospital

Parkville, Victoria, 3050, Australia

RECRUITING

Royal Children's Hospital Melbourne - PIN

Parkville, Victoria, 3052, Australia

NOT YET RECRUITING

Instituto Fernandes Figueira

Rio de Janeiro, Rio de Janeiro, 22250-020, Brazil

RECRUITING

Hospital de Clinicas de Porto Alegre (HCPA) - PPDS

Porto Alegre, Rio Grande do Sul, 90560-030, Brazil

RECRUITING

Hospital Pequeno Principe

Curitiba, Brazil

NOT YET RECRUITING

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

Calgary, Alberta, T3B 6A8, Canada

RECRUITING

University of Alberta Medical Genetics Clinic

Edmonton, Alberta, T6G 2B7, Canada

NOT YET RECRUITING

Centre Hospitalier de l'Universite de Montreal-1000 rue Saint-Denis

Montreal, Quebec, H2X 0C1, Canada

NOT YET RECRUITING

SphinCS GmbH

Höchheim, 65239, Germany

RECRUITING

Amrita Institute of Medical Sciences and Research Centre

Ernākulam, Kerala, 682041, India

RECRUITING

All India Institute of Medical Sciences (AIIMS) - New Delhi

New Delhi, National Capital Territory of Delhi, 110029, India

RECRUITING

JK Lone Hospital

Jaipur, Rajasthan, 302004, India

RECRUITING

Christian Medical College and Hospital

Vellore, Tamil Nadu, 632004, India

NOT YET RECRUITING

Fondazione IRCCS Istituto Neurologico Carlo Besta

Milan, 20133, Italy

RECRUITING

Hospital Universitario Dr. Jose Eleuterio González

Monterrey, Nuevo León, 64460, Mexico

NOT YET RECRUITING

Centenario Hospital Miguel Hidalgo

Aguascalientes, 20000, Mexico

NOT YET RECRUITING

The Children's Hospital & the University of Child Health Lahore

Lahore, Punjab Province, Pakistan

NOT YET RECRUITING

ULS de Santo António, EPE - Centro Materno Infantil Norte

Porto, Porto District, 4050-651, Portugal

RECRUITING

Hospital Universitario Vall d'Hebron - PPDS

Barcelona, Barcelona, 8035, Spain

RECRUITING

Hospital Infantil Universitario Niño Jesus - PIN

Madrid, Madrid, 28009, Spain

RECRUITING

Inselspital - Universitätsspital Bern

Bern, Canton of Bern, 3010, Switzerland

RECRUITING

Balcali Hastanesi Saglik Uygulama ve Arastirma Merkezi

Adana, Adana, 1250, Turkey (Türkiye)

RECRUITING

Gazi Universitesi Saglik Arastirma ve Uygulama Merkezi

Çankaya, Ankara, 6500, Turkey (Türkiye)

RECRUITING

Ege Universitesi Tip Fakultesi

Bornova, İzmir, 35100, Turkey (Türkiye)

RECRUITING

Great Ormond Street Hospital

London, London, WC1N 3JH, United Kingdom

RECRUITING

University College London Hospitals (UCLH)

London, Middlesex, WC1N 3BG, United Kingdom

NOT YET RECRUITING

Royal Manchester Children's Hospital

Manchester, M13 9WL, United Kingdom

NOT YET RECRUITING

MeSH Terms

Conditions

Niemann-Pick Disease, Type C

Condition Hierarchy (Ancestors)

Niemann-Pick Diseases; Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Histiocytosis, Non-Langerhans-Cell; Histiocytosis; Lymphatic Diseases; Hemic and Lymphatic Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Central Study Contacts

Patient Advocacy Representative

CONTACT

Please reach out by email; info@azafaros.com

Contact for Healthcare Professionals

CONTACT

Please reach out by email; medinfo@azafaros.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 27, 2025
• First Posted: July 24, 2025
• Study Start: June 30, 2025
• Primary Completion (Estimated): May 14, 2028
• Study Completion (Estimated): June 14, 2028
• Last Updated: August 4, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will not share

Locations

ME (2); PA (1); AU (3); ES (2); US (5); AR (1); CH (1); TU (3); IT (1); GB (3); IN (4); DE (1); CA (3); BR (3); PT (1)