Clinical trials: Mitochondrial Metabolism
Chronic Kidney Diseases
Effects of GLP1-RA on Ectopic Fat Deposition in Chronic Kidney Disease
Chronic kidney disease (CKD) is a burden of morbidity and mortality. Increased protein breakdown in skeletal muscle (wasting) and ectopic fat deposition are important determinants of poor clinical outcome in patient with CKD.
- Trial name
- GLIMP (NCT05254418)
- Testing
- dulaglutide injection
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Vanderbilt University Medical Center
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Mitochondrial Diseases, Mitochondrial Myopathies
Promoting Resilience in Stress Management (PRISM) and Clinical-focused Narrative (CFN) Pilot in Adults With Primary Mitochondrial Disease (PMD).
The goal of this study is to find the best way to help people with primary mitochondrial disease deal with the stress of their condition, and to help these people be better able to "bounce back," or be resilient.
- Conditions
- Mitochondrial Diseases, Mitochondrial Myopathies, Mitochondrial Encephalomyopathies, MELAS
- Trial name
- NCT06051448
- Testing
- Promoting Resilience in Stress Management (PRISM), Clinical-focused narrative (CFN)
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Children's Hospital of Philadelphia
Obesity & Overweight, Healthy Adult
Vitis Intervention Trial in Substrate Metabolism
Obesity is associated with reduced metabolic flexibility (MetFlex), the capacity to shift between carbohydrate and lipid oxidation in response to nutrient availability.
- Trial name
- VITIS (NCT07800988)
- Testing
- Freeze-Dried Whole Grape Powder
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- University of Alabama at Birmingham
Leber Congenital Amaurosis 10, Blindness
A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)
The purpose of this double-masked, randomized, controlled, multiple-dose study is to evaluate the efficacy, safety, tolerability and systemic exposure of sepofarsen (QR-110) administered via intravitreal injection in subjects…
- Conditions
- Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders
- Trial name
- ILLUMINATE (NCT03913143)
- Testing
- sepofarsen
- Where
- 14 sites in United States, Belgium and 7 more
- Volunteers
- 36 people
- Sponsor
- ProQR Therapeutics
Leber's Hereditary Optic Neuropathy (LHON)
Study to Assess the Efficacy and Safety of Raxone in LHON Patients
LEROS is an open-label interventional Phase IV study, designed to further assess the efficacy and safety of Raxone® in the long-term treatment of LHON patients.
- Trial name
- LEROS (NCT02774005)
- Testing
- Idebenone
- Where
- 29 sites in United States, Austria and 8 more
- Volunteers
- 199 people
- Sponsor
- Santhera Pharmaceuticals
Leber Hereditary Optic Neuropathy
Safety Evaluation of Gene Therapy in Leber Hereditary Optic Neuropathy (LHON) Patients
The purpose of this study is to evaluate the safety and tolerability profile of ascending doses of GS010 in Leber Hereditary Optic Neuropathy (LHON) patients.
- Trial name
- NCT02064569
- Testing
- GS010
- Where
- France
- Volunteers
- 19 people
- Sponsor
- GenSight Biologics
Inherited Mitochondrial Disease, Including Leigh Syndrome
Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease
To evaluate safety, tolerability and efficacy of cysteamine bitartrate delayed-release capsules (RP103) administered at a target maintenance dose of 1.3 g/m²/day in two divided doses, every 12 hours, for up to 6 months in…
- Trial name
- MITO-001 (NCT02023866)
- Testing
- Cysteamine Bitartrate
- Where
- 5 sites in United States
- Volunteers
- 36 people
- Sponsor
- Amgen
Polycystic Ovary Syndrome
Involvement of Reticulum Endoplasmic Stress in the Physiopathology of Polycystic Ovary Syndrome
The main objective of the present project is to evaluate the relevance of reticulum stress in the pathogenesis of polycystic ovary syndrome (PCOS), focusing particularly on the underlying mechanisms of insulin resistance, which…
- Trial name
- PI12/1984 (NCT02302326)
- Testing
- Metformin, Myo-inositol + folic acid
- Where
- Spain
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Fundación para el Fomento de la Investigación Sanitaria y Biomédica de la Comunitat Valenciana
Friedreich Ataxia
A Study to Learn How BIIB141 (Omaveloxolone) Affects the Health of Participants With Friedrich's Ataxia Who Took it During Pregnancy and/or During Breastfeeding and About the Health of Their Babies
In this study, researchers will learn more about the safety of BIIB141, also known as omaveloxolone or SKYCLARYS. This is a drug available for doctors to prescribe for people with Friedrich's Ataxia, also known as FA.
