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Not yet recruitingNCT07887412Updated Oct 21, 2026

Efficacy and Safety of Modafinil to Treat Fatigue in People With Mitochondrial Disease.

A Phase 3 interventional study of Modafinil (Placebo) in Mitochondrial Disease, sponsored by Kenneth Myers, MD. Not yet recruiting at 1 site in Canada. Open to participants aged 6 Years to 60 Years. Per ClinicalTrials.gov, last updated 2026-10-21.

Sponsored by Kenneth Myers, MD · Phase 3, Interventional, and Treatment

Updated Oct 21, 2026Newly registeredGo to Updates ↓
Phase
Phase 3
Study type
Interventional
Enrollment
15
Allocation
Randomized
Ages
6 Years to 60 Years
Sex
All
01

Study summary

Mitochondrial diseases are genetic disorders that affect the body's ability to produce energy. Fatigue is a common symptom that can significantly affect daily activities, physical functioning, and quality of life. This study will evaluate whether modafinil, a medication used to promote wakefulness, can improve fatigue and physical endurance in people with mitochondrial disease. The study will also assess the safety and tolerability of modafinil in this population.

Fatigue prevents patients from engaging in activities necessary to maintain a healthy body and adequate brain stimulation. By treating fatigue, we could improve the quality of life of patients with mitochondrial DNA depletion syndrome by promoting better brain stimulation.

15 participants aged 6 to 60 years who have a confirmed diagnosis of mitochondrial disease and clinically significant fatigue will be enrolled. Participants must be able to walk independently for at least 15 meters and meet the other study eligibility criteria.

This is a single-center, randomized, double-blind, placebo-controlled crossover study Participants will be randomly assigned to one of two treatment sequences. One group will receive modafinil first, followed by a placebo, while the other group will receive placebo first, followed by modafinil. Each treatment period will last 7 weeks, with a 1-week washout period between treatments. Modafinil and placebo will be administered orally according to the study dosing schedule. Participants and study investigators /research teams will not know which treatment is being received during each treatment period, only the pharmacy team is unblended and know which study drug vs placebo you are assigned in which group.

The primary outcome measure is the distance walked during the 6-minute walk test, which assesses physical endurance. Secondary outcome measures include walking speed and gait, fatigue severity, the impact of fatigue on daily functioning, and quality of life. Fatigue will be assessed using validated questionnaires appropriate for the participants' age Safety will be evaluated through physical and neurological examinations, blood pressure and heart rate measurements, electrocardiograms, blood tests, and the recording of adverse events.

The treatment period will last approximately 16 weeks per participant, with additional follow-up contact planned for up to 6 months after the participant completes the trial. The study aims to determine whether modafinil improves physical endurance and fatigue in people with mitochondrial disease and to characterize its safety in this population.

02

Conditions studied

  • Mitochondrial Disease

Keywords

  • Mitochondrial Diseases, Fatigue, Modafinil
03

In context

Mitochondrial Diseases

186 studies on the registry are indexed under Mitochondrial Diseases; 57 are open to participants now.

This study's planned enrollment of 15 is below the median of 29 across 103 interventional studies indexed under Mitochondrial Diseases.

Browse Mitochondrial Diseases studies →

Lead sponsor

Kenneth Myers, MD is the lead sponsor of 3 studies on the registry; 2 are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
6 Years to 60 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

    • Written informed consent obtained.

      • Age 6 - 60 years (Weight ≥ 20 kg).
      • Genetic diagnosis of a mitochondrial disease.
      • Clinical diagnosis of a mitochondrial disorder by a neurologist or geneticist.
      • Patient/family report that fatigue is impacting function and/or quality of life.
      • Fatigue severity scale (FSS) score > 36 (patients age 18 years or older) or PROMIS pediatric fatigue score > 60 (patients aged 6-17 years)
      • Able to walk at least 15 metres independently.
      • Have blood pressure values greater than those for the 5th percentile and less than the 95th percentile on the National High Blood Pressure Education Program guidelines for blood pressure levels for boys and girls ages 6 to 16 years
      • Able to swallow a capsule similar in size and shape to the study drug tablet
      • Negative urine drug screen (UDS) for any illicit drug, alcohol (ethanol), stimulants at screening; if positive for stimulants (prescribed for excessive sleepiness) at screening, UDS to be repeated after a washout period and before baseline
      • Females of childbearing age:
  • Negative urinary pregnancy test at screening
  • Agree to use effective contraception for the duration of the study

Exclusion criteria

Exclusion Criteria:

    • Inability of the patient or a parent or legal guardian to give informed consent for any reason.

      • Cardiac disease
      • Hepatic dysfunction
      • Life threatening and/or unstable clinical condition which in the opinion of the investigator might compromise trial completion
      • Systemic steroid therapy within 30 days
      • Known hypersensitivity or intolerance, to modafinil or related substances or to any component of the formulation.
      • Sleep apnea
      • Narcolepsy
      • Started an experimental drug in the previous 12 months
      • Received any investigational drug within 4 weeks of the baseline visit
      • Any disorder that could interfere with drug absorption, distribution, metabolism, or excretion (including previous gastrointestinal surgery)
      • Any clinically significant deviation from the normal range(s) in the physical examination or ECG findings, or clinical laboratory test results (ie, serum chemistry, hematology) at the screening or baseline visit
      • Absolute neutrophil count (ANC) below the lower limit of normal at screening (NOTE: If the ANC is below the lower limit of normal at the baseline visit, the medical monitor will be consulted for continued eligibility in the study.)
      • A history of alcohol, narcotic, or any other substance abuse
      • A pregnant or lactating/nursing female
      • Current or past history of psychosis, mania, major depressive disorder, or other clinically significant psychiatric disorders.
05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Crossover assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
15 participants (estimated)

Study arms

  • Experimental
    Modafinil

    Cycle 1 Modafinil vs Cycle 2 Placebo

    Drug: Modafinil (Placebo)

  • Placebo comparator
    Placebo

    Cycle 1 Placebo vs Cycle 2 Modafinil

    Drug: Modafinil (Placebo)

Interventions

  • DrugModafinil (Placebo)

    Formulation: Blue colour capsules, for oral administration, containing 100 mg of active modafinil or matching placebo Route(s) of administration: Oral DOSE REGIMEN Arm 1: Modafinil * Week #1: 2 caps (50 mg) daily (am) * Weeks #2 to 6: 2 caps (50 mg), bid (am and pm) * Week #7: 2 caps (50 mg) daily (am) * Week # 8: Washout Placebo * Week #9: 2 caps daily (am) * Week #10 to 14: 2 caps bid (am and pm) * Week # 15: 2 caps daily (am) OR Arm 2: Placebo * Week #1: 2 caps daily (am) * Week #2 to 6: 2 caps bid (am and pm) * Week #7: 2 caps daily (am) * Week #8: Washout Modafinil * Week #9: 2 caps (50 mg) daily (am) * Weeks #10 to 14: 2 caps (50 mg) bid (am and pm) * Week #15: 2 caps (50 mg) daily (am)

06

What researchers measure

Primary outcomes

  1. 6-Minute Walk Test (6MWT) Distance

    The 6-minute walk test measures the distance walked in a 6-minute period (in metres), and is a validated measure of exercise endurance that has been used as an outcome in previous clinical trials for mitochondrial disease.

    Time frame: Baseline, Visit 2 (42 days) , Visit 3 (56 days) and Visit 4 (98 days)

Secondary outcomes

  1. 10-Metre Walk Assessment (10 MWA) Distance

    10-metre walk with gait assessment: The first 10 metres of the 6-minute walk test will be videotaped and timed. The timed 10-metre walk is a separate outcome that is also considered a good evaluation of functional level in ambulatory individuals. Video will be analyzed by a neurologist for any changes in ataxia and gait pattern.

    Time frame: Baseline, Visit 2 (42 days) , Visit 3 (56 days) and Visit 4 (98 days)

  2. Fatigue Scale

    Time frame: Baseline, Visit 2 (42 days) , Visit 3 (56 days) and Visit 4 (98 days)

07

Study locations

1 site
  • Researc Institue of McGill University Health Centre- Children Hospital of Montreal
    Montreal, Quebec H4A 3H9, Canada
    • Dr. Kenneth Myers, MD, PhD, Associate Professor · Contact · myersresearch@rimuhc.ca · 514-934-1934
    • Kenneth Myers, MD, PhD, Associate Professor · Principal investigator
08

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

09

Updates

1 registry update since Sep 25, 2026
Registered
First appeared on the registry. No changes since
Oct 21, 2026
Show all 1 update
  1. Oct 21, 2026
    First appeared on the registry

From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗

10

Registry details

Key details

Study ID
NCT07887412
Lead sponsor
Kenneth Myers, MD
Responsible party
Kenneth Myers, MD (Division Director of Child Neurology (Division Director, Pediatric Neurology) at the Montreal Children's Hospital and an Associate Professor in the Department of Pediatrics and Department of Neurology & Neurosurgery at McGill University., McGill University Health Centre/Research Institute of the McGill University Health Centre) — Sponsor-investigator
First posted
Oct 21, 2026
Start date
Dec 1, 2026 (estimated)
Primary completion
Jan 31, 2029 (estimated)
Completion
Jan 31, 2031 (estimated)
Last update
Oct 21, 2026

Study contacts

Dr. Kenneth Myers, MD, PhD, Associate Professor
Contact
kenneth.myers@mcgill.ca
514-934-1934 ext. 76204
Research Team, PhD
Contact
myersresearch@rimuhc.ca
514-934-1934 ext. 76204

Oversight

Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is not yet recruiting, as verified in Oct 2026. You cannot join it, but the record below documents what was studied.

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