Mitochondrial disease

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ORPHA:68380
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11Active trials53Specialists8Treatment centers

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UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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Overview

Orphanet ↗NORD ↗

FDA & Trial Timeline

10 events
Mar 2026A Phase 2 Study to Assess the Effects of SUL-238 on High Energy Phosphates With ³¹P-MRS in Patients With Early, Untreated Parkinson's Disease

GEN İlaç ve Sağlık Ürünleri A.Ş. — PHASE2

TrialRECRUITING
Mar 2026Mitochondrial Redox Modulation in Newly Diagnosed Type 2 Diabetes: A Randomized Controlled Trial Comparing Imeglimin vs. Metformin Monotherapy

Mansoura University — PHASE4

TrialNOT YET RECRUITING
Feb 2026Mitochondrial Function After Ketamine

Sheba Medical Center

TrialNOT YET RECRUITING
Feb 2026Understanding Alpha-Synuclein Spread in Parkinson's Disease Through Blood Biomarkers and Neuroimaging

University of Pavia — NA

TrialNOT YET RECRUITING
Dec 2025Kaempferol Absorption and Pharmacokinetics Evaluation

University of Pittsburgh — NA

TrialRECRUITING
Dec 2025Metabolic and Muscle Profile in ICU Survivors

Anne-Françoise Rousseau — NA

TrialRECRUITING
Oct 2025Mitochondrial Function in Peripheral Blood Mononuclear Cells, Muscle, and Skin of Long COVID Patients

Universitair Ziekenhuis Brussel

TrialNOT YET RECRUITING
Jul 2025Mitochondrial Assessment in Critical Ill Patients in Intensive Care

University Hospital Southampton NHS Foundation Trust

TrialNOT YET RECRUITING
May 2025Elucidating Hepatic Metabolism in Non-alcoholic Fatty Liver Disease

Helsinki University Central Hospital

TrialENROLLING BY INVITATION
May 2025Innsbruck Health Promotion Program

VASCage GmbH — NA

TrialRECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

No FDA-approved treatments are currently listed for Mitochondrial disease.

11 clinical trialsare actively recruiting — trials can provide access to cutting-edge therapies.

View clinical trials →

Clinical Trials

11 recruitingView all trials with filters →
Phase 31 trial
Intermittent Versus Continuous Surface O2 During HMP of DCD Kidneys
Phase 3
Actively Recruiting
PI: Tom Darius, MD, PhD (Cliniques universitaires Saint-Luc- Université Cat) · Sites: Brussels, Woluwé-Saint-Lambert · Age: 18–99 yrs
View on ClinicalTrials.gov ↗I'm interested →
Phase 12 trials
Validation of Oxygen Nanosensor in Mitochondrial Myopathy
Phase 1
Actively Recruiting
PI: Zarazuela Zolkipli-Cunningham (Children's Hospital of Philadelphia) · Sites: Philadelphia, Pennsylvania · Age: 18–65 yrs
View on ClinicalTrials.gov ↗I'm interested →
Validation of Nanosensor Oxygen Measurement
Phase 1
Actively Recruiting
PI: Zarazuela Zolkipli-Cunningham, MBChB, MRCP (Children's Hospital of Philadelphia) · Sites: Philadelphia, Pennsylvania · Age: 18–65 yrs
View on ClinicalTrials.gov ↗I'm interested →
N/A1 trial
Digital Health Technology for People With Mitochondrial Disease
N/A
Actively Recruiting
PI: Carolyn M Sue, MBBS, PhD, FRACP (Professor and Director, Kinghorn Chair, Neurodegen) · Sites: Sydney, New South Wales · Age: 18–99 yrs
View on ClinicalTrials.gov ↗I'm interested →
Other7 trials
Mitochondrial Substrate Utilization in the Diabetic Human Heart
Actively Recruiting
PI: Malte Kelm, Prof. (Clinic for Cardiology, Pneumology and Angiology at) · Sites: Düsseldorf · Age: 20–85 yrs
View on ClinicalTrials.gov ↗I'm interested →
Neurometabolic Profile of Individuals With Primary Mitochondrial Disease
Actively Recruiting
· Sites: Philadelphia, Pennsylvania · Age: 8–75 yrs
View on ClinicalTrials.gov ↗I'm interested →
MITOMICS : a Multi-OMICS Approach for the Diagnosis of Mitochondrial Diseases
Actively Recruiting
PI: SYLVIE BANNWARTH (Centre Hospitalier Universitaire de Nice) · Sites: Nice, CHU de NICE; Brest, France +7 more
View on ClinicalTrials.gov ↗I'm interested →
The Natural History of Mitochondrial Diseases
Actively Recruiting
PI: Carolyn M Sue, MBBS (Kinghorn Chair, Neuroscience Research Australia) · Sites: Randwick, New South Wales · Age: 18–99 yrs
View on ClinicalTrials.gov ↗I'm interested →
Mitochondrial Donation: An 18 Month Outcome Study.
Actively Recruiting
· Sites: Newcastle upon Tyne, Tyne & Wear
View on ClinicalTrials.gov ↗I'm interested →
Acute Infection in Mitochondrial Disease: Metabolism, Infection and Immunity
Actively Recruiting
PI: Eliza M Gordon-Lipkin, M.D. (National Human Genome Research Institute (NHGRI)) · Sites: Bethesda, Maryland · Age: 0–115 yrs
View on ClinicalTrials.gov ↗I'm interested →
CrCest Study in Primary Mitochondrial Disease
Enrolling by Invitation
PI: Zarazuela Zolkipli Cunningham, MBChB MRCP (Children's Hospital of Phiadelphia) · Sites: Philadelphia, Pennsylvania · Age: 7–75 yrs
View on ClinicalTrials.gov ↗I'm interested →

