CClinicalTrials.gg
Sign In
RecruitingNCT07399704PRISMAUpdated Jun 18, 2026

A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease

A Phase 2 interventional study of AZ-3102 in GM2 Gangliosidosis and Niemann-Pick Type C Disease, sponsored by Azafaros B.V.. Recruiting at 3 sites in Brazil. Open to participants aged 12 Years and older. Per ClinicalTrials.gov, last updated 2026-06-18.

Sponsored by Azafaros B.V. · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
21
Allocation
Not applicable
Ages
12 Years and older
Sex
All
01

Study summary

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).

Read the detailed description

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:

  • Cohort 1: Patients who previously took part in Phase 2 Study AZA-001-5A2-01 (RAINBOW) and wish to continue in this open-label study
  • Cohort 2: Approximately 10 patients with NPC disease, aged ≥12 years who received full-dose Miglustat for more than 12 months, have stable or worsening disease over the 2 previous clinic visits, and who wish to stop Miglustat treatment and transition to Nizubaglustat.
02

Conditions studied

  • GM2 Gangliosidosis
  • Niemann-Pick Type C Disease

Keywords

  • Nizubaglustat
03

Who can participate

Ages eligible
12 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

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

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.
04

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
21 participants (estimated)

Study arms

  • Experimental
    All patients

    Arms (both cohorts 1 and 2): Nizubaglustat (AZ-3102)

    Drug: AZ-3102

Interventions

  • DrugAZ-3102

    Daily oral intake of AZ-3102 dispersible tablets

05

What researchers measure

Primary outcomes

  1. 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

  2. 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

  3. Change from baseline in seizures

    Seizure duration (minutes) as per the seizure diary.

    Time frame: Through study completion, an average of 4 years

  4. Change from baseline in seizures

    Seizure frequency (number) as per seizure diary.

    Time frame: Through study completion, an average of 4 years

  5. Maximum observed plasma concentration (Cmax)

    Time frame: Baseline , Month 1 (Cohort 2 only) and Month 6

  6. Time to Cmax (Tmax)

    Time frame: Baseline, Month 1 (Cohort 2 only) and Month 6

  7. Concentration at trough (Ctrough)

    Time frame: Baseline, Month 1 (Cohort 2 only) and Month 6

  8. 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

Secondary outcomes

  1. Change from Baseline in the concentrations of Glucosylceramide (GlcCer) C16:0; C18:0

    Time frame: Baseline, Month 1 (Cohort 2 only) and Month 6

  2. Change from Baseline in the concentrations of Neurofilament light chain (NfL)

    Time frame: Through study completion, an average of 4 years

  3. For GM2 gangliosidosis patients: Change from Baseline in the concentrations of Monosialoganglioside GM2 (GM2)

    Time frame: Through study completion, an average of 4 years

  4. For GM2 gangliosidosis patients: Change from Baseline in the concentrations of Lyso-monosialoganglioside GM2

    Time frame: Through study completion, an average of 4 years

  5. 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

06

Study locations

3 of 3 sites recruiting
  • Associação Hospitalar de Prot à Infância Dr. Raul Carneiro
    Água Verde, Curitiba 80250-060, Brazil
    • Daniel Almeida do Valle · Contact · daniel.valle@hpp.org.br · +55 (41) 2108-3861 - option 3
    • Daniel Almeida do Valle · Principal investigator
    Recruiting
  • Hospital de Clinicas de Porto Alegre
    Porto Alegre, Rio Grande do Sul 90035-903, Brazil
    • Roberta Souto · Contact · rsouto@hcpa.edu.br · +55 51 9985-0919
    • Roberto Giugliani, Prof. Dr. · Principal investigator
    Recruiting
  • Instituto Nacional de Saúde da Mulher, da Criança e do Adolescente Fernandes Figueira
    Rio de Janeiro, 22250, Brazil
    Recruiting
07

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

08

Registry details

Key details

Study ID
NCT07399704
Lead sponsor
Azafaros B.V.
Responsible party
Sponsor
First posted
Feb 10, 2026
Start date
Feb 4, 2026
Primary completion
Apr 15, 2030 (estimated)
Completion
Aug 7, 2030 (estimated)
Last update
Jun 18, 2026

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Interested in this study?

Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.

No contact was published for this record. The registry link below has the sponsor’s details.

Follow this study

Get an email when the registry record changes — status, dates, results — or when someone posts here.

Sign in to follow

Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.

Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Other studies in this condition

Gangliosidoses, GM2→

Other studies from this sponsor

Azafaros B.V.

Start the discussion