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

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. According to ClinicalTrials.gov, the official US trial registry.

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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.

Primary Outcome

Change from baseline compared to placebo at week 52 and at other relevant study visits of the Quality of Life in Neurological Disorders Fatigue Short Form version 1 (Neuro-QoL Fatigue - SF v1): The Neuro-QoL Fatigue SF v1 is an 8-item self-assessment questionnaire evaluating the perception of fatigue and its impact in daily life activities. Each question is scored as following: 1=Never, 2=Rarely, 3=Sometimes, 4=Often, and 5=Always. Total raw scores range from 8-40. T-scores are calculated from t

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
Khondrion

1 total trials

What NCT06451757 shows while recruiting

NCT06451757 is an interventional study that assigns participants to a tested intervention. The registered 220 participants enrollment target is mid-sized for trials with a published cap, below the 245-participant average among 7 other Mitochondrial Diseases trials with a reported enrollment target (10% lower).

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 lists 10 locations in 10 states (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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More recruiting context for NCT06451757

Source: ClinicalTrials.gov NCT06451757, the US trial registry maintained by the National Library of Medicine. NCT06451757 (mid enrollment · multi site footprint · recruiting) retrieved and formatted by PlainTrial, see methodology.