Clinical trials: Mitochondrial Disease
Mitochondrial Diseases, Mitochondrial Respiratory Chain Deficiencies
A Phase Ia/Ib, SAD and MAD Study of of KL1333 in Healthy Subjects and Patients With Primary Mitochondrial Disease
This will be a double blind, randomised, placebo controlled, single and multiple oral dose study conducted in 3 parts: Part A, Part B and Part C.
- Conditions
- Mitochondrial Diseases, Mitochondrial Respiratory Chain Deficiencies, MELAS Syndrome, Mitochondrial Myopathies
- Trial name
- NCT03888716
- Testing
- KL1333
- Where
- 2 sites in United Kingdom
- Volunteers
- 72 people, healthy volunteers welcome
- Sponsor
- Abliva AB
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.
Mitochondrial Diseases, Developmental Delay Disorder
Reference Values of Intraepidermal Nerve Fiber Density in Children and Small Fiber Neuropathy in Neurodevelopmental Disorders
Background: Small-Fiber-Neuropathy describes the degeneration of mildly or unmyelinated nerve fibers and causes neuropathic pain and autonomic dysfunction.
- Conditions
- Mitochondrial Diseases, Developmental Delay Disorder, Small Fiber Neuropathy, Cerebral Palsy
- Trial name
- SFN_children (NCT05645874)
- Testing
- Skin biopsy
- Where
- Germany
- Volunteers
- 203 people, healthy volunteers welcome
- Sponsor
- Heinrich-Heine University, Duesseldorf
Osteoarthritis, Knee, Pain
Biologic Mechanisms for Pain Variation After Physical Activity in Osteoarthritis
Osteoarthritis (OA) in the knee is characterized by chronic inflammatory pain that is not necessarily related to the amount of joint damage.
- Conditions
- Osteoarthritis, Knee, Pain, Physical Activity, Mitochondrial Pathology
- Trial name
- NCT03344913
- Testing
- Walking
- Where
- United States
- Volunteers
- 13 people, healthy volunteers welcome
- Sponsor
- University of Maryland, Baltimore
Obesity
Effects of High-intensity Exercise With Hypoxia and Taurine Supplementation on Metabolic and Mitochondrial Parameters
The prevalence of obesity in Brazil is one of the highest in Latin America. As an inflammatory disease, obesity needs to be treated, and one of the best interventions is exercise.
- Trial name
- NCT06579508
- Testing
- Cycle ergometer
- Where
- 2 sites in Brazil
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- University of Sao Paulo
Acute Kidney Injury (Nontraumatic), Critical Illness
Impact of CRRT on Serum Carnitine and Micronutrient Levels
This study aims to investigate the changes in blood levels of micronutrients and carnitine in critically ill patients with Acute Kidney Injury (AKI) who are undergoing Continuous Renal Replacement Therapy (CRRT).
- Trial name
- NCT07467889
- Testing
- Continuous Renal Replacement Therapy (CRRT), Standard of Care (Non-CRRT Management)
- Where
- Turkey (Türkiye)
- Volunteers
- 43 people
- Sponsor
- Gulhane Training and Research Hospital
MELAS, LHON
A Dose-escalating Clinical Trial With KH176
Mitochondrial Diseases are rare progressive, multi-system, often early fatal disorders affecting both children and adults. KH176 is a novel chemical entity currently under development for the treatment of inherited mitochondrial…
- Conditions
- MELAS, LHON, Leigh Syndrome, Mitochondrial Disease
- Trial name
- NCT02544217
- Testing
- KH176
- Where
- Belgium
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Khondrion BV
Friedreich Ataxia
Methylprednisolone Treatment of Friedreich Ataxia
This study will explore whether methylprednisolone treatment is safe, well-tolerated, and beneficial in patients that are diagnosed with Friedreich Ataxia (FRDA).
- Trial name
- NCT02424435
- Testing
- Methylprednisolone
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Children's Hospital of Philadelphia
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
Autism Spectrum Disorder
An Exploratory Open Label Study of EPI-743 (Vincerinone TM) in Children With Autism Spectrum Disorder
The investigators hypothesize that EPI-743 may provide clinical benefit to children with Autism Spectrum Disorder.
- Trial name
- Autism (NCT02226458)
- Testing
- EPI-743
- Sponsor
- Edison Pharmaceuticals 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
Alzheimer's Disease
Safety and Efficacy Study Evaluating Dimebon in Patients With Mild to Moderate Alzheimer's Disease on Donepezil
The purpose of this study is to determine if Dimebon is safe and effective in patients with mild to moderate Alzheimer's disease on Donepezil.
- Trial name
- CONCERT (NCT00829374)
- Testing
- Dimebon
- Where
- 101 sites in United States, Australia and 8 more
- Volunteers
- 1,003 people
- Sponsor
- Medivation, Inc.
Obese, Growth Hormone Secretion Abnormality
Growth Hormone Treatment on Phosphocreatine Recovery in Obesity
Obesity is associated with reduced growth hormone (GH) secretion. Reduced GH secretion in obesity is associated with increased cardiovascular disease risk.
- Trial name
- NCT01421589
- Testing
- Growth hormone treatment
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Massachusetts General Hospital
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
Cystinosis, Native Kidney
Cystinosis and Mitochondrial Metabolism
Cystinosis is a monogenic autosomal recessive lysosomal storage disease with complete penetrance, caused by a biallelic mutation in the CTNS gene (17p13.2) encoding cystinosin, a ubiquitous membrane protein whose role is to clear…
- Trial name
- CYSTI-MITO (NCT07319091)
- Testing
- Mitochondrial metabolism
- Where
- 9 sites in France
- Volunteers
- 25 people
- Sponsor
- Hospices Civils de Lyon
Myocardial Ischemia, Coronary Artery Bypass Grafting
The Effects of Allicor on Patients After Coronary Arteria Revascularization Treatment
Coronary revascularization interventions such as coronary artery bypass graft (CABG) and percutaneous coronary intervention (PCI) are the standard of surgical treatment of patients with myocardial ischemia.
- Conditions
- Myocardial Ischemia, Coronary Artery Bypass Grafting, Coronary Arteriosclerosis, Percutaneous Coronary Revascularization
- Trial name
- TEA-CART (NCT05803759)
- Testing
- Allicor
- Where
- Russia
- Volunteers
- 200 people
- Sponsor
- Institute for Atherosclerosis Research, Russia
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
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
Breast Cancer
FBnTP Imaging of Breast Cancer
Our preclinical studies suggest the capacity of the positron emission tomography imaging agent 18F-fluorobenzyl triphenyl phosphonium (FBnTP) to detects early-stage small breast tumors (e.g., DCIS), and differentiates benign from…
- Trial name
- NCT02204462
- Testing
- FBnTP
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Friedreich's Ataxia
Effect of Pioglitazone Administered to Patients With Friedreich's Ataxia: Proof of Concept
Friedreich's ataxia (FA) is a rare progressive neurological disorder affecting approximately 1/30, 000 individuals. No treatment is presently available to counteract the neurodegeneration of this extremely severe disease.
- Trial name
- ACTFRIE (NCT00811681)
- Testing
- pioglitazone
- Where
- France
- Volunteers
- 40 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris