Mitochondrial DiseasePhase 3NCT07887412
Efficacy and Safety of Modafinil to Treat Fatigue in People With Mitochondrial Disease.
A Phase 3 study of Mitochondrial Disease, run by Kenneth Myers, MD at 1 site in Canada.
Trial 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. Read more about this study
- Who is it for?
- Ages 6 to 60 years
- Conditions the study is for
- Mitochondrial Disease
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 15 participants
- Where
- 1 siteCanada
- Study dates
- 1 Dec 2026to 31 Jan 2031
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- • 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)
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- 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
Who cannot join
- • 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.
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- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Dr. Kenneth Myers, MD, PhD, Associate Professor contact
Canada1 site
Researc Institue of McGill University Health Centre- Children Hospital of Montreal
Montreal, Quebec, H4A 3H9
Dr. Kenneth Myers, MD, PhD, Associate Professor, 514-934-1934 ext. 76204, myersresearch@rimuhc.ca
Kenneth Myers, MD, PhD, Associate Professor
About this study
Official title: Randomized Double-blind Placebo-controlled Cross Over Trial of Modafinil to Treat Fatigue in People With Mitochondrial Disease.
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.
Read more
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.
- Conditions
- Mitochondrial Disease
- Being tested
- Modafinil (Placebo)
- Keywords
- Mitochondrial Diseases, Fatigue, Modafinil
- Medical subject headings
- Mitochondrial Diseases, Fatigue
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Modafinil (Placebo)Drug
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)
| Study group | Role | What they receive |
|---|---|---|
| Modafinil | Experimental | Cycle 1 Modafinil vs Cycle 2 Placebo Receives: Drug: Modafinil (Placebo) |
| Placebo | Placebo comparator | Cycle 1 Placebo vs Cycle 2 Modafinil Receives: Drug: Modafinil (Placebo) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Baseline, Visit 2 (42 days) , Visit 3 (56 days) and Visit 4 (98 days)
Secondary outcomes (2)
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.
Measured: Baseline, Visit 2 (42 days) , Visit 3 (56 days) and Visit 4 (98 days)
Fatigue Scale
Measured: Baseline, Visit 2 (42 days) , Visit 3 (56 days) and Visit 4 (98 days)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Oct 2026
- Study start1 Dec 2026 (estimated)
- Primary completion31 Jan 2029 (estimated)
- Study completion31 Jan 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Mitochondrial DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Kenneth Myers, MD
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Kenneth Myers, MD, McGill University Health Centre/Research Institute of the McGill University Health Centre
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07887412
- Sponsor's protocol ID
- RIMUHC
- Status last verified
- Oct 2026
- First submitted
- 14 Oct 2026
- Last updated
- 21 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No