Mitochondrial Diseases

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27 Mitochondrial Diseases Trials Near You

Power is an online platform that helps thousands of Mitochondrial Diseases patients discover FDA-reviewed trials every day. Every trial we feature meets safety and ethical standards, giving patients an easy way to discover promising new treatments in the research stage.

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No Placebo
Highly Paid
Stay on Current Meds
Pivotal Trials (Near Approval)
Breakthrough Medication
This trial is testing KL1333, a new medicine, to see if it can help people with mitochondrial disease feel less tired and improve their physical abilities. The study targets people with primary mitochondrial disease because they often suffer from severe fatigue. KL1333 works by boosting energy production in cells.

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 2

180 Participants Needed

This trial is testing the safety of vatiquinone, a medication that helps cells produce energy, in people with inherited mitochondrial disease. These participants have already used vatiquinone in previous studies. The study will continue for an extended period. Vatiquinone, also known as EPI-743, is a new oral antioxidant that has been evaluated for mitochondrial diseases.
No Placebo Group
Pivotal Trial (Near Approval)

Trial Details

Trial Status:Active Not Recruiting

102 Participants Needed

PRIZM is a Phase 2b randomized, double-blind, placebo-controlled, 3-treatment, 2-period, crossover study evaluating the efficacy and safety of oral zagociguat 15 and 30 mg vs. placebo when administered daily for 12 weeks in participants with genetically and phenotypically defined MELAS.

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 2

44 Participants Needed

MitoQ for Sickle Cell Disease

Pittsburgh, Pennsylvania
MitoQ is commercially available as a dietary supplement and it has been tested as a potential drug in other diseases, but it has never been tested in patients with sickle cell disease. The goal of this research is to study if MitoQ, a molecule that works as an antioxidant by removing potentially damaging agents in a living organism, improves platelet function in patients with sickle cell disease (SCD).
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Unphased

15 Participants Needed

This is a parallel arm non-randomized dose-escalation, open-label basket exploratory phase 1 clinical trial where Mitochondrial encephalopathy, lactic acidosis, stroke-like episodes (MELAS) and Leber's hereditary optic neuropathy-Plus (LHON-Plus) participants will undergo simultaneous enrollment in two disease-based arms and receive daily oral doses of glycerol tributyrate to assess its safety and potential for efficacy using clinical, biochemical, and molecular evidence. This study will utilize a two-month baseline lead-in phase to establish and document the clinical baseline for each participant in both arms in order to compare the molecular and clinical parameters. This is clinically relevant in light of the high clinical heterogeneity among subjects affected by the same mitochondrial disease (MELAS or LHON-Plus). For ethical concerns prompted by the lack of treatment for these two intractable and progressive mitochondrial diseases, there will not be a placebo control group. Thus, each participant will act as their own control and receive oral doses of glycerol tributyrate, eliminating the need for a placebo. Considering the high clinical heterogeneity among participants affected by MELAS or LHON-Plus and some clinical divergence between MELAS and LHON-Plus, this strategy is beneficial to every enrolled participants, as each will receive the investigational drug, glycerol tributyrate. In addition, this approach will determine the subject-specific maximal optimized dose in a personalized medicine-based approach. After approval of the IRB protocol from the Institutional Review Board Data and signed consent form from all participants, this investigational basket clinical trial has three phases spanning over 20 months: * A baseline lead-in phase (2 months) to collect participant-specific baseline for clinical, biochemical, molecular and metabolic biomarkers that will be monitored throughout the subsequent dose-escalation and clinical phases. * A dose-escalation phase (6 months) to determine the participant-specific maximum tolerated dose (MTD) during which participant-specific clinical and biochemical biomarkers are collected every month. * A clinical phase at a fixed subject-specific MTD dose (12 months) to collect participant-specific clinical, biochemical, molecular and metabolic biomarkers and to perform three scheduled skin biopsies: at the outset, mid-point, and the end of this clinucal phase. We have planned for a 12-month-long clinical phase at a fixed participant-specific MTD considering the absence of reliable predictors that makes idiosyncratic disease-specific symptoms for MELAS and LHON-Plus impossible to forecast among participant for assessing the potential efficacy of glycerol tributyrate by monitoring clinical symptoms specific for each disease. During the 12-month-long time-frame, disease-specific clinical symptoms will be collected as preliminary evidence of efficacy of glycerol tributyrate using disease-specific biomarkers. Finally, discharge procedure during which the clinical investigator will record non-serious adverse events or serious adverse events for 7 or 30 days, respectively, after the last day of study participation.
No Placebo Group

