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

Part of paid clinical trials in Boston, Massachusetts.

Sponsor
Khondrion BV
Study ID
NCT06451757
Phase
PHASE3
Status
Recruiting
Apply to this trial

Conditions

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

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • Sonlicromanol — DRUG
    Administration of 90 mg sonlicromanol (100 mg sonlicromanol.HCl) twice daily during 52 weeks
  • Placebo — DRUG
    Administration of 100 mg placebo twice daily during 52 weeks

Study Details

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.

Key Dates

Start date
Feb 28, 2026
Status verified
Jan 2026
Primary completion
Sep 30, 2028
Completion
Sep 30, 2028

Study Design

Enrollment
220 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Sonlicromanol (KH176)
    Administration of 90 mg sonlicromanol (equivalent to 100 mg sonlicromanol.HCl) twice daily
  • Placebo Comparator: Matching Placebo
    Administration of 100 mg matching placebo twice daily

Primary Outcome Measure

Neuro-QoL Fatigue Short Form v1 [ Time Frame: Screening, Baseline (Day 1), Weeks 13, 26, 39, 52 (End of Trial Visit) ]

Central Contacts

Locations (3)

FacilityCityStateZIPSite coordinators
Massachusetts General HospitalBostonMassachusetts02114
Erica Kelly
[email protected]
(617) 724-7726
Amel Karaa, Dr. (PRINCIPAL_INVESTIGATOR)
Cleveland Clinic Neurological Institute Mellen CenterClevelandOhio44195
Jamie Jobson
[email protected]
Sumit Parikh, Dr. (PRINCIPAL_INVESTIGATOR)
The University of Texas Health Science Center at HoustonHoustonTexas77030
Lindsey Miller
[email protected]
(713) 500-7143
Antoinette Bernal Tent
[email protected]
(713) 500-6991
Mary Koenig, Dr. (PRINCIPAL_INVESTIGATOR)
Sam Nicholas Russo, Dr. (SUB_INVESTIGATOR)

Find similar trials in Boston, MA

By research site
Massachusetts General Hospital· Boston, MACleveland Clinic Neurological Institute Mellen Center· Cleveland, OHThe University of Texas Health Science Center at Houston· Houston, TX

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