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Mitochondrial MyopathiesEX-MITO-DYS-IRNCT06080594

Exercise-mediated Rescue of Mitochondrial Dysfunctions Driving Insulin Resistance

A study testing High-intensity exercise training in people with Mitochondrial Myopathies and Mitochondrial Diseases, run by Copenhagen University Hospital, Rigshospitalet at 1 site in Denmark.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The overarching aim of this intervention study is to interrogate the interconnection between the muscle mitochondrial adaptations and the changes in muscle insulin sensitivity elicited by exercise training in individuals harbouring pathogenic mitochondrial DNA mutations associated with an insulin-resistant phenotype. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
High-intensity exercise training
Could I receive a placebo?
No, everyone receives an active treatment
How many people
11 participants
Where
1 siteDenmark
Study dates
22 Sep 2024to 28 Feb 2025
Phase
Interventional
Are there results?
None posted in the registry

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 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Known m.3243A>G mutation in the MT-TL1 gene encoding the mitochondrial leucyl-tRNA 1 gene
  • Other known mtDNA point mutations

Who cannot join

  • Use of antiarrhythmic medications or other medications which, in the opinion of the investigators, have the potential to affect outcome measures.
  • Diagnosed severe heart disease, dysregulated thyroid gland conditions, or other dysregulated endocrinopathies, or other conditions which, in the opinion of the investigators, have the potential to affect outcome measures.
  • Pregnancy

Trial locations and contacts

1 site in 1 country.

Denmark1 site
  • Rigshospitalet

    Copenhagen, Denmark, 2100

Other trials in Denmark

About this study

Official title: Exercise-mediated Rescue of Mitochondrial Derangements Driving Insulin Resistance in Humans (EX-MITO-DYS-IR)

The overarching aim of this intervention study is to interrogate the interconnection between the muscle mitochondrial adaptations and the changes in muscle insulin sensitivity elicited by exercise training in individuals harbouring pathogenic mitochondrial DNA mutations associated with an insulin-resistant phenotype.

In a within-subject parallel-group longitudinal design, participants will undergo an exercise training intervention with one leg, while the contralateral leg will serve as an inactive control. After the exercise intervention, patients will attend an experimental trial including:

  • A hyperinsulinemic-euglycemic clamp combined with measurements of femoral artery blood flow and arteriovenous difference of glucose
  • Muscle biopsy samples
Read the detailed description

Background: Peripheral insulin resistance is a major risk factor for metabolic diseases such as type 2 diabetes. Skeletal muscle accounts for the majority of insulin-stimulated glucose disposal, hence restoring insulin action in skeletal muscle is key in the prevention of type 2 diabetes. Mitochondrial dysfunction is implicated in the etiology of muscle insulin resistance. Also, as mitochondrial function is determined by its proteome quantity and quality, alterations in the muscle mitochondrial proteome may play a critical role in the pathophysiology of insulin resistance. However, insulin resistance is multifactorial in nature and whether mitochondrial derangements are a cause or a consequence of impaired insulin action is unclear. In recent years, the study of humans with genetic mutations has shown enormous potential to establish the mechanistic link between two physiological variables; indeed, if the mutation has a functional impact on one of those variables, then the direction of causality can be readily ascribed. Mitochondrial myopathies are genetic disorders of the mitochondrial respiratory chain affecting predominantly skeletal muscle. Mitochondrial myopathies are caused by pathogenic mutations in either nuclear or mitochondrial DNA (mtDNA), which ultimately lead to mitochondrial dysfunction. Although the prevalence of mtDNA mutations is just 1 in 5,000, the study of patients with mtDNA defects has the potential to provide unique information on the pathogenic role of mitochondrial derangements that is disproportionate to the rarity of affected individuals. The m.3243A>G mutation in the MT-TL1 gene encoding the mitochondrial leucyl-tRNA 1 gene is the most common mutation leading to mitochondrial myopathy in humans. The m.3243A>G mutation is associated with impaired glucose tolerance and insulin resistance in skeletal muscle. Most importantly, insulin resistance precedes impairments of β-cell function in carriers of the m.3243A>G mutation, making these patients an ideal human model to study the causative nexus between muscle mitochondrial dysfunction and insulin resistance. Exercise training is a potent stimulus to enhance muscle insulin action, improve mitochondrial function, and promote mitochondrial proteome remodeling. Accordingly, rescue of mitochondrial dysfunction has been proposed to play a role in the insulin-sensitizing effect of exercise. Yet, numerous mechanisms may contribute to the pathophysiology of insulin resistance and the beneficial effects of exercise may be linked to amelioration of multiple factors, thus challenging the interpretation of the functional significance of improved muscle mitochondrial function per se. Nevertheless, since mitochondrial dysfunction is likely the primary cause of muscle insulin resistance in carriers of the m.3243A>G mutation, prospective studies including an in-depth analysis of the mitochondrial adaptations elicited by exercise training in this cohort of patients may offer a unique opportunity to identify those mitochondrial derangements that, once rescued, drive enhancements in insulin sensitivity.

