Clinical trials: Mitochondrial Diseases
Cardiac Surgery
Risk of Oxygen During Cardiac Surgery Trial
The investigators will recruit and randomize 200 elective cardiac surgery patients to receive physiologic oxygenation (normoxia) or hyper-oxygenation (hyperoxia) during surgery to test the hypothesis that intraoperative…
- Trial name
- ROCS (NCT02361944)
- Testing
- Oxygen - normoxia, Oxygen - hyperoxia
- Where
- United States
- Volunteers
- 213 people
- Sponsor
- Vanderbilt University
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
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
Dyslipidemias
Effect of Vitamin D Supplementation on Exercise Adaptations in Patients on Statin Therapy
Statins along with lifestyle modifications including exercise are commonly prescribed to patients with type 2 diabetes. American diabetes association recommends using moderate-intensity statin and lifestyle therapy for patients…
- Trial name
- NCT02030041
- Testing
- Vitamin D, Simvastatin
- Where
- India
- Volunteers
- 33 people
- Sponsor
- Post Graduate Institute of Medical Education and Research, Chandigarh
Friedreich's Ataxia
A First in Human Study of RT001 in Patients With Friedreich's Ataxia
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of RT001 in patients with Friedreich's ataxia.
- Trial name
- NCT02445794
- Testing
- Low dose cohort, High dose cohort
- Where
- 2 sites in United States
- Volunteers
- 19 people
- Sponsor
- Biojiva LLC
HIV Infection, Mitochondrial Dysfunction
Switching From Efavirenz to Atazanavir/ Ritonavir in HIV-infected Subjects With Good Virologic Suppression
The purposes of this study are to evaluate if switching an antiretroviral medication from efavirenz (EFV) to atazanavir/ ritonavir (ARV/r) will, in a 96-week period, change: 1.
- Trial name
- NCT01194856
- Testing
- Atazanavir/ritonavir, Efavirenz
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- The Cleveland Clinic
Type 1 Diabetes
Effects of Acipimox on Insulin Action, Vascular Function, and Muscle Function in Type 1 Diabetes
Insulin resistance (IR) is an important contributor to increased cardiovascular disease risk in type 1 diabetes (T1D). Non-esterified fatty acid elevation is a significant contributor to IR in T1D and may be a target of…
- Trial name
- AcT1 (NCT01816165)
- Testing
- Acipimox
- Where
- United States
- Volunteers
- 28 people, healthy volunteers welcome
- Sponsor
- University of Colorado, Denver
Leber Congenital Amaurosis 10, Blindness
Extension Study to Study PQ-110-001 (NCT03140969)
Subjects completing participation in study PQ-110-001 (EudraCT 2017-000813-22 / NCT03140969) will be given the opportunity to enroll into the extension study for continued dosing if available data support current and/or future…
- Conditions
- Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders
- Trial name
- INSIGHT (NCT03913130)
- Testing
- QR-110
- Where
- 3 sites in United States, Belgium
- Volunteers
- 9 people
- Sponsor
- Laboratoires Thea
Leber's Congenital Amaurosis
Study to Evaluate QR-110 in Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene
The purpose of this study is to evaluate the safety and tolerability of QR-110 administered via intravitreal injection in subjects with LCA due to the CEP290 p.Cys998X mutation.
- Trial name
- NCT03140969
- Testing
- QR-110
- Where
- 3 sites in United States, Belgium
- Volunteers
- 11 people
- Sponsor
- Laboratoires Thea
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
Primary Mitochondrial Myopathy
A Trial to Evaluate Safety and Efficacy of Elamipretide Primary Mitochondrial Myopathy Followed by Open-Label Extension
This is a multicenter phase 3 randomized, double-blind, parallel-group, placebo-controlled trial to evaluate the safety and efficacy of daily subcutaneous injections of elamipretide in subjects with primary mitochondrial…
- Trial name
- MMPOWER-3 (NCT03323749)
- Testing
- elamipretide, elamipretide open label treatment
- Where
- 27 sites in United States, Canada and 5 more
- Volunteers
- 218 people
- Sponsor
- Stealth BioTherapeutics Inc.
Hemodialysis-Induced Symptom, Mitochondrial Diseases
Mitochondria and Chronic Kidney Disease
The overarching goal of this study is to determine the role of chronic kidney disease and the activation of the kallikrein-kinin system during hemodialysis on the development of mitochondrial dysfunction; the investigators will…
- Trial name
- MitoCKD (NCT03177798)
- Testing
- Icatibant
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Vanderbilt University Medical Center
HIV Infections, Lipodystrophy
Effectiveness of Nucleoside Supplementation or Switch to Tenofovir in Reversing Fat Loss in HIV Infected Adults
HIV lipoatrophy is a condition marked by fat loss; it occurs in many patients taking antiretroviral (ARV) therapy that includes nucleoside reverse transcriptase inhibitors (NRTIs).
- Conditions
- HIV Infections, Lipodystrophy, Metabolic Diseases, Nutrition Disorders
- Trial name
- NCT00119379
- Testing
- NucleomaxX, Tenofovir Disoproxil Fumarate
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Abdominal Obesity, Insulin Resistance
Effects of Acipimox on Mitochondrial Function in Obesity
The purpose of the study is to examine whether a medication called acipimox can improve your body's mitochondria. Mitochondria are the "power house" of the cell and make energy for your body.
- Conditions
- Abdominal Obesity, Insulin Resistance, Hypertriglyceridemia
- Trial name
- NCT01488409
- Testing
- Acipimox
- Where
- United States
- Volunteers
- 39 people
- Sponsor
- Massachusetts General Hospital
HIV/AIDS, Antiviral Toxicity
Relative Mitochondrial Toxicity of Tenofovir Alafenamide (TAF) vs. Tenofovir Disoproxil Fumarate (TDF)
Increased comorbidities such as cardiovascular disease (CVD), are emerging problems in HIV infection but the mechanisms are unclear.
- Conditions
- HIV/AIDS, Antiviral Toxicity, Antiviral Drug Adverse Reaction, Mitochondrial Alteration
- Trial name
- UCLACAREMITO (NCT03251144)
- Testing
- Switch to E/C/FTC/TAF daily
- Where
- United States
- Volunteers
- 26 people, healthy volunteers welcome
- Sponsor
- University of California, Los Angeles
Multiple Sclerosis, Fatigue
MitoQ for Fatigue in Multiple Sclerosis (MS)
The purpose of this study is to determine whether MS patients who receive Oral mitoquinone (MitoQ) have less fatigue than those receiving a placebo.
- Trial name
- MitoQ (NCT04267926)
- Testing
- 20 mg MitoQ, 40mg of MitoQ
- Where
- United States
- Volunteers
- 45 people
- Sponsor
- VA Office of Research and Development
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
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)
Mitochondrial Diseases, Drug Resistant Epilepsy
A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
This is a parallel-arm, double-blind, placebo-controlled study with a screening phase that includes a 28-day run-in phase to establish baseline seizure frequency, followed by a 24-week, randomized, placebo-controlled phase.
- Conditions
- Mitochondrial Diseases, Drug Resistant Epilepsy, Leigh Disease, Leigh Syndrome
- Trial name
- MIT-E (NCT04378075)
- Testing
- Vatiquinone
- Where
- 27 sites in United States, Canada and 7 more
- Volunteers
- 68 people
- Sponsor
- PTC Therapeutics
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)