Recruiting
Phase 2
Phase 3

MitoQ

Sponsor:

Mclean Hospital

Code:

NCT06191965

Conditions

Schizophrenia and Related Disorders

Mitochondrial Alteration

Cognitive Impairment

Eligibility Criteria

Sex: All

Age: 18 - 35

Healthy Volunteers: Not accepted

Interventions

MitoQ

Placebo

Study Details

Brief summary:

The goal of this double-blind, placebo-controlled randomized clinical trial is to test the effect of 12 weeks of orally administered MitoQ (mitoquinol mesylate) supplementation on cognition in 50 people with early phase schizophrenia-spectrum disorders (E-SSD) who have mitochondrial dysfunction (called high risk, or HR). Cognitive impairments in SSD can cause significant disability. Yet, there are no effective treatments for cognitive impairments in SSD. It has been shown that alterations in a certain type of brain cell (parvalbumin interneurons, or PVI) underlie cognitive deficits in SSD. These PVI, which fire at a fast rate, utilize high amounts of energy from the mitochondria and are highly vulnerable to oxidative stress. MitoQ is an antioxidant. Research has shown that, in mice, MitoQ can reduce oxidative stress in the mitochondria. The main question that this clinical trial aims to answer is:

• Does MitoQ supplementation, compared to placebo, improve cognition in HR patients?

Secondary questions that this clinical trial aims to answer are the following: Does MitoQ supplementation, compared to placebo:

  • Improve positive and negative symptoms of SSD in HR patients?
  • Improve functioning in HR patients?
  • Improve/normalize blood markers of mitochondrial dysfunction in HR patients?

The investigators will enroll 100 individuals with E-SSD. These enrolled participants will participate in an initial screening visit to determine if they qualify for the actual clinical trial. At the screening visit, the investigators will ask about psychiatric history to determine diagnosis; ask about medical history; do a physical exam; collect blood and urine samples; do a pregnancy test; and ask participants to bring in their current medications in their original packaging so it is known what they are taking.

After the screening visit, the investigators will invite 50 HR patients (identified with a blood test) to continue with the clinical trial. Participants who qualify for the clinical trial will be asked to:

  • Take a supplement (MitoQ or placebo) once per day for 12 weeks in addition to their usual medications.
  • Come in for a study visit every 4 weeks over the 16-week study period. At these study visits, the investigators will do a physical exam; ask about symptoms and side effects; take blood and urine samples; and ask questions about general health and well-being, quality of life, mental health, emotional health, and mood. At visits 1 (baseline) and 4 (12 weeks), participants will also take a cognitive assessment.

Conditions

Schizophrenia and Related Disorders

Mitochondrial Alteration

Cognitive Impairment

Study ID

NCT06191965

Start date

Jun 1, 2024

Status verified date

Sep, 2025

Completion date

Jan 31, 2027

Anticipated

Primary completion date

Jan 31, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18 - 35

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • Male or female aged 18 to 35 years old
  • Patients who have been diagnosed with one of the following schizophrenia-spectrum disorders: schizophreniform disorder, schizophrenia, schizoaffective disorder, unspecified psychosis.
  • Less than five years in treatment for psychosis (note that the duration of psychosis may be longer than 5 years, but this is more difficult to ascertain and therefore less reliable as an inclusion criterion).
  • PANSS score < 75
  • Ability to provide informed consent.

Exclusion Criteria:

