A randomised placebo-controlled, double-blind phase II study to explore the safety, efficacy, and pharmacokinetics of sonlicromanol in children with genetically confirmed mitochondrial disease and motor symptoms (“KHENERGYC”)

BACKGROUND: METHODS: The KHENERGYC trial will be a phase II, randomised, double-blinded, placebo-controlled (DBPC), parallel-group study in the paediatric population (birth up to and including 17 years). The study will be recruiting 24 patients suffering from motor symptoms due to genetically confir...

Descripción completa

Detalles Bibliográficos
Autores principales: Smeitink, Jan, van Maanen, Rob, de Boer, Lonneke, Ruiterkamp, Gerrit, Renkema, Herma
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2022
Materias:
Study Protocol
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9044835/
https://www.ncbi.nlm.nih.gov/pubmed/35477351
http://dx.doi.org/10.1186/s12883-022-02685-3

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9044835/
https://www.ncbi.nlm.nih.gov/pubmed/35477351
http://dx.doi.org/10.1186/s12883-022-02685-3

Ejemplares similares