Clinical trials: Mitochondrial Metabolism
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
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
MELAS Syndrome, Mitochondrial Respiratory Chain Deficiencies
Safety, Tolerability and Pharmacokinetic Study of KL1333 in Healthy Male Volunteers
The purpose of this First In Human study is to investigate the safety and tolerability of KL1333 after a single oral dose and to investigate the pharmacokinetic characteristics of KL1333 after a single oral dose.
- Trial name
- NCT03056209
- Testing
- KL1333 25 mg, KL1333 50 mg, KL1333 100 mg
- Where
- South Korea
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Yungjin Pharm. Co., Ltd.
Friedreich Ataxia, Friedreich's Ataxia
A Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects With Friedreich's Ataxia
To evaluate the efficacy and safety of subcutaneous nomlabofusp in adult and pediatric subjects with Friedreich's ataxia
- Trial name
- FORWARD-FA (NCT07778836)
- Testing
- Nomlabofusp
- Where
- 4 sites in United States
- Volunteers
- 150 people
- Sponsor
- Larimar Therapeutics, Inc.
Optic Neuropathy
Biomechanics of Optic Neuropathy
The participant is being asked to be a subject in this research study because the participant may have a disorder that can cause optic nerve damage and impairment of his/her visual function which is called optic neuropathies…
- Trial name
- NCT02982499
- Testing
- magnetic resonance image (MRI), Optical Coherence Tomography (OCT)
- Where
- United States
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- University of Miami
Healthy, NAFLD (Non-Alcoholic Fatty Liver Disease)
Effect of Vitamin E on Non-Alcoholic Fatty Liver Disease
One-third of the US population has non-alcoholic fatty liver disease (NAFLD) due to obesity and ~8 million of these individuals have a progressive form of the disease, non-alcoholic steatohepatitis (NASH).
- Conditions
- Healthy, NAFLD (Non-Alcoholic Fatty Liver Disease), NASH (Non-Alcoholic Steatohepatitis)
- Trial name
- NCT02690792
- Testing
- NAFLD/NASH - Vitamin E
- Where
- United States
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- Jeffrey Browning
Peripheral Arterial Disease
Mechanisms That Produce the Leg Dysfunction of Claudication & Treatment Strategies
Intermittent claudication afflicts 5% of the US population older than 55 years of age and develops along with hardening of the arteries of the legs.
- Trial name
- NCT01970332
- Testing
- Revascularization Surgery, Supervised exercise therapy
- Where
- United States
- Volunteers
- 220 people
- Sponsor
- University of Nebraska
Friedreich Ataxia
A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
The primary objective of the study is to assess the pharmacokinetics (PK) and safety of vatiquinone administered in participants with Friedreich ataxia (FA) younger than 7 years.
- Trial name
- NCT05485987
- Testing
- Vatiquinone
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- PTC Therapeutics
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 Infections, Lipoatrophy
Study Comparing Reducing the Dose of Stavudine Versus Switching to Tenofovir in HIV-Infected Patients Receiving Antiretroviral Therapy
Background: Stavudine-containing regimens are associated with a potential for lipoatrophy and dyslipidemia. We assessed the safety and efficacy of reducing the dose of stavudine compared to switching to tenofovir or maintaining…
- Trial name
- NCT00312832
- Testing
- switching; dose reduction
- Volunteers
- 60 people
- Sponsor
- Hospital Clinic of Barcelona
Mitochondrial Myopathies, Mitochondrial DNA Mutation
Genomic Profiling of Mitochondrial Disease - Imaging Analysis for Precise Mitochondrial Medicine
This study is an observational longitudinal study involving the use of MRIs and video recordings taken at home of patients completing basic tasks.
- Conditions
- Mitochondrial Myopathies, Mitochondrial DNA Mutation, Mitochondrial Diseases, Chronic Progressive External Ophthalmoplegia With Myopathy
- Trial name
- NCT05012358
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Mayo Clinic
Friedreich's Ataxia (FA)
A Study of SGT-212 Gene Therapy in Friedreich's Ataxia
This is a phase 1b, first in-human, open-label, dose-finding study investigating the safety and tolerability of SGT-212 in participants with Friedreich's ataxia (FA).
