A Phase 2 interventional study of AZ-3102 in GM2 Gangliosidosis and Niemann-Pick Type C Disease, sponsored by Azafaros B.V.. Active, not recruiting at 3 sites in Brazil. Open to participants aged 12 Years and older. Per ClinicalTrials.gov, last updated 2026-10-21.
Sponsored by Azafaros B.V. · Phase 2, Interventional, and Treatment
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This open-label study aims to gather long-term safety, tolerability, PK, biomarker, and clinical efficacy data relating to daily administration of Nizubaglustat in participants previously enrolled in the Phase 2 RAINBOW study (Cohort 1). In addition, the study aims to assess safety, clinical, and biochemical impact of transitioning NPC disease patients to Nizubaglustat after prior treatment with stable, full-dose Miglustat (Cohort 2).
This is a multicenter, open-label study to assess the safety, tolerability, PK, PD, and efficacy of Nizubaglustat in male or female patients with late-infantile or juvenile onset GM2 gangliosidosis or NPC disease in two cohorts:
26 studies on the registry are indexed under Gangliosidoses, GM2; 6 are open to participants now.
This study's planned enrollment of 21 is close to the median of 21 across 14 interventional studies indexed under Gangliosidoses, GM2.
Browse Gangliosidoses, GM2 studies →Azafaros B.V. is the lead sponsor of 8 studies on the registry; 4 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Cohort 1 (NPC and GM2 patients):
OR
Cohort 2 (NPC patients):
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 form. Where appropriate, assent will also be sought for participants who have not reached the age of majority.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Arms (both cohorts 1 and 2): Nizubaglustat (AZ-3102)
Drug: AZ-3102
Daily oral intake of AZ-3102 dispersible tablets
Change from baseline in treatment-emergent adverse events (TEAEs)
Incidence and severity of all Adverse Events related to study drug treatment, study discontinuation or death
Time frame: Through study completion, an average of 4 years
Change from baseline in electrocardiogram (ECG)
ECG read out Normal, Abnormal, Not Clinically Significant, Abnormal, Clinically Significant and Not Done.
Time frame: Through study completion, an average of 4 years
Change from baseline in seizures
Seizure duration (minutes) as per the seizure diary.
Time frame: Through study completion, an average of 4 years
Change from baseline in seizures
Seizure frequency (number) as per seizure diary.
Time frame: Through study completion, an average of 4 years
Maximum observed plasma concentration (Cmax)
Time frame: Baseline , Month 1 (Cohort 2 only) and Month 6
Time to Cmax (Tmax)
Time frame: Baseline, Month 1 (Cohort 2 only) and Month 6
Concentration at trough (Ctrough)
Time frame: Baseline, Month 1 (Cohort 2 only) and Month 6
Area under the plasma concentration-time curve from the time of dosing (zero) to 24 hours post-dose
Time frame: Baseline, Month 1 (Cohort 2 only) and Month 6
Change from Baseline in the concentrations of Glucosylceramide (GlcCer) C16:0; C18:0
Time frame: Baseline, Month 1 (Cohort 2 only) and Month 6
Change from Baseline in the concentrations of Neurofilament light chain (NfL)
Time frame: Through study completion, an average of 4 years
For GM2 gangliosidosis patients: Change from Baseline in the concentrations of Monosialoganglioside GM2 (GM2)
Time frame: Through study completion, an average of 4 years
For GM2 gangliosidosis patients: Change from Baseline in the concentrations of Lyso-monosialoganglioside GM2
Time frame: Through study completion, an average of 4 years
For NPC disease patients: Change from Baseline in the concentrations of N-palmitoyl-O-phosphocholine-serine (PPCS)
Time frame: Through study completion, an average of 4 years
Plan to share: No
No publications or documents are linked to this record.
Started Feb 2026 (actual). Primary completion is estimated for Jun 2027, 16 months after the start.
Since we started tracking on Sep 25, 2026, the dates moved once, on Oct 21, 2026: primary completion Apr 2030 → Jun 2027 and completion Aug 2030 → Jun 2027.
Earlier changes are on the record history on ClinicalTrials.gov ↗
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
This study is active, not recruiting, as verified in Oct 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Nizubaglustat
Azafaros B.V.