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The KHENEREXT Study

NCT04604548 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 11

Last updated 2024-03-06

No results posted yet for this study

Summary

This is an open-label, multi-centre study in subjects with a genetically confirmed mitochondrial deoxyribonucleic acid (DNA) transfer ribonucleic acid (tRNA)Leu(UUR) m.3243A\>G mutation who completed study KH176-202. In the KH176-203 study subjects will be receiving KH176 100 mg BID or KH176 50 mg bid in die (BID) (as determined by the investigator based on safety / tolerability considerations) for a year, thereby ensuring continued treatment with KH176 after study KH176-202. A final follow-up visit is scheduled 4 weeks after the intake of the last dose of study medication for patients not rolling over into the compassionate use program. Primary safety data and secondary efficacy (endpoint) data will be monitored and reviewed every three months by an independent Data Safety Monitoring Board (DSMB) to evaluate potential risks and benefits.

Conditions

  • Mitochondrial Diseases
  • Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation
  • Maternally Inherited Diabetes and Deafness (MIDD)
  • Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke Like Episodes (MELAS)
  • Chronic Progressive External Ophthalmoplegia (CPEO)

Interventions

DRUG

Oral administration of 100 mg KH176 twice daily

Drug: KH176

Sponsors & Collaborators

  • Julius Clinical

    collaborator INDUSTRY

  • ProPharma Group

    collaborator INDUSTRY

  • Certara

    collaborator INDUSTRY

  • Khondrion BV

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-08-09
Primary Completion
2023-06-01
Completion
2023-06-01

Countries

  • Denmark
  • Germany
  • Netherlands
  • United Kingdom

Study Locations

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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 NCT04604548 on ClinicalTrials.gov