- Trial name
- FALCON (NCT07180355)
- Testing
- SGT-212
- Where
- 3 sites in United States
- Volunteers
- 10 people
- Sponsor
- Solid Biosciences Inc.
Mitochondrial Myopathies
MABs Therapy m.3243A>G Mutation Carriers
Rationale: Mitochondrial disorders are progressive, often fatal multisystem disorders, in 20-25% of the cases caused by heteroplasmic mutations in the mitochondrial DNA (mtDNA).
- Trial name
- MABS01 (NCT05063721)
- Testing
- autologous mesoangioblasts
- Where
- Netherlands
- Volunteers
- 6 people
- Sponsor
- Maastricht University
Prediabetes
Mitochondrial-related Platelet Transcript Expression Levels in Pre-diabetic Subjects Randomized to Metformin or Placebo
Diabetes mellitus (DM) imposes an approximate 2-fold increased risk of atherothrombosis. Patients with type 2 DM have a 2- to 4-fold increase in the risk of coronary artery disease (CAD) and atherothrombotic complications.
- Trial name
- MAP-2 (NCT02682121)
- Testing
- Metformin
- Where
- United States
- Volunteers
- 55 people
- Sponsor
- University of Utah
Mucopolysaccharidosis Disorders, Hurler Syndrome
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
This single-institution, phase II study is designed to test the ability to achieve donor hematopoietic engraftment while maintaining low rates of transplant-related mortality (TRM) using busulfan- and fludarabine-based…
- Conditions
- Mucopolysaccharidosis Disorders, Hurler Syndrome, Hunter Syndrome, Maroteaux Lamy Syndrome
- Trial name
- NCT02171104
- Testing
- Stem Cell Transplantation, IMD Preparative Regimen, Osteopetrosis Only Preparative Regimen
- Where
- United States
- Volunteers
- 149 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Cardiomyopathies, Amyloidosis Cardiac
Characterization of Patients With Cardiomyopathy to Identify Critical Patients Candidates for Cardiac Transplantation
The study aims to identify new diagnostic and prognostic markers for CMP that can help predict disease progression. In particular, the study will focus on microRNAs (miRNAs) and spatial transcriptomics, which are emerging…
- Conditions
- Cardiomyopathies, Amyloidosis Cardiac, Fabry Disease, Arrhythmogenic Cardiomyopathy
- Trial name
- CMPMIRNA (NCT06813443)
- Where
- 3 sites in Italy
- Volunteers
- 700 people
- Sponsor
- IRCCS Azienda Ospedaliero-Universitaria di Bologna
Nephropathy, Hypoxia
Acute Effects of SGLT2 Inhibition on Renal Oxygenation and Autonomic Function in Type 1 Diabetes
Background: Inhibiting the sodium-glucose cotransporter-2 (SGLT2) has been observed to reduce risk of cardiovascular events and kidney failure in type 2 diabetes.
- Conditions
- Nephropathy, Hypoxia, Mitochondrial Alteration, Type 1 Diabetes
- Trial name
- Astronaut (NCT04193566)
- Testing
- Forxiga
- Where
- Denmark
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Steno Diabetes Center Copenhagen
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
Primary Mitochondrial Disease
OMT-28 in Patients With Primary Mitochondrial Disease (PMD) (PMD-OPTION)
The goal of this clinical trial is to learn about the treatment effects of the investigational new drug OMT-28 in patients with Primary Mitochondrial Disease.
- Trial name
- PMD-OPTION (NCT05972954)
- Testing
- OMT-28
- Where
- 9 sites in Germany, Italy and 1 more
- Volunteers
- 28 people
- Sponsor
- Omeicos Therapeutics GmbH
HIV Infections
Metabolic Effects of Enfuvirtide in Healthy Volunteers
The metabolic effects of T-20 are not completely known, since the drug is used in combination with other antiretroviral agents. A short-term study in healthy volunteers, with a double-blind crossover design vs.
- Trial name
- NCT00657761
- Testing
- Enfuvirtide
- Where
- Spain
- Volunteers
- 15 people, healthy volunteers welcome
- Sponsor
- Hospital Clinic of Barcelona