Condition: GM2 Gangliosidosis · Niemann-Pick Type C Disease · Sponsor: Azafaros B.V.
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 description comes directly from the study's public registry record.
Contact details are listed per location below or on the registry record.
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| Associação Hospitalar de Prot à Infância Dr. Raul Carneiro | Água Verde, Curitiba, Brazil | Recruiting |
| Hospital de Clinicas de Porto Alegre | Porto Alegre, Rio Grande do Sul, Brazil | Recruiting |
| Instituto Nacional de Saúde da Mulher, da Criança e do Adolescente Fernandes Figueira | Rio de Janeiro, Brazil | Recruiting |
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Source record: clinicaltrials.gov/study/NCT07399704