  • Meeting DSM-5 criteria for any substance use disorder diagnosis in the past 6 months will be exclusionary EXCEPT tobacco and mild/moderate cannabis use disorder, which will be included
  • Any acute medical condition requiring actively changing treatment (e.g., autoimmune disorders, acute infections, HIV/AIDS, cancer, renal failure, hepatic dysfunction, cardiovascular disease, or abnormal thyroid findings). Individuals with chronic medical conditions that are stable will not be excluded (e.g., person with hypothyroidism who is taking thyroid hormone replacement and has TSH levels within the normal range; person with well-managed diabetes; etc.)
  • Epilepsy or another seizure disorder
  • Intellectual disability (e.g., history of IQ < 70).
  • Under legal guardianship
  • Not English speaking. The questionnaires, instruments, cognitive assessments used in this research study have not been translated, validated, or studied extensively in non-English-speaking individuals. For this reason, we will not enroll individuals who do not speak English to maintain validity in the study.
  • MitoQ allergy
  • Treatment with antioxidants: omega3 (fish oil), Vitamin E, Vitamin C, multivitamins, NAC (N-acetyl cysteine) within the last 14 days. If the treatment is taken without prescription, we will ask the patient to stop using it for at least 14 days to become eligible for the present study.
  • Children and adolescents, pregnant women, women who have the intention to become pregnant during the course of the study, and breastfeeding women are excluded from the study. This is because no MitoQ pharmacokinetic data are available in pediatric populations, pregnancy or breastfeeding.
  • Lack of safe contraception, defined as: female participants of childbearing potential, not using and not willing to continue using a medically reliable method of contraception for the entire study duration, such as oral, injectable, implantable contraceptives, or intrauterine contraceptive devices, or who are not using any other method considered sufficiently reliable by the investigator in individual cases. Female participants who are surgically sterilized/hysterectomized or post-menopausal for longer than 2 years are not considered as being of childbearing potential.
  • Enrollment of study staff, their family members, and other dependent persons

Study Design

Enrollment

100 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: MitoQ

20 mg 2 capsules once daily for 12 weeks (total daily dose 40 mg)

placebo comparator: Placebo

2 capsules (identical in appearance to MitoQ capsules) once daily for 12 weeks

Interventions

MitoQ

MitoQ is a synthetic analogue of coenzyme Q10. It is produced by the company Antipodean Pharmaceuticals and is formulated as a stable yellow powder suitable for oral formulation, prepared as capsules of white color. It is a commercial dietary supplement sold over the counter as an antioxidant, to be taken orally once or twice a day. It has subsequently been tested for various clinical conditions in humans.

1. Molecular formula: C38H47O7PS (C37H44O4P.CH3O3S)
2. CAS number: 444890-41-9 (phosphonium cation)
3. Molecular weight: 678.81
4. MitoQ capsules: The standard commercial posology is a 20 mg daily dose. All formulations are produced following Good Manufacturing Practice (GMP) standards (https://www.who.int/medicines/areas/quality\_safety/quality\_assurance/TRS986annex2.pdf?ua=1).
5. MitoQ will be administered in oral capsules provided by the manufacturer Antipodean Pharma, to be taken once daily, one hour before breakfast. The daily dose will be 2 pills, i.e., 40 mg MitoQ.

Placebo

Placebo pills in identical appearance to the MitoQ capsules will be produced and provided by Antipodean Pharma and given to the patients in the control arm two per day to be taken one hour before breakfast. The composition of the placebo is tapioca starch, microcrystalline cellulose, hypromellose, silica-colloidal anhydrous, purified water, carrageenan, and pectin.

Primary outcome measure

  • Change from baseline in the MATRICS Consensus Cognitive Battery (MCCB) composite score at week 12 [ Time Frame: Baseline and week 12 ]

Central Contacts and Locations

Central contacts

Dost Ongur, MD, PhD

6178553922dongur@mgb.org

Steve Prete, RN

6178552273sprete@mgb.org

Locations

McLean Hospital

Recruiting

Belmont, Massachusetts, United States, 02478

Contacts

Dost Ongur, MD, PhD

617-855-3922dongur@mgb.org

Ann K Shinn, MD

617-855-3053akshinn@mgb.org

More Information

Sponsor

Mclean Hospital

Last update posted

Sep 25, 2025

Last verified

Sep, 2025

Trial information was received from ClinicalTrials.gov and was last updated on 2026-10-25. This information was provided to ClinicalTrials.gov by Mclean Hospital on 2025-09-25. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.