Objective: To study the effects of exercise training on muscle insulin sensitivity, muscle mitochondrial function, and the muscle mitochondrial proteome in individuals harboring pathogenic mitochondrial DNA (mtDNA) mutations associated with an insulin-resistant phenotype.

Study design: Within-subject parallel-group longitudinal study in individuals with pathogenic mtDNA mutations undergoing an exercise training intervention with one leg (contralateral leg as inactive control).

Endpoint: Differences between the trained and the untrained leg.

Being tested
High-intensity exercise training
Keywords
Mitochondrial disease, Muscle metabolism, Insulin resistance, Exercise training, Mitochondrial dysfunction
Medical subject headings
Mitochondrial Myopathies, Mitochondrial Diseases, Insulin Resistance

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • High-intensity exercise trainingBehavioural

    Also called High-intensity interval training

    Eight sessions of high-intensity interval exercise using a single-leg cycle ergometer are conducted on separate days over a 2-week period.

Study groups in NCT06080594
Study groupRoleWhat they receive
Exercise legExperimentalHigh-intensity exercise training for one leg
Receives: Behavioral: High-intensity exercise training
Control legNo interventionNo exercise training for the controlateral leg

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Skeletal muscle insulin sensitivity

    Insulin-stimulated muscle glucose uptake is determined by the hyperinsulinemic-euglycemic clamp method integrated with measurements of femoral artery blood flow and arteriovenous difference of glucose

    Measured: 90-150 minutes after initiation of a hyperinsulinemic euglycemic clamp

  2. Muscle mitochondrial respiration

    Mitochondrial O2 flux is measured by high-resolution respirometry in permeabilized fibers from muscle biopsy samples

    Measured: Baseline

  3. Muscle mitochondrial reactive oxygen species (ROS) production

    Mitochondrial H2O2 emission rates are measured by high-resolution fluorometry in permeabilized fibers from muscle biopsy samples

    Measured: Baseline

  4. Muscle mitochondrial proteome

    Mitochondrial proteome signatures are determined by mass spectrometry-based proteomics in muscle biopsy samples

    Measured: Baseline

Secondary outcomes (6)
  1. Muscle mtDNA heteroplasmy

    mtDNA mutation load is measured in muscle biopsy samples from the patients with mitochondrial myopathy

    Measured: Baseline

  2. Muscle insulin signaling

    Insulin-mediated changes in the abundance of (phosphorylated) proteins modulating insulin action are measured by immunoblotting in muscle and fat biopsy samples

    Measured: Before (baseline) and 150 minutes after initiation of the hyperinsulinemic-euglycemic clamp

  3. Muscle integrated stress response signaling proteins

    Abundance of (phosphorylated) proteins governing the integrated stress response pathway is measured by immunoblotting in muscle biopsy samples.

