Clinical trials: Mitochondrial Disease
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
Mitochondrial Diseases
Phase III Trial of Coenzyme Q10 in Mitochondrial Disease
To show that oral CoQ10 is a safe and effective treatment for children with inborn errors of mitochondrial energy metabolism due to defects in specific respiratory chain (RC) complexes or mitochondrial DNA (mtDNA) mutations, and…
- Trial name
- NCT00432744
- Testing
- CoenzymeQ10
- Where
- 3 sites in United States, Canada
- Volunteers
- 24 people
- Sponsor
- University of Florida
Friedreich's Ataxia
Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia Study
The purpose of this phase 3 multi-center, open-label extension study is to evaluate the long-term safety of ACTIMMUNE® (interferon-γ 1b) in participants with Friedreich's Ataxia.
- Trial name
- STEADFAST (NCT02593773)
- Testing
- Interferon γ-1b
- Where
- 4 sites in United States
- Volunteers
- 86 people
- Sponsor
- Amgen
Friedreich's Ataxia
Safety and Pharmacology Study of VP 20629 in Adults With Friedreich's Ataxia
The objectives of the study are: To evaluate the safety and tolerability of single and multiple oral doses of VP 20629 in subjects with Friedreich's ataxia (FA).
- Trial name
- NCT01898884
- Testing
- VP 20629
- Where
- 5 sites in United States
- Volunteers
- 46 people
- Sponsor
- Shire
Mitochondrial Myopathy
Safety, Tolerability, and Efficacy of MTP-131 for the Treatment of Mitochondrial Myopathy
Phase 1/2, multi-center, randomized, double-blind, multiple ascending dose, placebo-controlled study that enrolled 36 subjects with mitochondrial myopathy associated with genetically confirmed mitochondrial disease to evaluate…
- Trial name
- MMPOWER (NCT02367014)
- Testing
- elamipretide (low dose), elamipretide (intermediate dose), elamipretide (high dose)
- Where
- 4 sites in United States
- Volunteers
- 36 people
- Sponsor
- Stealth BioTherapeutics Inc.
Propionic Acidemia, Type I and/or Type II, Methylmalonic Acidemia
Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia
The overall objective of this drug trial is to determine whether the treatment of acute hyperammonemia with N-carbamyl-L-glutamate (NCG, Carglumic acid) in propionic acidemia (PA), methylmalonic acidemia (MMA), late-onset CPS1…
- Conditions
- Propionic Acidemia, Type I and/or Type II, Methylmalonic Acidemia, Carbamoyl-Phosphate Synthase I Deficiency Disease, Ornithine Carbamoyltransferase Deficiency
- Trial name
- STO (NCT01599286)
- Testing
- Carbaglu
- Where
- 9 sites in United States
- Volunteers
- 35 people
- Sponsor
- Mendel Tuchman
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
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)
PreDiabetes, Neuropathy
Heat Shock Therapy to Improve Mitochondrial Function in Neuropathy
Sensory dysfunction as a result of peripheral nerve damage is a significant problem that leads to reduced quality of life for patients.
- Trial name
- HOTFUN (NCT05070741)
- Testing
- Heat Therapy
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- University of Kansas Medical Center
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
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)
Becker Muscular Dystrophy
(-)- Epicatechin Becker Muscular Dystrophy
This is a 48-week open-label extension of our initial proof-of-concept study (UCD0113) in patients with Becker muscular dystrophy who participated in the earlier trial.
- Trial name
- NCT03236662
- Testing
- (-)-Epicatechin
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Craig McDonald, MD
Mitochondrial Disease, Neurology
EPI-743 for Metabolism or Mitochondrial Disorders
Background: Mitochondria are the parts of cells that help produce energy. Metabolism is the process by which the body uses energy to help cells grow and reproduce.
- Conditions
- Mitochondrial Disease, Neurology, Myopathy
- Trial name
- NCT01642056
- Testing
- EPI-743
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
MItochondrial Myopathies
RTA 408 Capsules in Patients With Mitochondrial Myopathy - MOTOR
Mitochondrial myopathies are a multisystemic group of disorders that are characterized by a wide range of biochemical and genetic mitochondrial defects and variable modes of inheritance.
- Trial name
- NCT02255422
- Testing
- Omaveloxolone capsules, 2.5 mg, omaveloxolone capsules, 5 mg, omaveloxolone capsules, 10 mg
- Where
- 9 sites in United States, Denmark
- Volunteers
- 53 people
- Sponsor
- Biogen
Type 2 Diabetes, Coronary Heart Disease
Myocardial Dysfunction in Type 2 Diabetes Mellitus (T2DM)
The purpose of this study is to examine the existence of heart abnormalities in patients with diabetes and the effect of pioglitazone in correcting these abnormalities.
- Trial name
- NCT01588470
- Testing
- pioglitazone
- Volunteers
- 130 people, healthy volunteers welcome
- Sponsor
- The University of Texas Health Science Center at San Antonio
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)
Friedreich Ataxia
FRDA Investigator Initiated Study (IIS) With Elamipretide
To evaluate the safety, tolerability, and activity of Elamipretide in treating vision loss in Friedreich Ataxia (FRDA).
- Trial name
- ELViS-FA (NCT05168774)
- Testing
- Elamipretide
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Children's Hospital of Philadelphia
Inherited Mitochondrial Disease
A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
The primary objective of this study is to assess the safety of vatiquinone in participants with inherited mitochondrial disease who had prior exposure to vatiquinone in a PTC/BioElectron sponsored (previously Edison) clinical…
- Trial name
- NCT05218655
- Testing
- Vatiquinone
- Where
- 26 sites in United States, France and 5 more
- Volunteers
- 101 people
- Sponsor
- PTC Therapeutics
Inflammatory Bowel Diseases
Regulation of Mucosal Healing in Inflammatory Bowel Disease
The objective of the current study is to compare non-healing colonic ulcers in patients with inflammatory bowel disease (IBD) with iatrogenic colonic ulcers (biopsy sites) in healthy control patients and patients with rheumatoid…
- Trial name
- NCT04504136
- Testing
- Serial Biopsy
- Where
- United States
- Volunteers
- 26 people, healthy volunteers welcome
- Sponsor
- Terrence A Barrett
Parkinson's Disease
Safety and Biomarker Study of PTC-589 in Participants With Parkinson's Disease
Open-label study with 30-day run-in phase and adaptive design component to include more participants if deemed appropriate by investigators.
- Trial name
- NCT02462603
- Testing
- PTC-589
- Where
- 5 sites in United States, Germany and 1 more
- Volunteers
- 44 people
- Sponsor
- Edison Pharmaceuticals Inc