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

Study acronym: PRISMA
StatusActive, Not Recruiting
PhasePhase 2
Started
View on ClinicalTrials.gov ↗

Record History

See every version on ClinicalTrials.gov ↗

  1. Enrollment closed, study ongoing
    • Eligibility criteria expanded, about 11 words
    • Site details revised at 3 of 3 sites
    • Study description expanded, about 2 words
  2. Re-verified, no change to tracked fieldsv5
  3. Changed Sponsor changed: Azafaros A.G. to Azafaros B.V.
  4. Site details revised at 1 of 3 sitesv4
  5. Site details revised at 1 of 3 sitesv3
  6. Trial status: Not Yet Recruiting to Recruiting
    • Start date changed from estimate of Jan 21, 2026 to Feb 4, 2026
    • Site details revised at 3 of 3 sites
    v2
  7. Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
Azafaros B.V.
6 trials monitored
See 3 trials at risk
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).
Trial Details
NCT Number NCT07399704
Lead Sponsor Azafaros B.V.
Conditions
(from sponsor)		 GM2 Gangliosidosis, Niemann-Pick Type C Disease
Related terms Gangliosidoses, GM2, Niemann-Pick Disease, Type C
Enrollment 21 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov