Clinical trials: Muscle-Eye-Brain Disease
Mitochondrial Diseases, Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation
The KHENEREXT Study
This is an open-label, multi-centre study in subjects with a genetically confirmed mitochondrial deoxyribonucleic acid (DNA) transfer ribonucleic acid (tRNA)Leu(UUR) m.3243A>G mutation who completed study KH176-202.
- Conditions
- Mitochondrial Diseases, Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation, Maternally Inherited Diabetes and Deafness (MIDD), Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke Like Episodes (MELAS)
- Trial name
- NCT04604548
- Testing
- Oral administration of 100 mg KH176 twice daily
- Where
- 4 sites in Denmark, Germany and 2 more
- Volunteers
- 11 people
- Sponsor
- Khondrion BV
Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor
Amifostine to Protect From Side Effects of PSCT in Treating Patients With Solid Tumors
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining chemotherapy with peripheral stem cell transplantation may allow the doctor to give higher doses of…
- Conditions
- Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Chordoma, Kidney Cancer
- Trial name
- NCT00003926
- Testing
- amifostine trihydrate, busulfan, filgrastim
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Sjogren-Larsson Syndrome
ADX-629 Therapy for Sjogren-Larsson Syndrome
This research study will determine whether orally administered ADX-629 is safe and has biochemical efficacy in participants with Sjögren-Larsson syndrome (SLS), a rare inherited disorder of fatty aldehyde metabolism The disease…
- Trial name
- NCT05443685
- Testing
- ADX-629
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- University of Nebraska
Mitochondrial Diseases, Mitochondrial Myopathies
Nicotinamide Riboside and Mitochondrial Biogenesis
Mitochondria are important parts of the cell that are responsible for producing energy. The amount of energy they produce depends on how much energy the body needs to function and this energy production can be severely impaired…
- Conditions
- Mitochondrial Diseases, Mitochondrial Myopathies, Progressive External Ophthalmoplegia, Progressive Ophthalmoplegia
- Trial name
- NCT03432871
- Testing
- Nicotinamide Riboside
- Where
- United Kingdom
- Volunteers
- 13 people
- Sponsor
- Cambridge University Hospitals NHS Foundation Trust
Fibromyalgia
QEEG and Qualitative EEG for the Identidification of Abnormal Patterns in Fibromyalgia Patients
Fibromyalgia is a relatively young condition recently recognized by the WHO as a separated clinical entity. Part of the medical comunity thinks of it as a mixed condition between depresion and rheumatic pain, however, functional…
- Trial name
- QEEGFP (NCT02662270)
- Where
- Spain
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- Spanish Foundation for Neurometrics Development
Pericarditis, Tako-TSUBO Cardiomyopathy
Inflammatory Disease Biobank for Immunophenotyping and Cardiovascular Research
INFLAME-BANK is a French multicenter prospective observational ancillary study of the international EACVI-INFLAME project. It aims to establish a biobank and perform immunophenotyping and proteomic analyses in patients with…
- Conditions
- Pericarditis, Tako-TSUBO Cardiomyopathy, Myocarditis, Inflammatory Cardiomyopathies
- Trial name
- INFLAME-BANK (NCT07737470)
- Volunteers
- 300 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Adenovirus, Anesthesia
Pharmacokinetics of Understudied Drugs Administered to Children Per Standard of Care
Understudied drugs will be administered to children per standard of care as prescribed by their treating caregiver and only biological sample collection during the time of drug administration will be involved.
- Conditions
- Adenovirus, Anesthesia, Anxiety, Anxiolysis
- Trial name
- PTN_POPS (NCT01431326)
- Testing
- The POPS study is collecting PK data on children prescribed the following drugs of interest per standard of care:
- Where
- 52 sites in United States, Canada and 3 more
- Volunteers
- 3,520 people
- Sponsor
- Daniel Benjamin
Amblyopia
Proof of Concept Study of Binocular Videogames Versus Patching for Amblyopia
The purpose of this PoC study was to evaluate the potential therapeutic efficacy of binocular video games played on a tablet and to compare the efficacy of binocular video games versus patching in amblyopic patients 4 to 7 years…
- Trial name
- NCT04784390
- Testing
- Binocular video games, Patching of the sound eye
- Where
- 8 sites in United States, Australia and 1 more
- Volunteers
- 27 people
- Sponsor
- Novartis Pharmaceuticals
All Diagnosed Health Conditions, ADD/ADHD
At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions
We are the missing link in clinical trials, connecting patients and researchers seamlessly and conveniently using a mobile health platform to advance medical research.