    Measured: Baseline

  4. Muscle integrated stress response genes

    mRNA content of genes governing the integrated stress response pathway is measured by Real-Time PCR in muscle biopsy samples.

    Measured: Baseline

  5. Muscle release of FGF21 and GDF15

    Skeletal muscle production of FGF21 and GDF15 is determined by measurements of femoral artery blood flow and arteriovenous difference of plasma FGF21 and GDF15

    Measured: Before (baseline) and 0-150 minutes after initiation of the hyperinsulinemic-euglycemic clamp

  6. Whole-body insulin sensitivity

    Whole-body insulin sensitivity is determined by the hyperinsulinemic-euglycemic clamp method

    Measured: 90-150 minutes after initiation of a hyperinsulinemic euglycemic clamp

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted12 Oct 2023
  2. Study start22 Sep 2024
  3. Primary completion28 Feb 2025
  4. Study completion28 Feb 2025

Resources

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 N/A
Primary purpose
Basic science
Allocation
Randomised
Intervention model
Parallel groups
Model description
In a within-subject parallel-group longitudinal design, participants sustain an exercise training intervention with one leg, while the contralateral leg serves as an inactive control.
Masking
None (open label)
Enrolment
11 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06080594
Sponsor's protocol ID
EX-MITO-DYS-IR
Acronym
EX-MITO-DYS-IR
Status last verified
Jun 2025
First submitted
29 Sep 2023
Last updated
22 Jun 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • DeFronzo RA, Ferrannini E. Insulin resistance. A multifaceted syndrome responsible for NIDDM, obesity, hypertension, dyslipidemia, and atherosclerotic cardiovascular disease. Diabetes Care. 1991 Mar;14(3):173-94. doi: 10.2337/diacare.14.3.173.

    PubMed 2044434
  • DeFronzo RA, Simonson D, Ferrannini E. Hepatic and peripheral insulin resistance: a common feature of type 2 (non-insulin-dependent) and type 1 (insulin-dependent) diabetes mellitus. Diabetologia. 1982 Oct;23(4):313-9. doi: 10.1007/BF00253736.

    PubMed 6754515
  • DeFronzo RA, Gunnarsson R, Bjorkman O, Olsson M, Wahren J. Effects of insulin on peripheral and splanchnic glucose metabolism in noninsulin-dependent (type II) diabetes mellitus. J Clin Invest. 1985 Jul;76(1):149-55. doi: 10.1172/JCI111938.

    PubMed 3894418
  • Diaz-Vegas A, Sanchez-Aguilera P, Krycer JR, Morales PE, Monsalves-Alvarez M, Cifuentes M, Rothermel BA, Lavandero S. Is Mitochondrial Dysfunction a Common Root of Noncommunicable Chronic Diseases? Endocr Rev. 2020 Jun 1;41(3):bnaa005. doi: 10.1210/endrev/bnaa005.

    PubMed 32179913
  • Hesselink MK, Schrauwen-Hinderling V, Schrauwen P. Skeletal muscle mitochondria as a target to prevent or treat type 2 diabetes mellitus. Nat Rev Endocrinol. 2016 Nov;12(11):633-645. doi: 10.1038/nrendo.2016.104. Epub 2016 Jul 22.

    PubMed 27448057
  • Parish R, Petersen KF. Mitochondrial dysfunction and type 2 diabetes. Curr Diab Rep. 2005 Jun;5(3):177-83. doi: 10.1007/s11892-005-0006-3.

    PubMed 15929863
  • Zabielski P, Lanza IR, Gopala S, Heppelmann CJ, Bergen HR 3rd, Dasari S, Nair KS. Altered Skeletal Muscle Mitochondrial Proteome As the Basis of Disruption of Mitochondrial Function in Diabetic Mice. Diabetes. 2016 Mar;65(3):561-73. doi: 10.2337/db15-0823. Epub 2015 Dec 30.

