MedTrace Logo

The KHENERGYC Study

NCT04846036 · Status: SUSPENDED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 24

Last updated 2025-01-10

No results posted yet for this study

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").

Conditions

  • Mitochondrial Diseases
  • Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation
  • MELAS
  • Subacute Necrotizing Encephalomyelopathy

Interventions

DRUG

Sonlicromanol

Oral administration of sonlicromanol twice daily

DRUG

Placebo

Oral administration of matching placebo twice daily

Sponsors & Collaborators

  • Julius Clinical

    collaborator INDUSTRY

  • ProPharma Group

    collaborator INDUSTRY

  • Europees Fonds voor Regionale Ontwikkeling (EFRO)

    collaborator UNKNOWN

  • Ardena

    collaborator UNKNOWN

  • Certara

    collaborator INDUSTRY

  • Khondrion BV

    lead INDUSTRY

Principal Investigators

  • Lonneke de Boer, MD · Radboud University Medical Center Nijmegen, Netherlands

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
0 Years
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

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

Countries

  • Netherlands

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04846036 on ClinicalTrials.gov