Specialists

Showing 25 of 53View all specialists →
CF
Cheuk Wing Fung
Specialist
PI on 1 active trial10 Mitochondrial disease publications
PP
Patrick Schrauwen, PhD
Specialist
PI on 5 active trials
CP
Charalampos Tzoulis, MD, PhD
Specialist
PI on 3 active trials
SM
Steven Grinspoon, MD
BOSTON, MA
Specialist
PI on 3 active trials
TD
Thomas Klopstock, Prof. Dr.
Specialist
PI on 3 active trials
ZM
Zarazuela Zolkipli-Cunningham, MBChB, MRCP
Specialist
PI on 2 active trials
WP
William T Cade, PT, PhD
HARKER HEIGHTS, TX
Specialist
PI on 5 active trials
BP
Bret Goodpaster, PhD
Specialist
PI on 3 active trials
RM
Richard E Frye, M.D./Ph.D.
DURHAM, NC
Specialist
PI on 3 active trials
MP
Michael J Goldenthal, Ph.D.
Specialist
PI on 1 active trial
MM
Mary K Koenig, MD
Specialist
PI on 1 active trial
RM
Robert Ritch, MD
NEW YORK, NY
Specialist
PI on 3 active trials
PM
Prof Patrick F Chinnery, MD
Specialist
PI on 1 active trial
AP
Alfredo Sadun, MD, PhD
PASADENA, CA
Specialist
PI on 1 active trial
TM
Theresa A Zesiewicz, MD
TAMPA, FL
Specialist
PI on 2 active trials
PM
Prof Thomas Klopstock, MD
Specialist
PI on 1 active trial
EP
Exnevia Gomo, PhD
Specialist
PI on 1 active trial
AP
Alla A Garganeeva, Ph.D.
Specialist
PI on 1 active trial
KM
Kristine Erlandson, MD
AURORA, CO
Specialist
PI on 1 active trial
AR
Allison Webel, PhD, RN
Specialist
PI on 1 active trial
KP
Kerina Duri, Phd
Specialist
PI on 1 active trial
MM
Mathilde Latreille-Barbier, MD
Specialist
PI on 2 active trials
VM
Victor DY Beck, MS
COLUMBIA, TN
Specialist
PI on 1 active trial
LC
Leorey N Saligan, C.R.N.P.
Specialist
PI on 3 active trials
JM
Jatinder Bhardwaj, MD
SHREVEPORT, LA
Specialist
PI on 1 active trial

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center ↗

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center ↗

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program ↗

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site ↗

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site ↗

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site ↗

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine ↗

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program ↗

UCLA Health

📍 Los Angeles, CA

Travel Grants

No travel grants are currently matched to Mitochondrial disease.

Search all travel grants →NORD Financial Assistance ↗

Community

Open Mitochondrial diseaseForum →

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Latest news about Mitochondrial disease

Disease timeline:

New recruiting trial: Impacts of Nicotinamide Riboside on Functional Capacity and Muscle Physiology in Older Veterans

A new clinical trial is recruiting patients for Mitochondrial disease

New recruiting trial: Effects of High-intensity Exercise With Hypoxia and Taurine Supplementation on Metabolic and Mitochondrial Parameters

A new clinical trial is recruiting patients for Mitochondrial disease

New recruiting trial: Impact of Metabolite Supplementation to Restore Mitochondrial Dysfunction During Septic Shock: a Preclinical Study

A new clinical trial is recruiting patients for Mitochondrial disease

New recruiting trial: Idebenone for the Preventive Treatment of Migraine

A new clinical trial is recruiting patients for Mitochondrial disease

New recruiting trial: Maintaining Immune and Mitochondrial Functions in Old Adults With SAfe Nutrition.

A new clinical trial is recruiting patients for Mitochondrial disease

New recruiting trial: Study of the Link Between MASH ( Metabolic Dysfunction-Associated Steatohepatitis) and MAMs (Mitochondria-Associated Membranes ) Alteration in Patients Undergoing Bariatric Surgery - MAMBA

A new clinical trial is recruiting patients for Mitochondrial disease

New recruiting trial: Effects of an Antioxidant Supplement on Blood Vessel Health

A new clinical trial is recruiting patients for Mitochondrial disease

New recruiting trial: Circulating Tumor Mitochondrial DNA (ct-mtDNA) As a Biomarker for Biliary Tract Cancer Recurrence Surveillance

A new clinical trial is recruiting patients for Mitochondrial disease

New recruiting trial: Neurometabolic Profile of Individuals With Primary Mitochondrial Disease

A new clinical trial is recruiting patients for Mitochondrial disease

New recruiting trial: Acute Infection in Mitochondrial Disease: Metabolism, Infection and Immunity

A new clinical trial is recruiting patients for Mitochondrial disease

Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Common questions about Mitochondrial disease

Are there clinical trials for Mitochondrial disease?

Yes — 11 recruiting clinical trials are currently listed for Mitochondrial disease on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Mitochondrial disease?

25 specialists and care centers treating Mitochondrial disease are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.