    PubMed 26718503
  • Petersen MC, Shulman GI. Mechanisms of Insulin Action and Insulin Resistance. Physiol Rev. 2018 Oct 1;98(4):2133-2223. doi: 10.1152/physrev.00063.2017.

    PubMed 30067154
  • O'Rahilly S. "Treasure Your Exceptions"-Studying Human Extreme Phenotypes to Illuminate Metabolic Health and Disease: The 2019 Banting Medal for Scientific Achievement Lecture. Diabetes. 2021 Jan;70(1):29-38. doi: 10.2337/dbi19-0037.

    PubMed 33355307
  • Saleheen D, Natarajan P, Armean IM, Zhao W, Rasheed A, Khetarpal SA, Won HH, Karczewski KJ, O'Donnell-Luria AH, Samocha KE, Weisburd B, Gupta N, Zaidi M, Samuel M, Imran A, Abbas S, Majeed F, Ishaq M, Akhtar S, Trindade K, Mucksavage M, Qamar N, Zaman KS, Yaqoob Z, Saghir T, Rizvi SNH, Memon A, Hayyat Mallick N, Ishaq M, Rasheed SZ, Memon FU, Mahmood K, Ahmed N, Do R, Krauss RM, MacArthur DG, Gabriel S, Lander ES, Daly MJ, Frossard P, Danesh J, Rader DJ, Kathiresan S. Human knockouts and phenotypic analysis in a cohort with a high rate of consanguinity. Nature. 2017 Apr 12;544(7649):235-239. doi: 10.1038/nature22034.

    PubMed 28406212
  • DiMauro S. Mitochondrial myopathies. Curr Opin Rheumatol. 2006 Nov;18(6):636-41. doi: 10.1097/01.bor.0000245729.17759.f2.

    PubMed 17053512
  • Gorman GS, Schaefer AM, Ng Y, Gomez N, Blakely EL, Alston CL, Feeney C, Horvath R, Yu-Wai-Man P, Chinnery PF, Taylor RW, Turnbull DM, McFarland R. Prevalence of nuclear and mitochondrial DNA mutations related to adult mitochondrial disease. Ann Neurol. 2015 May;77(5):753-9. doi: 10.1002/ana.24362. Epub 2015 Mar 28.

    PubMed 25652200
  • Elliott HR, Samuels DC, Eden JA, Relton CL, Chinnery PF. Pathogenic mitochondrial DNA mutations are common in the general population. Am J Hum Genet. 2008 Aug;83(2):254-60. doi: 10.1016/j.ajhg.2008.07.004.

    PubMed 18674747
  • Frederiksen AL, Jeppesen TD, Vissing J, Schwartz M, Kyvik KO, Schmitz O, Poulsen PL, Andersen PH. High prevalence of impaired glucose homeostasis and myopathy in asymptomatic and oligosymptomatic 3243A>G mitochondrial DNA mutation-positive subjects. J Clin Endocrinol Metab. 2009 Aug;94(8):2872-9. doi: 10.1210/jc.2009-0235. Epub 2009 May 26.

    PubMed 19470628
  • Lindroos MM, Majamaa K, Tura A, Mari A, Kalliokoski KK, Taittonen MT, Iozzo P, Nuutila P. m.3243A>G mutation in mitochondrial DNA leads to decreased insulin sensitivity in skeletal muscle and to progressive beta-cell dysfunction. Diabetes. 2009 Mar;58(3):543-9. doi: 10.2337/db08-0981. Epub 2008 Dec 10.

    PubMed 19073775

Questions about this trial

What is this clinical trial studying?

A study testing High-intensity exercise training in people with Mitochondrial Myopathies and Mitochondrial Diseases, run by Copenhagen University Hospital, Rigshospitalet at 1 site in Denmark.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 2 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 22 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Denmark. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Skeletal muscle insulin sensitivity (time frame: 90-150 minutes after initiation of a hyperinsulinemic euglycemic clamp). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06080594, which also lists 17 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Jun 2025.

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