Trial Outcomes & Findings for MitoQ for the Treatment of Metabolic Dysfunction in Asthma (NCT NCT04026711)

NCT ID: NCT04026711

Last Updated: 2025-12-11

Results Overview

change in airway reactivity, measured by change in the concentration of methacholine producing a 20 % decrease in forced expiratory volume in one second, from baseline

• Recruitment status: COMPLETED
• Study phase: PHASE1
• Target enrollment: 47 participants
• Primary outcome timeframe: Through study completion, 12 weeks
• Results posted on: 2025-12-11

Participant Flow

Recruitment period 1/1/2020-12/1/2023 Recruitment locations: two pulmonary clinics at academic medical centers in the U.S.

Run in period after screening prior to randomization 9 participants excluded 6 screen fails (2 low lung function, 3 negative methacholine, 1 unable to perform study procedures 3 withdrew (1 lack of transport, 2 research burden)

Participant milestones

Measure	MitoQ MitoQ 40 mg per day for 12 weeks	Placebo placebo daily for 12 weeks
Overall Study STARTED	20	18
Overall Study COMPLETED	18	18
Overall Study NOT COMPLETED	2	0

Reasons for withdrawal

Measure	MitoQ MitoQ 40 mg per day for 12 weeks	Placebo placebo daily for 12 weeks
Overall Study Withdrawal by Subject	1	0
Overall Study mental health crisis	1	0

Baseline Characteristics

one participant in the placebo group had poor quality test, and so could not be included in the analysis

Baseline characteristics by cohort

Measure	MitoQ n=20 Participants MitoQ 40 mg per day for 12 weeks	Placebo n=18 Participants placebo daily for 12 weeks	Total n=38 Participants Total of all reporting groups
Age, Continuous	50 years STANDARD_DEVIATION 13 • n=20 Participants	55 years STANDARD_DEVIATION 7 • n=18 Participants	52 years STANDARD_DEVIATION 11 • n=38 Participants
Sex: Female, Male Female	14 Participants n=20 Participants	15 Participants n=18 Participants	29 Participants n=38 Participants
Sex: Female, Male Male	6 Participants n=20 Participants	3 Participants n=18 Participants	9 Participants n=38 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=20 Participants	0 Participants n=18 Participants	0 Participants n=38 Participants
Race (NIH/OMB) Asian	0 Participants n=20 Participants	0 Participants n=18 Participants	0 Participants n=38 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=20 Participants	0 Participants n=18 Participants	0 Participants n=38 Participants
Race (NIH/OMB) Black or African American	5 Participants n=20 Participants	5 Participants n=18 Participants	10 Participants n=38 Participants
Race (NIH/OMB) White	14 Participants n=20 Participants	13 Participants n=18 Participants	27 Participants n=38 Participants
Race (NIH/OMB) More than one race	0 Participants n=20 Participants	0 Participants n=18 Participants	0 Participants n=38 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=20 Participants	0 Participants n=18 Participants	1 Participants n=38 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=20 Participants	0 Participants n=18 Participants	0 Participants n=38 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	20 Participants n=20 Participants	18 Participants n=18 Participants	38 Participants n=38 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=20 Participants	0 Participants n=18 Participants	0 Participants n=38 Participants
Region of Enrollment United States	20 Participants n=20 Participants	18 Participants n=18 Participants	38 Participants n=38 Participants
Asthma Control Test	16.4 total score STANDARD_DEVIATION 5.2 • n=20 Participants	16.3 total score STANDARD_DEVIATION 3.1 • n=18 Participants	16.3 total score STANDARD_DEVIATION 4.2 • n=38 Participants
Marks Asthma Quality of Life	2.19 score on questionnaire STANDARD_DEVIATION 0.98 • n=20 Participants	2.27 score on questionnaire STANDARD_DEVIATION 0.76 • n=18 Participants	2.23 score on questionnaire STANDARD_DEVIATION 0.87 • n=38 Participants
Airway reactivity to methacholine	44.5 μg methacholine n=20 Participants • one participant in the placebo group had poor quality test, and so could not be included in the analysis	113 μg methacholine n=17 Participants • one participant in the placebo group had poor quality test, and so could not be included in the analysis	110 μg methacholine n=37 Participants • one participant in the placebo group had poor quality test, and so could not be included in the analysis
FEV1	2.45 liters STANDARD_DEVIATION 0.74 • n=20 Participants	2.32 liters STANDARD_DEVIATION 0.75 • n=18 Participants	2.39 liters STANDARD_DEVIATION 0.74 • n=38 Participants
FVC	3.48 liters STANDARD_DEVIATION 1.04 • n=20 Participants	3.13 liters STANDARD_DEVIATION 0.95 • n=18 Participants	3.30 liters STANDARD_DEVIATION 1.03 • n=38 Participants
AX	37.03 cm H20/L n=20 Participants	59.06 cm H20/L n=18 Participants	42.61 cm H20/L n=38 Participants

PRIMARY outcome

Timeframe: Through study completion, 12 weeks

Population: 2 participants in the MitoQ group withdrew 4 participants in the placebo group did not perform methacholine at 12 week visit: Lung function too low (FEV1 less than 60%), and participants unable to complete testing

change in airway reactivity, measured by change in the concentration of methacholine producing a 20 % decrease in forced expiratory volume in one second, from baseline

