New hope for rare brain diseases: long-term drug trial now recruiting
NCT ID NCT07399704
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests the long-term safety and effectiveness of a daily medication called nizubaglustat in people with two rare genetic diseases that affect the brain and body. About 21 participants who were in a previous study or who have been on a similar drug (miglustat) will take nizubaglustat for up to several years. Researchers will monitor side effects, seizures, and disease markers to see if the drug helps control the condition over time.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Associação Hospitalar de Prot à Infância Dr. Raul Carneiro
RECRUITINGÁgua Verde, Curitiba, 80250-060, Brazil
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Hospital de Clinicas de Porto Alegre
RECRUITINGPorto Alegre, Rio Grande do Sul, 90035-903, Brazil
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Instituto Nacional de Saúde da Mulher, da Criança e do Adolescente Fernandes Figueira
RECRUITINGRio de Janeiro, 22250, Brazil
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Could a single DNA test solve the mystery of rare brain diseases in kids?
- Experimental drug zavesca tested for rare fatal brain diseases in infants