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NCT06451757 · ClinicalTrials.gov registry record · Phase 3

KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases

A Phase 3 study of Mitochondrial Diseases and Maternally Inherited Diabetes and Deafness (MIDD), sponsored by Khondrion.

Recruiting
Registry status
Phase 3
Development phase
220
Enrollment target
10
Study locations

NCT06451757 is a Phase 3 study of Mitochondrial Diseases and Maternally Inherited Diabetes and Deafness (MIDD) that is actively recruiting participants, run by Khondrion. The registered enrollment target is 220 participants, below the 245-participant average among 7 other Mitochondrial Diseases trials with a reported enrollment target (10% lower). The trial reports 10 study locations across 10 states.

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The verdict

NCT06451757, a Phase 3 study of Mitochondrial Diseases and Maternally Inherited Diabetes and Deafness (MIDD), is actively recruiting participants, sponsored by Khondrion.

RECRUITING
Registry status
Phase 3
Development phase
220 participants
Enrollment target
10
Study locations

Study Summary

The KHENERFIN study aims to determine whether the study medicine, sonlicromanol, is able to reduce symptoms of fatigue and the impact of fatigue on daily life, and whether sonlicromanol is able to improve physical abilities of people like balance control and lower limb skeletal muscle strength in people with mitochondrial disease. In this study, the effects of sonlicromanol are compared against a placebo, a tablet identical in appearance and taste but without the active drug. Participants take either sonlicromanol or placebo twice daily for a treatment duration of 52 weeks. In addition to these primary objectives, the study evaluates the efficacy of sonlicromanol on secondary and exploratory outcomes, as well as its safety and tolerability after one year of treatment.

Conditions Studied

Mitochondrial Diseases Maternally Inherited Diabetes and Deafness (MIDD) Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes (MELAS) Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation

Interventions

  • DRUG Placebo
  • DRUG Sonlicromanol

Study Locations (10)

Massachusetts

  • Massachusetts General Hospital - Boston

Ohio

  • Cleveland Clinic Neurological Institute Mellen Center - Cleveland

Texas

  • The University of Texas Health Science Center at Houston - Houston

Region Sjælland

  • Rigshospitalet, University of Copenhagen - Kopenhagen

Gironde

  • CHU de Bordeaux - Hôpital Pellegrin Service Gynecologie Obstetrique - Bordeaux

Paris

  • Groupe Hospitalier Pitie-Salpetriere - Charles-Foix Clinical Investigation Center Paris-Est - Paris

Other

  • Klinikum der Universität München Friedrich-Baur-Institut - München

Milano

  • Fondazione IRCCS Istituto Neurologico Carlo Besta - Milan

Trial Details

FieldValue
Enrollment Target 220 participants
Start Date 2026-02
Est. Completion 2028-09
Phase Phase 3

Sponsor

Khondrion

1 total trials

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Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT06451757

The ClinicalTrials.gov registry entry for NCT06451757 describes a study currently listed as recruiting, categorized as Phase 3. The registered enrollment target is 220 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 245-participant average among 7 other Mitochondrial Diseases trials with a reported enrollment target (10% lower). The listed sponsor is Khondrion, which has 1 total studies on file at ClinicalTrials.gov.

The record links to 4 conditions, with Mitochondrial Diseases appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.

NCT06451757 reports 10 study locations spanning 10 distinct geographic areas - top geographies include Massachusetts, Ohio, Texas.

Frequently Asked Questions

What is clinical trial NCT06451757 about?

NCT06451757 is a clinical study titled "KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases". The KHENERFIN study aims to determine whether the study medicine, sonlicromanol, is able to reduce symptoms of fatigue and the impact of fatigue on daily life, and whether sonlicromanol is able to improve physical abilities of people like balance control and lower limb skeletal muscle strength in pe...

What is the current status of trial NCT06451757?

This trial is currently recruiting. It is a Phase 3 study. The enrollment target is 220 participants. The study started on 2026-02. Estimated completion is 2028-09.

What conditions does trial NCT06451757 study?

This clinical trial studies the following conditions: Mitochondrial Diseases, Maternally Inherited Diabetes and Deafness (MIDD), Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes (MELAS), Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation.

What interventions are being tested in trial NCT06451757?

The interventions under investigation include: Placebo (DRUG), Sonlicromanol (DRUG).

Who is sponsoring clinical trial NCT06451757?

This trial is sponsored by Khondrion, which has 1 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT06451757 being conducted?

This trial has 10 study locations across Massachusetts, Ohio, Texas, Region Sjælland, Gironde. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

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Data sourced from official public datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.