Clinical trials: Mitochondrial Disorders
Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase 2 (CPT2) Deficiency
Study of Triheptanoin for Treatment of Long-Chain Fatty Acid Oxidation Disorder
Humans eat long-chain fat in their diet and use it for energy during exercise and during periods of fasting. Patients with long-chain fatty acid oxidation disorders cannot use dietary fat for energy.
- Conditions
- Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase 2 (CPT2) Deficiency, Mitochondrial Trifunctional Protein (TFP) Deficiency, Long-chain 3 hydroxyacylCoA Dehydrogenase (LCHAD) Deficiency
- Trial name
- Triheptanoin (NCT01379625)
- Testing
- Triheptanoin
- Where
- 2 sites in United States
- Volunteers
- 32 people
- Sponsor
- Oregon Health and Science University
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
Healthy, Obesity
Mitochondrial Function in Circulating Cells and Muscle Tissue
The main goal of this project is to determine whether mitochondrial function in circulating cells is related to that measured in the muscle fibers of the same subjects.
- Conditions
- Healthy, Obesity, Insulin Sensitivity, Women
- Trial name
- MitoWomen (NCT02700698)
- Where
- 2 sites in United States
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- University of Arkansas
Friedreich's Ataxia
Patient Reported Outcomes in Friedreich's Ataxia Patients After Withdrawal From Treatment With Idebenone (PROTI)
This is a Phase IIIb Double-Blind, Randomised, Placebo-Controlled Study. The aim is to further investigate the effects of idebenone in patients with Friedreich's ataxia.
- Trial name
- PROTI (NCT01303406)
- Testing
- Idebenone
- Where
- 6 sites in Austria, Germany and 2 more
- Volunteers
- 29 people
- Sponsor
- Santhera Pharmaceuticals
Epithelial Ovarian Cancer, Peritoneal Neoplasms
Birinapant for Advanced Ovarian, Fallopian Tube, and Peritoneal Cancer
Background: Birinapant is an experimental cancer treatment drug. It removes certain proteins in cells, which helps to kill the cells.
- Conditions
- Epithelial Ovarian Cancer, Peritoneal Neoplasms, Fallopian Tube Neoplasms
- Trial name
- NCT01681368
- Testing
- Birinapant (TL32711)
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- National Cancer Institute (NCI)
Hepatic Steatosis, Bariatric Surgery Candidate
Hepatic Mitochondrial Function in Youth
Assess the impact of bariatric surgery on hepatic energy metabolism and glucose and insulin dynamics in obese youth
- Conditions
- Hepatic Steatosis, Bariatric Surgery Candidate, Adolescent Obesity, Obesity
- Trial name
- MANGO (NCT03587727)
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of Colorado, Denver
Cardiovascular Disease, Sarcopenia
Trial of Oral Glutamine on Mitochondrial Function in CKD
The primary goal of proposed investigation is to study the impact of oral glutamine supplementation on muscle mitochondrial and endothelial cell function measured mitochondrial energetics and vascular function using 31P magnetic…
- Conditions
- Cardiovascular Disease, Sarcopenia, Endothelial Dysfunction, Muscle Mitochondrial Function
- Trial name
- NCT02838979
- Testing
- First Intervention (14 days), Washout (3 weeks), Second Intervention (14 days)
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- University of Washington
Peripheral Arterial Disease, Peripheral Artery Disease
Impacts of Mitochondrial-targeted Antioxidant on Peripheral Artery Disease Patients
Peripheral artery disease (PAD) is a common cardiovascular disease, in which narrowed arteries reduce blood flow to the limbs, causing pain, immobility and in some cases amputation or death.
- Trial name
- NCT03506633
- Testing
- MitoQ
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- University of Nebraska
Friedreich Ataxia
Efficacy of EGb761 in Patients Suffering From Friedreich Ataxia
The purpose of this protocol is to determine the efficacy of EGb 761 120 mg bid versus placebo in patients suffering from Friedreich Ataxia
- Trial name
- NCT00824512
- Testing
- EGb 761 120 mg
- Where
- France
- Volunteers
- 22 people
- Sponsor
- Ipsen
Shock, Cardiovascular Diseases
Evaluation of Microcirculatory Function and Mitochondrial Respiration After Cardiovascular Surgery
This study will examine the differences in microcirculatory function and mitochondrial respiration in patients with shock after cardiovascular surgery.