Trial Details

Trial Status:Not Yet Recruiting
Trial Phase:Phase 1, 2
Age:18 - 65

24 Participants Needed

Pediatric Palliative Care for Rare Diseases

Washington, District of Columbia
The palliative care needs of family caregivers of children with rare diseases and their children are largely unmet, including the need for support to prepare for future medical decision making. This trial will test the FACE-Rare intervention to see if investigators can identify and meet those needs; and if FACE-Rare effects family caregivers' quality of life and child healthcare utilization. Finally, investigators will determine if the intersectionality of child-sex, family-race, Federal poverty level, and social connection influences family quality of life and child health care utilization longitudinally.
No Placebo Group

Trial Details

Trial Status:Not Yet Recruiting
Trial Phase:Unphased
Age:12 - 99

480 Participants Needed

This trial tests whether combining high-intensity interval training (HIIT) with Coenzyme Q10 (CoQ10) supplements can improve muscle function in patients with end-stage renal disease (ESRD) on dialysis. HIIT helps improve energy production in cells, and CoQ10 supports this process. The goal is to see if this combination can better enhance physical performance and muscle health compared to exercise alone.
Pivotal Trial (Near Approval)

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 3

156 Participants Needed

Skeletal muscle metabolic health is critical for mobility and an underrecognized target of metabolic acidosis in chronic kidney disease. Impaired muscle mitochondrial metabolism underlies poor physical endurance increasing the risk of mobility disability. The proposed project will use precise in vivo tools to study the pathophysiology of poor physical endurance in a clinical trial treating metabolic acidosis among persons living with chronic kidney disease.

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 2
Age:21 - 85

80 Participants Needed

Nanosensor for Mitochondrial Myopathy

Philadelphia, Pennsylvania
Past mitochondrial disease treatment studies have been unsuccessful in determining treatment efficacy, and a major factor has been the lack of validated biomarkers in mitochondrial myopathy (MM). There is currently a growing number of potential new treatments to be tested through MM clinical intervention trials, which has created a pressing need for quantitative biomarkers that reliably reflect MM disease severity, progression, and therapeutic response. The purpose of the study is to measure the efficacy of an electrochemical oxygen nanosensor to measure in vivo mitochondrial function in human muscle tissue, and its ability to discriminate MM patients from healthy volunteers. The data and results from this nanosensor study may contribute to current and future research, including improved diagnostic and therapeutic approaches for patients with mitochondrial disease.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 1
Age:18 - 65

96 Participants Needed

Past mitochondrial disease treatment studies have been unsuccessful in determining treatment efficacy, and a major factor has been the lack of validated biomarkers in mitochondrial myopathy (MM). There is currently a growing number of potential new treatments to be tested through MM clinical intervention trials, which has created a pressing need for quantitative biomarkers that reliably reflect MM disease severity, progression, and therapeutic response. The purpose of the study is to measure the efficacy of an electrochemical oxygen nanosensor to measure in vivo mitochondrial function in human muscle tissue, and its ability to discriminate MM patients from healthy volunteers. The data and results from this nanosensor study may contribute to current and future research, including improved diagnostic and therapeutic approaches for patients with mitochondrial disease.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 1
Age:18 - 65

24 Participants Needed

This is a multi-aim study, studying the effects of conventional exercise (measured through Cardiopulomary Exercises Testing or an in-bed pedal exercise) and passive exercise through periodic acceleration (pGz). Aim 1 will focus on the differences between primary mitochondrial disease (PMD) patients and healthy volunteers. Aim 2 is an exploratory aim, which will be studying the effects in patients admitted to the Children's Hospital of Philadelphia Pediatric Intensive Care Unit (PICU).
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 1
Age:10 - 60

90 Participants Needed

The proposed study is designed to test the hypothesis that treatment of resveratrol for 12 weeks will improve both endothelin-B receptor (aim 1) and skeletal muscle mitochondrial function (aim 2) in people with type 1 diabetes.

Trial Details

Trial Status:Recruiting
Trial Phase:Early Phase 1

24 Participants Needed

MT1621 for TK2 Deficiency

New York, New York
This is a Phase 2 prospective open-label treatment study of the safety and efficacy of doxecitine and doxribtimine in study participants with thymidine kinase 2 (TK2) deficiency who participated in the retrospective study MT-1621-101 \[NCT03701568\] or who were receiving nucleos(t)ide treatment and were approved by the Sponsor.
No Placebo Group

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 2

47 Participants Needed

N-Acetylcysteine (NAC), an anti-oxidant, will be studied to investigate the effects on brain glutathione levels, cognitive skills, motor skills, and quality of life. A group of 18 participants will take either 1800, 3600 or 5400 mg per day of N-acetylcysteine (NAC) for 3 months in this dose escalation study. The investigators want to determine first if the 3600 mg dose per day is safe and might provide some efficacy. If the 3600 mg dose is safe, then additional participants will be treated with 5400 mg per day of NAC, for up to a total of 18 participants. If the 3600 mg per day dose is unsafe, then participants will be treated with the 1800 mg per day dose. Data from this pilot study will be used to determine the most safe and effective dose of NAC for a future clinical trial.
No Placebo Group

