RECRUITING

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)

Conditions

Niemann-Pick Type C Disease Nizubaglustat

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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

Official Title

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 and in Late-infantile and Juvenile-onset Forms of GM1 Gangliosidosis or GM2 Gangliosidosis

Quick Facts

Study Start:2025-06-30

Study Completion:2027-11-04

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT07082725

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:4 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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	* 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

Inclusion Criteria

Exclusion 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

* 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

Contacts and Locations

Study Contact

Patient Advocacy Representative

CONTACT

Please reach out by email

patientadvocacy@azafaros.com

Contact for Healthcare Professionals

CONTACT

Please reach out by email

medinfo@azafaros.com

Study Locations (Sites)

Children's Hospital and Research Center at Oakland

Oakland, California, 94609

United States

Lysosomal Rare Disorders Research and Treatment Center

Fairfax, Virginia, 22030-7404

United States

Collaborators and Investigators

Sponsor: Azafaros A.G.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-06-30

Study Completion Date2027-11-04

Study Record Updates

Study Start Date2025-06-30

Study Completion Date2027-11-04

Terms related to this study

Keywords Provided by Researchers

Nizubaglustat

Additional Relevant MeSH Terms

Niemann-Pick Type C Disease