Clinical trials: Mitochondrial Disorder
Friedreich Ataxia
A Natural History Study to TRACK Brain and Spinal Cord Changes in Individuals with Friedreich Ataxia (TRACK-FA)
This is a natural history study prospectively investigating neuroimaging markers of disease progression in children and adults with Friedreich ataxia (FA). There will be three assessment periods (baseline, 12 and 24 months).
- Trial name
- (TRACK-FA) (NCT04349514)
- Testing
- Natural history
- Where
- 7 sites in United States, Australia and 3 more
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- Monash University
Inclusion Body Myositis, Myotonic Dystrophy 1
The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.
The planned project is an intervention study to assess the risk of falling after adaptation of an assistive gait devices in patients with the following neuromuscular diseases: Inclusion body myositis, myotonic dystrophy, limb…
- Conditions
- Inclusion Body Myositis, Myotonic Dystrophy 1, Myotonic Dystrophy 2, Facio-Scapulo-Humeral Dystrophy
- Trial name
- Risk of fall (NCT07072676)
- Testing
- Assistive gait devices combined with physiotherapy
- Where
- Germany
- Volunteers
- 30 people
- Sponsor
- LMU Klinikum
Chronic Kidney Disease, Sarcopenia
Trial of Nicotinamide Riboside and Co-enzyme Q10 in Chronic Kidney Disease
Chronic kidney disease is associated with the loss of skeletal muscle mass and function. This process detrimentally impacts mobility, functional independence, and quality of life.
- Conditions
- Chronic Kidney Disease, Sarcopenia, Frailty
- Trial name
- CoNR (NCT03579693)
- Testing
- CoQ10, Nicotinamide riboside
- Where
- United States
- Volunteers
- 26 people
- Sponsor
- University of Washington
Primary Mitochondrial Diseases
A First in Human Study to Evaluate the Safety of Infusion of MNV-BM-PLC (Autologous CD34+ Cells Enriched With Placenta Derived Allogeneic Mitochondria) in Patients With Primary Mitochondrial Diseases Associated With Mitochondrial DNA Mutation or Deletion
The study objectives are to evaluate the safety of a single intravenous (IV) infusion of autologous CD34+ cells enriched with placenta-derived allogeneic mitochondria in participant with primary mitochondrial disease associated…
- Trial name
- NCT04548843
- Testing
- Bone Marrow mobilization, Apheresis, MNV-BM-PLC infusion
- Where
- Israel
- Sponsor
- Minovia Therapeutics Ltd.
Friedreich's Ataxia
Cardiac Output and Fatigue in Friedreich's Ataxia
This is a clinical trial examining to impact of aerobic training plus omaveloxolone in FRDA. Thirty individuals with FRDA will be recruited; 20 individuals will be on omaveloxolone treatment whereas the other ten individuals will…
- Trial name
- NCT07444333
- Testing
- Aerobic Exercise
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Scott Barbuto
Friedreich Ataxia
Methylprednisolone Treatment of Friedreich Ataxia
This study will explore whether methylprednisolone treatment is safe, well-tolerated, and beneficial in patients that are diagnosed with Friedreich Ataxia (FRDA).
- Trial name
- NCT02424435
- Testing
- Methylprednisolone
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Children's Hospital of Philadelphia
Leber's Hereditary Optic Neuropathy (LHON)
Near-infrared Light-emitting Diode (NIR-LED) Therapy for Leber's Hereditary Optic Neuropathy (LHON)
The overall objective of the proposed research is to test the hypothesis that Near-infrared Light-emitting Diode (NIR-LED) therapy will stimulate mitochondrial function, attenuate oxidative stress, and improve cell survival and…
- Trial name
- LHON (NCT01389817)
- Testing
- Near-infrared light-emitting diode (NIR-LED) therapy (Med Light 630 PRO (Medical Devices Inc.))
- Where
- 3 sites in United States
- Volunteers
- 4 people, healthy volunteers welcome
- Sponsor
- Medical College of Wisconsin
Gastrointestinal Dysfunction, Mitochondrial Disorders
Gastrointestinal Dysfunction in Children Affected With Mitochondrial Disorders
Hypothesis: Many patients with underlying mitochondrial disorders have feeding problems because of poor gastrointestinal motility; feeding problems lead to growth impairment and many affected children are malnourished.
- Trial name
- NCT01137240
- Testing
- gastric emptying scan
- Where
- United States
- Volunteers
- 26 people
- Sponsor
- The University of Texas Health Science Center, Houston
Leber Hereditary Optic Neuropathy
Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year
The goal of this clinical trial is to assess the efficacy and safety of GS010 gene therapy - (lenadogene nolparvovec) in subjects with LHON due to the G11778A ND4 mitochondrial mutation with a vision loss up to one year.
- Trial name
- REFLECT (NCT03293524)
- Testing
- GS010
- Where
- 13 sites in United States, Belgium and 5 more
- Volunteers
- 98 people
- Sponsor
- GenSight Biologics
Autism Spectrum Disorders, Mitochondrial Diseases
Does Clinical Treatment of Mitochondrial Dysfunction Impact Autism Spectrum Disorder (ASD)?