Trial Details

Trial Status:Recruiting

18 Participants Needed

Nonalcoholic steatohepatitis (NASH) is the aggressive form of nonalcoholic fatty liver disease, which is rapidly becoming a worldwide public health problem. It is more common in the military and Veteran population compared to the general US population. NASH may progress to end-stage liver disease and primary liver cancer, and hence there is critical need for effective treatment. The goal of this clinical trial is to test whether low dose thyroid hormone administered to Veterans diagnosed with NASH can be an effective therapy mediated by improvement in breaking down fat in the mitochondria. The study will be conducted in two stages, the first stage is for proof of concept to be followed by interim analysis. If the interim analysis supports the merit for continuing the study, the clinical trial will proceed to stage 2 for continuation. This study will provide new information and strategies for treatment of NASH using low dose thyroid hormone that will be highly relevant and impactful to the health of the Veteran population.

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 2

128 Participants Needed

MitoQ for Schizophrenia

New Haven, Connecticut
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.
Stay on current meds
Pivotal Trial (Near Approval)

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 2, 3
Age:18 - 35

100 Participants Needed

This trial is testing a vitamin B3 supplement called Nicotinamide Riboside (NR) in adults who have muscle problems due to mitochondrial myopathy. The supplement aims to help their muscle cells produce more energy. Researchers hope this will improve symptoms in these patients. Nicotinamide riboside (NR), a form of vitamin B3, has been shown to boost NAD(+) levels and improve mitochondrial function.

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 2

34 Participants Needed

This trial tests a treatment using specific DNA building blocks to help children with a severe genetic disorder that affects energy production in their cells. The goal is to see if this treatment can improve their condition by restoring the function of their mitochondria.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 2
Age:1 - 60

200 Participants Needed

This trial tests a treatment using busulfan and fludarabine to prepare patients with certain genetic disorders for a bone marrow transplant. The goal is to safely replace their bone marrow with healthy donor cells by carefully monitoring drug levels. Busulfan is used to clear out bone marrow cells before the transplant, and fludarabine is being tested as a less harmful alternative to another drug.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 2
Age:< 55

100 Participants Needed

The purpose of this study is to collect data to help researchers identify factors, such as certain proteins or genetic codes, that are secreted from muscle that are associated with the beneficial effects of exercise.
No Placebo Group

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Unphased
Age:18 - 40

56 Participants Needed

Why Other Patients Applied

"My orthopedist recommended a half replacement of my right knee. I have had both hips replaced. Currently have arthritis in knee, shoulder, and thumb. I want to avoid surgery, and I'm open-minded about trying a trial before using surgery as a last resort."

HZ
Arthritis PatientAge: 78

"As a healthy volunteer, I like to participate in as many trials as I'm able to. It's a good way to help research and earn money."

IZ
Healthy Volunteer PatientAge: 38

"I have dealt with voice and vocal fold issues related to paralysis for over 12 years. This problem has negatively impacted virtually every facet of my life. I am an otherwise healthy 48 year old married father of 3 living. My youngest daughter is 12 and has never heard my real voice. I am now having breathing issues related to the paralysis as well as trouble swallowing some liquids. In my research I have seen some recent trials focused on helping people like me."

AG
Paralysis PatientAge: 50

"I've been struggling with ADHD and anxiety since I was 9 years old. I'm currently 30. I really don't like how numb the medications make me feel. And especially now, that I've lost my grandma and my aunt 8 days apart, my anxiety has been even worse. So I'm trying to find something new."

FF
ADHD PatientAge: 31

"I changed my diet in 2020 and I’ve lost 95 pounds from my highest weight (283). I am 5’3”, female, and now 188. I still have a 33 BMI. I've been doing research on alternative approaches to continue my progress, which brought me here to consider clinical trials."

WR
Obesity PatientAge: 58
The study is a dose-escalation study, phase 1. The objective of this proposed clinical trial is to evaluate the safety of mitochondrially targeted ND4 gene therapy with the adeno-associated viral vector in appropriate LHON patients.
No Placebo Group

Trial Details

Trial Status:Active Not Recruiting
Age:15+

28 Participants Needed

This trial studies the effects of pioglitazone, a medication that helps the body use insulin more effectively, in patients with type 2 diabetes who have liver conditions like NAFL or NASH. Pioglitazone is a safe and effective option to manage patients with type 2 diabetes and nonalcoholic steatohepatitis (NASH). The goal is to see if this treatment can improve liver function and control blood sugar levels. Researchers will measure various aspects of liver metabolism and insulin sensitivity before and after a few months of treatment.

