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Studies

Interventional Active not recruiting Phase 2

NCT07399704 on ClinicalTrials.gov

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

Official title: Open-label Study to Evaluate the Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease, With or Without Previous Administration of Miglustat

  • Sponsor: Azafaros
  • Enrollment: 21 (Estimated)
  • Study start: 2026-02-04 (Actual)
  • Primary completion: 2027-06-30 (Estimated)
  • Study completion: 2027-06-30 (Estimated)
  • Allocation: N/a
  • InterventionModel: Single Group
  • Masking: None
  • PrimaryPurpose: Treatment

Brief 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).

Conditions

  • GM2 Gangliosidosis
  • Niemann-Pick Type C Disease

Intervention / Treatment

  • Drug: AZ-3102

Primary Outcome Measures

  • Change from baseline in treatment-emergent adverse events (TEAEs) (Through study completion, an average of 4 years)
    Incidence and severity of all Adverse Events related to study drug treatment, study discontinuation or death
  • Change from baseline in electrocardiogram (ECG) (Through study completion, an average of 4 years)
    ECG read out Normal, Abnormal, Not Clinically Significant, Abnormal, Clinically Significant and Not Done.
  • Change from baseline in seizures (Through study completion, an average of 4 years)
    Seizure duration (minutes) as per the seizure diary.
  • Change from baseline in seizures (Through study completion, an average of 4 years)
    Seizure frequency (number) as per seizure diary.
  • Maximum observed plasma concentration (Cmax) (Baseline , Month 1 (Cohort 2 only) and Month 6)
  • Time to Cmax (Tmax) (Baseline, Month 1 (Cohort 2 only) and Month 6)
  • Concentration at trough (Ctrough) (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 (Baseline, Month 1 (Cohort 2 only) and Month 6)

Countries

Brazil

Locations and Investigators

  • Associação Hospitalar de Prot à Infância Dr. Raul Carneiro, Água Verde, Brazil
  • Hospital de Clinicas de Porto Alegre, Porto Alegre, Brazil
  • Instituto Nacional de Saúde da Mulher, da Criança e do Adolescente Fernandes Figueira, Rio de Janeiro, Brazil