Clinical trials: Mitochondrial Diseases
Insulin Sensitivity, Multiple Mitochondrial Dysfunctions Syndrome
Study of Dapagliflozin on Mitochondrial Dysfunction and Impaired Insulin Signaling/Action
The purpose of this study is to examine the effect of the chronic treatment of type 2 diabetes (T2DM) with dapagliflozin on: (1) mitochondrial gene function/expression and insulin signaling/action and (2) oral glucose tolerance…
- Trial name
- DAPA MITO (NCT01439854)
- Testing
- Dapagliflozin
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- The University of Texas Health Science Center at San Antonio
Small Fiber Neuropathy, Fabry Disease
Assessment of Small Fiber Neuropathy in Rare Diseases Using Sudoscan
Sudoscan™ (Impeto Medical, Paris France) uses electrochemical skin conductance as a novel noninvasive method to detect sudomotor dysfunction.
- Conditions
- Small Fiber Neuropathy, Fabry Disease, Ehlers Danlos Syndrome, Mitochondrial Disease
- Trial name
- NCT02985710
- Testing
- Sudoscan, Skin biopsy, QSART
- Where
- United States
- Volunteers
- 102 people, healthy volunteers welcome
- Sponsor
- Massachusetts General Hospital
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
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
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
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.
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
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
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
Impact of Statin Therapy on Muscle Mitochondrial Function and Aerobic Capacity
The purpose of this study is to test how different doses of a statin, Lipitor, affect muscle health and function, and cardiovascular fitness.
- Trial name
- NCT03351998
- Testing
- Lipitor 20Mg Tablet, Lipitor 80Mg Tablet
- Where
- 2 sites in United States
- Volunteers
- 68 people, healthy volunteers welcome
- Sponsor
- University of Kansas Medical Center
Optic, Atrophy, Hereditary, Leber
Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation
The goal of this clinical trial is to assess the effectiveness of GS010, a gene therapy, in improving the visual outcome in participants with Leber Hereditary Optic Neuropathy (LHON) due to the G11778A ND4 mitochondrial mutation…
- Trial name
- RESCUE (NCT02652767)
- Testing
- GS010
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 39 people
- Sponsor
- GenSight Biologics
Primary Mitochondrial Disease
Safety, Tolerability, Efficacy of MTP-131 for Treatment of Mitochondrial Disease in Subjects From the MMPOWER Study
This randomized, double-blind, placebo-controlled, crossover study screened 32 subjects with primary mitochondrial myopathy (PMM) to evaluate the safety, tolerability, pharmacokinetics (PK), and efficacy of subcutaneous…
- Trial name
- MMPOWER-2 (NCT02805790)
- Testing
- Elamipretide
- Where
- 4 sites in United States
- Volunteers
- 30 people
- Sponsor
- Stealth BioTherapeutics Inc.
Blepharoptosis, Ptosis, Eyelid
Clinical Trial Comparing Two Non-Surgical Treatments for Severe Blepharoptosis
This clinical trial will evaluate two non-surgical devices designed to improve eye lid opening for patients with severe Blepharoptosis (incomplete opening of the eyelids).
- Conditions
- Blepharoptosis, Ptosis, Eyelid, Myasthenia Gravis, Stroke
- Trial name
- NCT04678115
- Testing
- Magnetic Levator Prosthesis (MLP), Kinesiotape Frontalis Sling (KTFS)
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Massachusetts Eye and Ear Infirmary
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.
Acute Lymphoblastic Leukemia, Hyperbilirubinemia
Levocarnitine and Vitamin B Complex in Treating PEG-Asparaginase or Inotuzumab Ozogamicin-Induced Hyperbilirubinemia in Patients With Acute Lymphoblastic Leukemia
This phase II trial studies how well levocarnitine and vitamin B complex works in treating abnormal high liver enzyme levels (hyperbilirubinemia) caused by treatment with PEG-asparaginase or inotuzumab ozogamicin in patients with…
- Trial name
- NCT03564678
- Testing
- Levocarnitine, Vitamin B Complex
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- M.D. Anderson Cancer Center
Chronic Kidney Disease, Sarcopenia
Trial of Nicotinamide Riboside and Co-enzyme Q10 in Chronic Kidney Disease
Chronic kidney disease is associated with the loss of skeletal muscle mass and function. This process detrimentally impacts mobility, functional independence, and quality of life.
- Conditions
- Chronic Kidney Disease, Sarcopenia, Frailty
- Trial name
- CoNR (NCT03579693)
- Testing
- CoQ10, Nicotinamide riboside
- Where
- United States
- Volunteers
- 26 people
- Sponsor
- University of Washington
Friedreich's Ataxia
A Study of Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia (FRDA) Patients
The purpose of this trial is to study the efficacy, safety and tolerability of idebenone in 12 months of treatment in children and adults with Friedreich's Ataxia.
- Trial name
- MICONOS (NCT00905268)
- Testing
- idebenone
- Where
- 13 sites in Austria, Belgium and 4 more
- Volunteers
- 232 people
- Sponsor
- Santhera Pharmaceuticals
Autism Spectrum Disorder
Effect of a Wide Spectrum Nutritional Supplement on Mitochondrial Function in Children With Autism Spectrum Disorder
The objective of this study is to evaluate the metabolic effects of a comprehensive wide-spectrum supplement for children with ASD to determine whether it physiologically targets mitochondrial pathways known to be abnormal in…
- Trial name
- NCT03835117
- Testing
- Wide-spectrum nutritional supplement
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Rossignol Medical Center
Diabetes Mellitus, Type 2, Skeletal Muscle Insulin Sensitivity
Effects of 5 Weeks Treatment With Dapagliflozin in Type 2 Diabetes Patients on How the Hormone Insulin Acts on Sugar Uptake in Muscles.
The purpose of this study is to investigate the effects of 5 weeks treatment with dapagliflozin in type 2 diabetes patients on how the hormone insulin acts on sugar uptake in muscles.
- Trial name
- DAPAMAAST (NCT03338855)
- Testing
- Dapagliflozin
- Where
- Netherlands
- Volunteers
- 26 people
- Sponsor
- AstraZeneca
Friedreich's Ataxia
Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia
The purpose of this phase 3 randomized, multi-center, double-blind, placebo-controlled study is to evaluate the efficacy and safety of ACTIMMUNE® (interferon-γ 1b) in the treatment of Friedreich's Ataxia (FA) and to evaluate the…
- Trial name
- STEADFAST (NCT02415127)
- Testing
- Interferon γ-1b
- Where
- 4 sites in United States
- Volunteers
- 92 people
- Sponsor
- Amgen