The KHENERGYC Study

Phase 2 Suspended 24 enrolled Khondrion BV Ardena · collab Certara · collab Europees Fonds voor Regionale Ontwikkeling (EFRO) · collab Julius Clinical · collab ProPharma Group · collab

RandomizedParallel-groupQuadruple-blindTreatment

Summary

This a randomized placebo controlled, double-blind phase II study to explore the pharmacokinetics, safety and efficacy of sonlicromanol in children (from birth to 17 years) with genetically confirmed mitochondrial disease of which the gene defect is known to decrease one or more oxidative phosphorylation system enzymes and who suffer from motor symptoms ("KHENERGYC").

Timeline

Start: 2021-02-01
Primary completion: 2025-12-01
Completion: 2025-12-01

Outcome

Outcome not reported

Stopped (Business): “Strategic company decision - not related to safety concerns”

Publications

Smeitink J, van Maanen R, de Boer L, Ruiterkamp G, Renkema H. 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"). BMC Neurol. 2022 Apr 27;22(1):158. doi: 10.1186/s12883-022-02685-3.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Sonlicromanol	Small molecule	—	Oral