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 4

60 Participants Needed

The purpose of this study is to assess whether a high quality preparation of ubiquinone (coenzyme Q10) benefits symptoms, function, and quality of life in veterans with Gulf War illness.
Pivotal Trial (Near Approval)

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 3
Age:50+

192 Participants Needed

Skeletal muscle dysfunction (sarcopenia) is an under-recognized target organ complication of CKD with substantial adverse clinical consequences of disability, hospitalization, and death. Sarcopenia in this proposal is defined by impaired metabolism and physical function associated with decreased skeletal muscle mass or function. Skeletal muscle tissue relies on mitochondria to efficiently utilize oxygen to generate ATP. Impaired mitochondrial energetics is a central mechanism of sarcopenia in CKD. The investigators propose a series of studies designed to shed light on the pathophysiology of sarcopenia in persons with CKD not treated with dialysis. Investigators will conduct a randomized-controlled intervention trial of combined resistance training and aerobic exercise vs. health education to assess changes in skeletal muscle mitochondrial function, metabolism and physical function. Investigators hypothesize that exercise improves mitochondrial function and physical function in persons with CKD. If successful, these experiments will identify novel pathophysiologic mechanisms for CKD-associated sarcopenia. The proposed study will provide useful insight into benefits associated with exercise among patients with CKD and investigate mechanisms associated with improved metabolism, muscle function and physical function in population.
No Placebo Group

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:30 - 75

32 Participants Needed

This trial involves taking small pieces of tissue from the colon to study how ulcers heal. It includes patients with inflammatory bowel disease, arthritis, and healthy individuals to compare healing processes. Researchers will examine how genes work and how cells produce energy to understand healing.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Unphased

60 Participants Needed

MitoQ for COPD

Richmond, Virginia
Cardiovascular health is a critical problem in patients with chronic obstructive pulmonary disease (COPD). Existing literature suggests oxidative stress from the mitochondria c driving some of the poor health outcomes in COPD. MitoQ is a mitochondrial-targeted antioxidant that has shown promise in improving cardiovascular outcomes in similar populations. Thus the purpose of this study is to test if MitoQ can improve cardiovascular health in COPD.

Trial Details

Trial Status:Recruiting
Age:40+

24 Participants Needed

Exercise for Enlarged Prostate

San Francisco, California
This is a single-center pilot randomized controlled trial among 68 physically "inactive" older men with lower urinary tract symptoms (LUTS) attributed to benign prostatic hyperplasia (LUTS/BPH) assessing a 12-week remote exercise intervention versus health education control.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Unphased
Age:60+
Sex:Male

68 Participants Needed

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Frequently Asked Questions

How much do Mitochondrial Diseases clinical trials pay?

Each trial will compensate patients a different amount, but $50-100 for each visit is a fairly common range for Phase 2–4 trials (Phase 1 trials often pay substantially more). Further, most trials will cover the costs of a travel to-and-from the clinic.

How do Mitochondrial Diseases clinical trials work?

After a researcher reviews your profile, they may choose to invite you in to a screening appointment, where they'll determine if you meet 100% of the eligibility requirements. If you do, you'll be sorted into one of the treatment groups, and receive your study drug. For some trials, there is a chance you'll receive a placebo. Across Mitochondrial Diseases trials 30% of clinical trials have a placebo. Typically, you'll be required to check-in with the clinic every month or so. The average trial length for Mitochondrial Diseases is 12 months.

How do I participate in a study as a "healthy volunteer"?

Not all studies recruit healthy volunteers: usually, Phase 1 studies do. Participating as a healthy volunteer means you will go to a research facility several times over a few days or weeks to receive a dose of either the test treatment or a "placebo," which is a harmless substance that helps researchers compare results. You will have routine tests during these visits, and you'll be compensated for your time and travel, with the number of appointments and details varying by study.

What does the "phase" of a clinical trial mean?

The phase of a trial reveals what stage the drug is in to get approval for a specific condition. Phase 1 trials are the trials to collect safety data in humans. Phase 2 trials are those where the drug has some data showing safety in humans, but where further human data is needed on drug effectiveness. Phase 3 trials are in the final step before approval. The drug already has data showing both safety and effectiveness. As a general rule, Phase 3 trials are more promising than Phase 2, and Phase 2 trials are more promising than phase 1.

Do I need to be insured to participate in a Mitochondrial Diseases medical study?

Clinical trials are almost always free to participants, and so do not require insurance. The only exception here are trials focused on cancer, because only a small part of the typical treatment plan is actually experimental. For these cancer trials, participants typically need insurance to cover all the non-experimental components.

What are the newest Mitochondrial Diseases clinical trials?

Most recently, we added Pediatric Palliative Care for Rare Diseases, Glycerol Tributyrate for MELAS Syndrome and Optic Neuropathy and Nanosensor for Mitochondrial Myopathy to the Power online platform.

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