Eligibility Module
The Eligibility Module contains detailed information about who can participate in the clinical trial. This includes eligibility criteria, age restrictions, gender requirements, healthy volunteer status, and study population descriptions, helping researchers understand who is eligible to participate in the study.
Eligibility Module path is as follows:
Study -> Protocol Section -> Eligibility Module
Eligibility Module
Ignite Creation Date:
2026-03-26 @ 3:16 PM
Ignite Modification Date:
2026-03-26 @ 3:16 PM
NCT ID:
NCT07399704
Eligibility Criteria:
Inclusion Criteria:
* Cohort 1 (NPC and GM2 patients):
* Have been randomized into Phase 2 Study AZA-001-5A2-01.
OR
Cohort 2 (NPC patients):
* Be male or female aged ≥12 years
* Have a genetically-confirmed diagnosis of NPC disease
* Have received full-dose Miglustat treatment for at least 12 months and experienced disease stabilization or worsening with treatment over the 2 previous clinic visits. Patients experiencing clinical improvement with Miglustat over the preceding 3 months should not be considered for this study.
* Wish to change treatment to Nizubaglustat for their NPC disease.
* Participants from Phase 2 Study AZA-001-5A2-01 (RAINBOW) who transitioned to Miglustat may be eligible for Cohort 2 if they meet all other criteria.
Participation is supported and deemed beneficial by the Principal Investigator. Be willing and able to be evaluated for all protocol assessments. The participant, parent, and/or legal guardian can read, understand, and sign the informed consent form. Where appropriate, assent will also be sought for participants who have not reached the age of majority.
Exclusion Criteria:
* A positive serum pregnancy test (only tested for women of childbearing potential).
* Female planning to breastfeed during the study.
* Any medical event/condition that prevents participation in the study based on the judgment of the Principal Investigator.
* Participation in another interventional or non-interventional study or early access program.
Healthy Volunteers:
False
Sex:
ALL
Minimum Age:
12 Years
Study:
NCT07399704
Study Brief:
Protocol Section:
NCT07399704