- Conditions
- All Diagnosed Health Conditions, ADD/ADHD, Alopecia Areata, Ankylosing Spondylitis
- Trial name
- NCT01931644
- Where
- United States
- Volunteers
- 17,667 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
MELAS Syndrome, Lebers Hereditory Optic Neuropathy With Extra Ocular Symptoms (LHON-Plus)
A Basket Clinical Study to Assess Glycerol Tributyrate in Patients With Mitochondrial Encephalopathy, Lactic Acidosis, Stroke-like Episodes (MELAS) or Leber's Hereditary Optic Neuropathy-Plus (LHON-Plus)
This is a parallel arm non-randomized dose-escalation, open-label basket exploratory phase 1 clinical trial where Mitochondrial encephalopathy, lactic acidosis, stroke-like episodes (MELAS) and Leber's hereditary optic…
- Trial name
- NCT06792500
- Testing
- Glycerol Tributyrate
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- George Washington University
ALS, Autism Spectrum Disorder
Pharmacokinetic (PK) and Pharmacodynamics (PD) Study of Ilera Specific Products
This is an observational study of medical marijuana manufactured and dispensed by Ilera and given as standard treatment for a variety of approved serious medical conditions as defined by individual state law.
- Conditions
- ALS, Autism Spectrum Disorder, Cancer, Spasticity, Muscle
- Trial name
- Ilera (NCT03886753)
- Testing
- Registry, PK microsampling of blood
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Children's Hospital of Philadelphia
Neurodegenerative Disorders
Development of IPS from Donated Somatic Cells of Patients with Neurological Diseases
Human fibroblasts and possibly other human somatic cells may be reprogrammed into induced pluripotent stem (iPS) cells by the forced expression of transcription factors (1-5).
- Trial name
- NCT00874783
- Where
- Israel
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Hadassah Medical Organization
Parkinson Disease, Multiple System Atrophy
Evaluation of the Efficacy of a Two-week EMST on Dysphagia in Parkinsonian Patients
This is an interventional therapy study designed to evaluate the efficacy of a two-week intervention, i.e. training with a specialized exhalation training device (called expiratory muscle strength training; EMST150 or EMST75…
- Conditions
- Parkinson Disease, Multiple System Atrophy, Progressive Supranuclear Palsy
- Trial name
- EMST-PS (NCT05139342)
- Testing
- expiratory muscle strentgh training (EMST)
- Where
- 3 sites in Germany
- Volunteers
- 75 people
- Sponsor
- Kliniken Beelitz GmbH
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
Frontotemporal Degeneration (FTD), ALS (Amyotrophic Lateral Sclerosis)
Biofluid Research on Age-Related or Inherited Neurodegeneration
The aim of this study is to contribute to and continue to sustain a repository of blood samples, nucleic acid (DNA and/or RNA) samples, family history information, and other demographic information from individuals with…
- Conditions
- Frontotemporal Degeneration (FTD), ALS (Amyotrophic Lateral Sclerosis), Primary Progressive Aphasia (PPA), Familial Frontotemporal Lobar Degeneration (fFTLD)
- Trial name
- BRAIN (NCT07798700)
- Where
- United States
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- University of Pennsylvania
Chronic Pain, Chronic Pain Syndrome
Outcomes Mandate National Integration With Cannabis as Medicine
This will be a multistate, multicenter clinical study to determine the efficacy and safety of medical cannabis for a wide variety of chronic medical conditions.
- Conditions
- Chronic Pain, Chronic Pain Syndrome, Chronic Pain Due to Injury, Chronic Pain Due to Trauma
- Trial name
- OMNI-Can (NCT03944447)
- Testing
- Cannabis, Medical, RYAH-Medtech Inhaler
- Where
- 17 sites in United States
- Volunteers
- 200,000 people, healthy volunteers welcome
- Sponsor
- OMNI Medical Services, LLC
Ewing's Family Tumors, Renal Tumors
Auto Transplant for High Risk or Relapsed Solid or CNS Tumors
This is a standard of care treatment guideline for high risk or relapsed solid tumors or CNS tumors consisting of a busulfan, melphalan, thiotepa conditioning (for solid tumors) or carboplatin and thiotepa conditioning (for CNS…
- Conditions
- Ewing's Family Tumors, Renal Tumors, Hepatoblastoma, Rhabdomyosarcoma
- Trial name
- NCT01505569
- Testing
- Ifosfamide, Etoposide, Mesna
- Where
- United States
- Volunteers
- 44 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Amyotrophic Lateral Sclerosis (ALS), Angina, Stable
Quest to Analyze One Thousand Humans Meditating
This study will be focused on assessing the molecular, physiological, and emotional correlates of an intensive meditation experience in the context of a retreat setting in a large 2000 plus-person cohort comprised of healthy and…
- Conditions
- Amyotrophic Lateral Sclerosis (ALS), Angina, Stable, Anxiety, Asthma
- Trial name
- QUANTUM (NCT06583395)
- Testing
- Advanced multi-component meditation practice
- Where
- United States
- Volunteers
- 3,000 people, healthy volunteers welcome
- Sponsor
- Tobias Moeller-Bertram
Fibromyalgia, Chronic Pain
Functional Neuroimaging in Fibromyalgia Patients Receiving tDCS
The purpose of this study is to evaluate effectiveness and cerebral neuronal ability to adaptation in patients with fibromyalgia who receive pregabalin and transcranial direct current stimulation.
- Trial name
- NCT01904097
- Testing
- Pregabalin, Transcranial Direct Current Stimulation
- Where
- Brazil
- Volunteers
- 34 people
- Sponsor
- Hospital de Clinicas de Porto Alegre