In this study, 50 children between 3 and 12 years old with formally diagnosed autistic spectrum disorders (ASD) and also having significant mitochondrial dysfunction will be treated for a 3 month period with the Mitochondrial…
- Trial name
- MitoASD (NCT02348125)
- Testing
- Mitochondrial Cocktail
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Drexel University
Esophageal Cancer
Prehabilitation on Fitness, Surgical Outcomes and Mitochondria Functionality in Patients With Esophageal Cancer
The most often reported complications of patients with resectable esophageal malignancies are pulmonary (25~57%), which may cause increased intensive care unit utilization, prolonged length of hospital stay, increased mortality…
- Trial name
- NCT04673968
- Testing
- prehabilitation
- Where
- Taiwan
- Volunteers
- 160 people
- Sponsor
- Chang Gung Memorial Hospital
Leber's Hereditary Optic Neuropathy
A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for Treatment of Leber's Hereditary Optic Neuropathy
This is a Phase 2, prospective, randomized, double-masked, vehicle controlled, single-center study in approximately 12 subjects with LHON to evaluate safety, tolerability and efficacy of elamipretide (MTP-131) topical ophthalmic…
- Trial name
- NCT02693119
- Testing
- elamipretide (MTP-131) 1% topical ophthalmic solution, Vehicle topical ophthalmic solution
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Stealth BioTherapeutics Inc.
Covid19
Mitochondrial DNA and Nuclear SNPs to Predict Severity of COVID-19 Infection
In December 2019, the first people got infected with COVID-19 in Wuhan, China. Within weeks, this highly infectious disease spread all over the world.
- Trial name
- mtDNA-COVID (NCT04750330)
- Where
- 3 sites in Greece, Italy and 1 more
- Volunteers
- 394 people
- Sponsor
- Maastricht University
HIV Infections
Rosiglitazone Effect on Mitochondria and Lipoatrophy
The purpose of this study is to examine the effect of rosiglitazone on limb fat and mitochondrial indices in HIV-1-infected subjects receiving stable antiretroviral therapy that does not contain stavudine (d4T) or zidovudine…
- Trial name
- NCT00367744
- Testing
- Rosiglitazone
- Where
- 2 sites in United States
- Volunteers
- 71 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Friedreich's Ataxia, Motor Disorders
Electroencephalogram in Patients With Friedreich's Ataxia for the Study of the Structural and Functional Connectome.
Prospective, exploratory, multicenter pilot study investigating the structural and functional connectome in patients with Friedreich's Ataxia (FRDA) using high-density electroencephalogram (HD-EEG).
- Trial name
- CONNETTOMA (NCT07095062)
- Testing
- HD-EEG recordings, combined with cognitive and motor assessment
- Where
- Italy
- Volunteers
- 30 people
- Sponsor
- IRCCS Eugenio Medea
Mitochondrial NeuroGastroIntestinal Encephalopathy (MNGIE)
The Natural History Study of Mitochondrial NeuroGastroIntestinal Encephalopathy (MNGIE)
This is a multi-center natural history study of Mitochondrial NeurogastroIntestinal Encephalopathy (MNGIE). Patients will be followed over time to assess clinical symptoms.
- Trial name
- NAHIM (NCT01694953)
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Columbia University
Obesity
resVida and Fat Oxidation
There is now a general consensus that the combination of excessive energy intake and a low capacity to oxidize fat will lead to muscular fat accumulation and insulin resistance.
- Trial name
- NCT00998504
- Testing
- resVida
- Where
- Netherlands
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Maastricht University Medical Center
Friedreich Ataxia, Cardiomyopathies
Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia
The purpose of this study is to test the safety and preliminary efficacy of AAVrh.10hFXN to treat the cardiomyopathy associated with Friedreich's ataxia (FA).
- Conditions
- Friedreich Ataxia, Cardiomyopathies, Cardiac Hypertrophy, Myocardial Fibrosis
- Trial name
- NCT05302271
- Testing
- AAVrh.10hFXN, serotype rh.10 adeno-associated virus (AAV) gene transfer vector expressing the cDNA coding for human FXN, Prednisone
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Weill Medical College of Cornell University
Mitochondrial Diseases, Obesity
Muscle OXPHOS and Nutrient Homeostasis
Investigators are recruiting adults (men and women, ages 18 to 65 years, inclusive) with a confirmed genetic diagnosis of mitochondrial disease.
- Trial name
- NCT02920671
- Testing
- Tracer-enhanced oral glucose tolerance test (OGTT*), Muscle MRI, Dual energy x-ray absorptiometry (DXA)
- Where
- 2 sites in United States
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- University of Pennsylvania
Friedreich's Ataxia
Biomarkers in Friedreich's Ataxia
The purpose of this project is to characterize measures of cardiac performance and neuromuscular physiology in FA patients using novel techniques, including echocardiography and magnetic resonance imaging (MRI), metabolic…
- Trial name
- NCT02497534
- Where
- United States
- Volunteers
- 203 people, healthy volunteers welcome
- Sponsor
- University of Florida