Clinical trials: Metabolic Myopathies
Muscular Dystrophy, Duchenne
Effect of EPA and DHA in the Inflammation and Metabolic Disorders in DMD/DMB Patients
The purpose of this study is to evaluate the effect of docosahexaenoic fatty acid and eicosapentaenoic fatty acid supplementation for six months on the inflammation state as well as the process of muscular regeneration and the…
- Trial name
- NCT01826422
- Testing
- EPA and DHA
- Where
- Mexico
- Volunteers
- 40 people
- Sponsor
- Coordinación de Investigación en Salud, Mexico
Type 1 Diabetes
The Summer Camp Study: Blood Glucose Control With a Bi-Hormonal Bionic Endocrine Pancreas
This study will test the hypothesis that a wearable automated bionic pancreas system that automatically delivers both insulin and glucagon can improved glycemic control vs.
- Trial name
- NCT01833988
- Testing
- Bi-hormonal Bionic Pancreas, Usual Care
- Where
- United States
- Volunteers
- 32 people
- Sponsor
- Massachusetts General Hospital
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.
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
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)
Infantile Neuroaxonal Dystrophy
Desipramine in Infantile Neuroaxonal Dystrophy (INAD).
This is a research study to find out if clinically prescribed desipramine is effective at improving the symptoms and slowing the progression of Infantile Neuroaxonal Dystrophy (INAD) in affected children.
- Trial name
- NCT03726996
- Testing
- Desipramine
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Duke University
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
Dermatomyositis, Idiopathic Inflammatory Myopathies
Sodium Thiosulfate for Treatment of Calcinosis Associated With Juvenile and Adult Dermatomyositis
Background: Dermatomyositis (DM) and juvenile dermatomyositis (JDM) cause inflammation in the muscles. People with DM and JDM can develop calcium deposits in places they should not, known as calcinosis.
- Trial name
- NCT03267277
- Testing
- Sodium Thiosulfate
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- National Institute of Environmental Health Sciences (NIEHS)
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
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
Type 1 Diabetes
The Summer Camp Study 2: Blood Glucose Control With a Bi-Hormonal Endocrine Pancreas
This study will test the hypothesis that a wearable automated bionic pancreas system that automatically delivers both insulin and glucagon can improve glycemic control vs.
- Trial name
- NCT02105324
- Testing
- Bionic Pancreas, Usual Care
- Where
- United States
- Volunteers
- 19 people
- Sponsor
- Massachusetts General Hospital
MELAS Syndrome
Study of Idebenone in the Treatment of Mitochondrial Encephalopathy Lactic Acidosis & Stroke-like Episodes
The purpose of this study is to compare the efficacy of two (2) different doses of idebenone with that of a placebo over a one month period on cerebral lactate concentration as measured by magnetic resonance spectroscopy.
- Trial name
- MELAS (NCT00887562)
- Testing
- Idebenone
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- Michio Hirano
Osteoporosis
An Efficacy and Safety Trial of Intravenous Zoledronic Acid Twice Yearly in Osteoporotic Children Treated With Glucocorticoids
This study was designed to evaluate the efficacy and safety of zoledronic acid compared to placebo in osteoporotic children treated with glucocorticoids
- Trial name
- NCT00799266
- Testing
- Zoledronic acid
- Where
- 12 sites in Australia, Canada and 4 more
- Volunteers
- 34 people
- Sponsor
- Novartis Pharmaceuticals
Mitochondrial Diseases
A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease
A long-term extension study to assess the safety, tolerability and efficacy of cysteamine bitartrate delayed-release capsules (RP103) in children with inherited mitochondrial diseases who previously enrolled into study…
- Trial name
- NCT02473445
- Testing
- Cysteamine Bitartrate
- Where
- 5 sites in United States
- Volunteers
- 22 people
- Sponsor
- Amgen
Osteoporosis
1 Year Open-label Extension to CZOL446H2337 Safety and Efficacy Trial of Zoledronic Acid Twice Yearly in Osteoporotic Children Treated With Glucocorticoids
This 1-year open-label extension to CZOL446H2337 is designed to evaluate the safety and efficacy of zoledronic acid twice yearly in osteoporotic children treated with glucocorticoids.
- Trial name
- NCT01197300
- Testing
- Zoledronic acid
- Where
- 10 sites in Australia, Canada and 4 more
- Volunteers
- 25 people
- Sponsor
- Novartis Pharmaceuticals
Inherited Mitochondrial Disease, Including Leigh Syndrome
Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease
To evaluate safety, tolerability and efficacy of cysteamine bitartrate delayed-release capsules (RP103) administered at a target maintenance dose of 1.3 g/m²/day in two divided doses, every 12 hours, for up to 6 months in…
- Trial name
- MITO-001 (NCT02023866)
- Testing
- Cysteamine Bitartrate
- Where
- 5 sites in United States
- Volunteers
- 36 people
- Sponsor
- Amgen