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...

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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
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author Smeitink, Jan
van Maanen, Rob
de Boer, Lonneke
Ruiterkamp, Gerrit
Renkema, Herma
author_facet Smeitink, Jan
van Maanen, Rob
de Boer, Lonneke
Ruiterkamp, Gerrit
Renkema, Herma
author_sort Smeitink, Jan
collection PubMed
description 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 confirmed PMD. The trial will be divided into two phases. The first phase of the study will be an adaptive pharmacokinetic (PK) study with four days of treatment, while the second phase will include randomisation of the participants and evaluating the efficacy and safety of sonlicromanol over 6 months. DISCUSSION: Effective novel therapies for treating PMDs in children are an unmet need. This study will assess the pharmacokinetics, efficacy, and safety of sonlicromanol in children with genetically confirmed PMDs, suffering from motor symptoms. TRIAL REGISTRATION: clinicaltrials.gov: NCT04846036, registered April 15, 2021. European Union Clinical Trial Register (EUDRACT number: 2020–003124-16), registered October 20, 2020. CCMO registration: NL75221.091.20, registered on October 7, 2020.
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spelling pubmed-90448352022-04-28 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”) Smeitink, Jan van Maanen, Rob de Boer, Lonneke Ruiterkamp, Gerrit Renkema, Herma BMC Neurol Study Protocol 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 confirmed PMD. The trial will be divided into two phases. The first phase of the study will be an adaptive pharmacokinetic (PK) study with four days of treatment, while the second phase will include randomisation of the participants and evaluating the efficacy and safety of sonlicromanol over 6 months. DISCUSSION: Effective novel therapies for treating PMDs in children are an unmet need. This study will assess the pharmacokinetics, efficacy, and safety of sonlicromanol in children with genetically confirmed PMDs, suffering from motor symptoms. TRIAL REGISTRATION: clinicaltrials.gov: NCT04846036, registered April 15, 2021. European Union Clinical Trial Register (EUDRACT number: 2020–003124-16), registered October 20, 2020. CCMO registration: NL75221.091.20, registered on October 7, 2020. BioMed Central 2022-04-27 /pmc/articles/PMC9044835/ /pubmed/35477351 http://dx.doi.org/10.1186/s12883-022-02685-3 Text en © The Author(s) 2022 https://creativecommons.org/licenses/by/4.0/Open AccessThis article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/ (https://creativecommons.org/licenses/by/4.0/) . The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/ (https://creativecommons.org/publicdomain/zero/1.0/) ) applies to the data made available in this article, unless otherwise stated in a credit line to the data.
spellingShingle Study Protocol
Smeitink, Jan
van Maanen, Rob
de Boer, Lonneke
Ruiterkamp, Gerrit
Renkema, Herma
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”)
title 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”)
title_full 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”)
title_fullStr 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”)
title_full_unstemmed 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”)
title_short 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”)
title_sort 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”)
topic Study Protocol
url 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
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