- Trial name
- NCT06628687
- Testing
- Omaveloxolone
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Biogen
Mitochondrial Diseases, Mitochondrial Myopathies
Nicotinamide Riboside and Mitochondrial Biogenesis
Mitochondria are important parts of the cell that are responsible for producing energy. The amount of energy they produce depends on how much energy the body needs to function and this energy production can be severely impaired…
- Conditions
- Mitochondrial Diseases, Mitochondrial Myopathies, Progressive External Ophthalmoplegia, Progressive Ophthalmoplegia
- Trial name
- NCT03432871
- Testing
- Nicotinamide Riboside
- Where
- United Kingdom
- Volunteers
- 13 people
- Sponsor
- Cambridge University Hospitals NHS Foundation Trust
Lactic Acidosis, Metformin
Study On the Role of Mitochondrial Dysfunction in the Pathogenesis of Metformin-associated Lactic Acidosis
Metformin is the first line drug of choice for the treatment of type II diabetes. Lactic acidosis can develop as a side effect, especially when renal failure leads to drug accumulation.
- Trial name
- NCT00942123
- Where
- Italy
- Volunteers
- 10 people
- Sponsor
- Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
PolG, Primary Mitochondrial Myopathy
Mitochondrial Video Assessment Source Material Study
Source material collection of videos to develop the Mitochondrial Video Assessment (MVA) for patients with mitochondrial myopathies.
- Trial name
- NCT06180421
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- The Emmes Company, LLC
Heart Failure, Diabetes Mellitus
Metabolic Determinants of Cardiac Function
Assessment of cardiac energy metabolism in patients with impaired glucose tolerance
- Trial name
- NCT03386864
- Where
- Germany
- Volunteers
- 400 people
- Sponsor
- German Diabetes Center
Friedreich's Ataxia
A Study Investigating the Long-term Safety and Efficacy of Deferiprone in Patients With Friedreich's Ataxia
The primary objective of this study is to evaluate the long-term safety and tolerability of deferiprone in subjects with Friedreich's ataxia (FRDA).
- Trial name
- NCT00897221
- Testing
- Deferiprone oral solution 100mg/mL, Deferiprone oral solution 100 mg/mL
- Where
- 4 sites in Belgium, France and 2 more
- Volunteers
- 36 people
- Sponsor
- ApoPharma
Obese, Growth Hormone Secretion Abnormality
Growth Hormone Treatment on Phosphocreatine Recovery in Obesity
Obesity is associated with reduced growth hormone (GH) secretion. Reduced GH secretion in obesity is associated with increased cardiovascular disease risk.
- Trial name
- NCT01421589
- Testing
- Growth hormone treatment
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Massachusetts General Hospital
Mitochondrial Diseases
Open Label Extension to Assess Long Term Safety and Efficacy of KL1333 in Patients With Primary Mitochondrial Disease
The purpose of this study is to investigate if the study medicine, KL1333, is safe, well-tolerated and effective long-term in improving the symptoms of fatigue and impacts on daily living and functional capacity (physical…
- Trial name
- Falcon-OLE (NCT07514338)
- Testing
- Napazimone
- Where
- 23 sites in United States, Belgium and 7 more
- Volunteers
- 140 people
- Sponsor
- Pharming Technologies B.V.
Mitochondrial Disease, Metabolic Myopathy
GDF-15 as a Biomarker for Mitochondrial Disease
Mitochondrial disorders are a group of inherited disorders causing malfunctional mitochondria. Mitochondria are found in every cell of the body, and the disorders therefore give symptoms from every tissue, especially those with…
- Conditions
- Mitochondrial Disease, Metabolic Myopathy, Muscular Dystrophy
- Trial name
- GDF-15 (NCT02745938)
- Where
- Denmark
- Volunteers
- 97 people, healthy volunteers welcome
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
Mitochondrial Diseases
RG2133 (2',3',5'-Tri-O-Acetyluridine) in Mitochondrial Disease
The objective of the study is to determine the safety and tolerability of RG2133 in patients with Mitochondrial Disease.
- Trial name
- NCT00060515
- Testing
- RG2133 (2',3',5'-tri-O-acetyluridine)
- Volunteers
- 12 people
- Sponsor
- Repligen Corporation
Heart Failure, Type2diabetes
Mitochondrial Substrate Utilization in the Diabetic Human Heart
Diabetes can lead to heart failure independently, but the underlying causes remain incompletely understood. The main aim of this study is to identify differential regulation of mitochondrial substrate utilization and complex…
- Conditions
- Heart Failure, Type2diabetes, Insulin Resistance, Mitochondrial Diseases
- Trial name
- NCT05958706
- Where
- Germany
- Volunteers
- 500 people
- Sponsor
- Heinrich-Heine University, Duesseldorf