Outcome measures

Measure	MitoQ n=18 Participants MitoQ 40 mg per day for 12 weeks	Placebo n=14 Participants placebo daily for 12 weeks
Change in Airway Reactivity	0 μg methacholine Interval -25.5 to 1.4	-0.8 μg methacholine Interval -57.0 to 0.0

SECONDARY outcome

Timeframe: Through study completion, 12 weeks

Population: 2 withdrew from mitoQ group

To collect data on asthma symptoms measured by asthma control questionnaire with units on a scale from 5-25 (the lower the score, the worse the asthma symptoms)

Outcome measures

Measure	MitoQ n=18 Participants MitoQ 40 mg per day for 12 weeks	Placebo n=18 Participants placebo daily for 12 weeks
Change in Asthma Control Test (ACT)	1 score Interval -1.0 to 2.0	1 score Interval -2.0 to 2.0

SECONDARY outcome

Timeframe: Through study completion, 12 weeks

Adherence determined from pill counts (classified as adherent if used at least 80% of predicted pills)

Outcome measures

Measure	MitoQ n=18 Participants MitoQ 40 mg per day for 12 weeks	Placebo n=18 Participants placebo daily for 12 weeks
Number of Participants With Adherence at 12 Weeks	15 Participants	12 Participants

SECONDARY outcome

Timeframe: Through study completion, 12 weeks

The Marks Asthma Quality of Life Questionnaire (Marks AQLQ). units on a scale from 1 to 5. Higher score indicates better quality of life

Outcome measures

Measure	MitoQ n=18 Participants MitoQ 40 mg per day for 12 weeks	Placebo n=18 Participants placebo daily for 12 weeks
Change in Asthma Quality of Life Questionnaire Score	-0.08 questionnaire score Interval -0.35 to 0.1	-0.05 questionnaire score Interval -0.3 to 0.15

SECONDARY outcome

Timeframe: Through study completion, 12 weeks

Population: one participant unable to complete spirometry testing at 12 week visit

Change in forced expiratory volume in one second between baseline and 12 week visit

Outcome measures

Measure	MitoQ n=18 Participants MitoQ 40 mg per day for 12 weeks	Placebo n=17 Participants placebo daily for 12 weeks
Change in FEV1	-0.01 liters Interval 0.18 to 0.24	0.03 liters Interval -0.13 to 0.14

SECONDARY outcome

Timeframe: Through study completion, 12 weeks

Population: one participant in the placebo group unable to perform lung function testing at 12 week visit

Change in forced vital capacity from baseline to 12 weeks measured in liters

Outcome measures

Measure	MitoQ n=18 Participants MitoQ 40 mg per day for 12 weeks	Placebo n=17 Participants placebo daily for 12 weeks
Change in FVC	-0.01 liters Interval -0.19 to 0.02	0.01 liters Interval -0.18 to 0.22

Adverse Events

MitoQ

Serious events: 0 serious events

Other events: 12 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 10 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	MitoQ n=20 participants at risk MitoQ 40 mg per day for 12 weeks	Placebo n=18 participants at risk placebo daily for 12 weeks
Gastrointestinal disorders abdominal pain	15.0% 3/20 • From enrollment until study end (after 12 weeks on study drug)	16.7% 3/18 • From enrollment until study end (after 12 weeks on study drug)
Skin and subcutaneous tissue disorders skin rash	20.0% 4/20 • From enrollment until study end (after 12 weeks on study drug)	11.1% 2/18 • From enrollment until study end (after 12 weeks on study drug)
Musculoskeletal and connective tissue disorders muscle pain	30.0% 6/20 • From enrollment until study end (after 12 weeks on study drug)	33.3% 6/18 • From enrollment until study end (after 12 weeks on study drug)
General disorders fatigue	5.0% 1/20 • From enrollment until study end (after 12 weeks on study drug)	5.6% 1/18 • From enrollment until study end (after 12 weeks on study drug)
General disorders dizziness	5.0% 1/20 • From enrollment until study end (after 12 weeks on study drug)	0.00% 0/18 • From enrollment until study end (after 12 weeks on study drug)
Musculoskeletal and connective tissue disorders joint pain	30.0% 6/20 • From enrollment until study end (after 12 weeks on study drug)	33.3% 6/18 • From enrollment until study end (after 12 weeks on study drug)
General disorders headache	40.0% 8/20 • From enrollment until study end (after 12 weeks on study drug)	33.3% 6/18 • From enrollment until study end (after 12 weeks on study drug)
Respiratory, thoracic and mediastinal disorders upper respiratory tract infection	20.0% 4/20 • From enrollment until study end (after 12 weeks on study drug)	11.1% 2/18 • From enrollment until study end (after 12 weeks on study drug)
Gastrointestinal disorders gastroesophageal reflux/ nausea	35.0% 7/20 • From enrollment until study end (after 12 weeks on study drug)	27.8% 5/18 • From enrollment until study end (after 12 weeks on study drug)
Gastrointestinal disorders diarrhea	20.0% 4/20 • From enrollment until study end (after 12 weeks on study drug)	27.8% 5/18 • From enrollment until study end (after 12 weeks on study drug)

Additional Information

Anne Dixon, Professor of Medicine

University of Vermont

Phone: 802 847 0000

Email: anne.dixon@uvmhealth.org

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place