- Conditions
- Shock, Cardiovascular Diseases, Critical Illness
- Trial name
- MicroRESUS (NCT05330676)
- Testing
- coronary artery bypass grafting, valve repair/replacement
- Where
- United States
- Volunteers
- 142 people
- Sponsor
- University of Pennsylvania
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.
MELAS Syndrome
Responses to Influenza Vaccine in Patients With Mitochondrial Disorders (MELAS)
This pilot clinical study evaluated the safety and metabolic responses to a licensed inactivated seasonal influenza vaccine (TIV) in mitochondrial encephalopathy, lactic acidosis, and stroke-like episodes syndrome (MELAS)…
- Trial name
- NCT01831934
- Testing
- Fluzone®
- Where
- United States
- Volunteers
- 22 people, healthy volunteers welcome
- Sponsor
- Stanford University
Primary Progressive Multiple Sclerosis
Clinical Trial of Idebenone in Primary Progressive Multiple Sclerosis (IPPoMS)
Background: Multiple sclerosis (MS) is an inflammatory disorder of the central nervous system that progressively weakens and destroys the pathways of the nervous system.
- Trial name
- IPPoMS (NCT00950248)
- Testing
- Idebenone
- Where
- United States
- Volunteers
- 85 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Leber Hereditary Optic Neuropathy
RESCUE and REVERSE Long-term Follow-up
The goal of this clinical trial is to assess the long-term safety and efficacy of GS010, a gene therapy, and assess the quality of life in subjects with LHON due to the G11778A ND4 mitochondrial mutation and who were treated in…
- Trial name
- RESTORE (NCT03406104)
- Testing
- GS010
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 62 people
- Sponsor
- GenSight Biologics
Friedreich Ataxia
A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
The primary objective of the study is to evaluate the efficacy (using the modified Friedreich Ataxia Rating Scale [mFARS]) and safety of vatiquinone in participants with Friedreich ataxia (FA).
- Trial name
- MOVE-FA (NCT04577352)
- Testing
- Vatiquinone
- Where
- 14 sites in United States, Australia and 7 more
- Volunteers
- 146 people
- Sponsor
- PTC Therapeutics
Bipolar Depression
The Use of a Mitochondrial Enhancement Treatment in Bipolar Disorder
The primary objective of this 15-week clinical trial is to test the hypothesis that treatment with two proven mitochondrial enhancers, acetyl-L-carnitine (ALCAR) and α-lipoic acid (ALA), has significantly greater efficacy than…
- Trial name
- NCT00719706
- Testing
- acetyl-l-carnitine PLUS alpha-lipoic acide
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Mclean Hospital
Friedreich's Ataxia
(+) Epicatechin to Treat Friedreich's Ataxia
This 24-week study will test the safety and effectiveness of synthetically produced (+) Epicatechin in treating patients who have Friedreich's Ataxia, a neurological disorder.
- Trial name
- NCT02660112
- Testing
- (+)-Epicatechin
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Ralitza Gavrilova
Friedreich Ataxia
Neurology Measures in FA Children
The purpose of this study is to identify ways to follow progression of Friedreich's Ataxia (FA) and be able to measure changes over time in children with FA.
- Trial name
- NCT03418740
- Where
- 3 sites in United States
- Volunteers
- 108 people
- Sponsor
- Children's Hospital of Philadelphia
MELAS, LHON
A Dose-escalating Clinical Trial With KH176
Mitochondrial Diseases are rare progressive, multi-system, often early fatal disorders affecting both children and adults. KH176 is a novel chemical entity currently under development for the treatment of inherited mitochondrial…
- Conditions
- MELAS, LHON, Leigh Syndrome, Mitochondrial Disease
- Trial name
- NCT02544217
- Testing
- KH176
- Where
- Belgium
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Khondrion BV
Cytomegalovirus Infection
CMV Antiviral Prevention Strategies in D+R-Liver Transplants ("CAPSIL")
This is a trial of preemptive therapy vs. prophylaxis for prevention of Cytomegalovirus (CMV) disease in R-D+ liver transplant patients.
- Trial name
- NCT01552369
- Testing
- Valganciclovir
- Where
- 6 sites in United States
- Volunteers
- 205 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)