Clinical trials: Muscle-Eye-Brain Disease
Blepharospasm, Hemifacial Spasm
Post Marketing Surveillance Study of Dysport
The purpose of this study is to provide further information regarding the risks and benefits of Dysport in marketed indications.
- Conditions
- Blepharospasm, Hemifacial Spasm, Cervical Dystonia, Spasmodic Torticollis
- Trial name
- NCT00210431
- Where
- 36 sites in France, Germany and 1 more
- Volunteers
- 783 people
- Sponsor
- Ipsen
Mitochondrial Diseases, Kearns-Sayre Syndrome
Global Registry and Natural History Study for Mitochondrial Disorders
The main goal of the project is provision of a global registry for mitochondrial disorders to harmonize previous national registries, enable world-wide participation and facilitate natural history studies, definition of outcome…
- Conditions
- Mitochondrial Diseases, Kearns-Sayre Syndrome, MIDD, SANDO
- Trial name
- GENOMIT (NCT05554835)
- Where
- 34 sites in Austria, Germany and 1 more
- Volunteers
- 6,000 people
- Sponsor
- LMU Klinikum
Rheumatic Diseases, Adult Onset Still Disease
Rheumatology Patient Registry and Biorepository
To facilitate clinical, basic science, and translational research projects involving the study of rheumatic diseases.
- Conditions
- Rheumatic Diseases, Adult Onset Still Disease, Ankylosing Spondylitis, Psoriatic Arthritis
- Trial name
- NCT04402086
- Where
- United States
- Volunteers
- 5,000 people, healthy volunteers welcome
- Sponsor
- Yale University
Congenital Muscular Dystrophy With ITGA7 (Integrin Alpha-7) Deficiency, Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy and Abnormal Glycosylation of Dystroglycan With Severe Epilepsy)
Congenital Muscle Disease Study of Patient and Family Reported Medical Information
The Congenital Muscle Disease Patient and Proxy Reported Outcome Study (CMDPROS) is a longitudinal 10 year study to identify and trend care parameters, adverse events in the congenital muscle diseases using the Congenital Muscle…
- Conditions
- Congenital Muscular Dystrophy With ITGA7 (Integrin Alpha-7) Deficiency, Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy and Abnormal Glycosylation of Dystroglycan With Severe Epilepsy), Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Fatty Liver and Infantile-onset Cataract Caused by TRAPPC11 Mutations), Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Hypoglycosylation of Dystroglycan)
- Trial name
- CMDPROS (NCT01403402)
- Where
- United States
- Volunteers
- 4,000 people
- Sponsor
- Cure CMD
PolG, Primary Mitochondrial Myopathy
Prospective Natural History Study of POLG Disease
The PIONEER study is a prospective, natural history study dedicated to characterizing the clinical progression of POLG-related disorders.
- Trial name
- PIONEER (NCT07775872)
- Where
- 21 sites in United States, Australia and 7 more
- Volunteers
- 300 people
- Sponsor
- The POLG Foundation
Solid Tumors With PTCH1 Loss-of-function Mutations
A Study Evaluating the Safety and Efficacy of ENV-101 (Taladegib) in Patients With Advanced Solid Tumors Harboring PTCH1 Loss of Function Mutations
This study employs a 2-stage design that aims to evaluate the efficacy and safety of ENV- 101, a potent Hedgehog (Hh) pathway inhibitor, in patients with refractory advanced solid tumors characterized by loss of function (LOF)…
- Trial name
- NCT05199584
- Testing
- ENV-101 (taladegib)
- Where
- 16 sites in United States
- Volunteers
- 20 people
- Sponsor
- Endeavor Biomedicines, Inc.
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Systemic Lupus Erythematosus, Lupus Nephritis
Fourth-gen CAR T Cells Targeting BCMA/CD19 for Refractory Systemic Lupus Erythematosus (SLE)
This study is a preliminary investigation, with a single-group design, not randomized and transparent, focusing on treatment. Its purpose is to identify the highest dose of BH002 injection (CD19-BCMA CAR-T cells) that patients…
- Conditions
- Systemic Lupus Erythematosus, Lupus Nephritis, Autoimmune Diseases, Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
- Trial name
- BAH242 (NCT06350110)
- Testing
- CD19- BCMA CAR-T cells
- Where
- China
- Volunteers
- 75 people
- Sponsor
- Essen Biotech
Age-Related Macular Degeneration, Allergies
Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives
To collect, preserve, and/or distribute annotated biospecimens and associated medical data to institutionally approved, investigator-directed biomedical research to discover and develop new treatments, diagnostics, and…
- Conditions
- Age-Related Macular Degeneration, Allergies, Alpha-Gal Syndrome, Alzheimer Disease
- Trial name
- NCT05635266
- Testing
- Specimen sample
- Where
- United States
- Volunteers
- 20,000 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
Strabismus, Amblyopia
Exercise Interventions of Eye Muscles Post Strabismus Surgery
It will be hypothesized that: Eye exercises have a positive effect on correction of remaining ocular deviation post strabismus surgery. Eye exercises have a positive effect on improving amplyobia.
- Trial name
- NCT04303832
- Testing
- eye exercises, eye glasses
- Where
- Egypt
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Cairo University
Eye Diseases Hereditary, Retinal Disease
Inherited Retinal Degenerative Disease Registry
The My Retina Tracker® Registry is sponsored by the Foundation Fighting Blindness and is for people affected by one of the rare inherited retinal degenerative diseases studied by the Foundation.
- Conditions
- Eye Diseases Hereditary, Retinal Disease, Achromatopsia, Bardet-Biedl Syndrome
- Trial name
- MRTR (NCT02435940)
- Where
- United States
- Volunteers
- 20,000 people, healthy volunteers welcome
- Sponsor
- Foundation Fighting Blindness
Neuromuscular Diseases, Obesity (Disorder)
Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic Pathology
The ActiLiège-Adult study is a prospective, longitudinal, observational study designed to collect natural history data on adult patients with neurological or metabolic diseases affecting movement.
- Conditions
- Neuromuscular Diseases, Obesity (Disorder), Myotonic Dystrophy 1, Myasthenic Syndrome
- Trial name
- Acti-Adult (NCT07136844)
- Testing
- Syde, Dynamometric measurements of muscle strength, The 6-minute walk test (6MWT)
- Where
- Belgium
- Volunteers
- 300 people
- Sponsor
- Centre Hospitalier Universitaire de Liege
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
PolG, Primary Mitochondrial Myopathy
Mitochondrial Video Assessment Source Material Study
Source material collection of videos to develop the Mitochondrial Video Assessment (MVA) for patients with mitochondrial myopathies.
- Trial name
- NCT06180421
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- The Emmes Company, LLC
Fibromyalgia, Multiple Sclerosis
e-Mobile Tablet for People With Chronic Conditions
Maintaining a healthy diet and engaging in routine physical activity may help decrease severity of symptoms, prevent secondary conditions, and slow functional decline in people with disabling neuromusculoskeletal conditions.
- Conditions
- Fibromyalgia, Multiple Sclerosis, Osteoarthritis, Sjögren's Syndrome
- Trial name
- NCT02833311
- Testing
- Computer Tablet Group, Paper and Pencil Group, Contact Control Group
- Where
- United States
- Volunteers
- 46 people
- Sponsor
- Case Western Reserve University
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
Muscular Dystrophy
Clinical Trial Readiness for the Dystroglycanopathies
The purpose of the study is to describe the early signs and symptoms of the dystroglycanopathies, and to gather information that will be required for future clinical trials.
- Trial name
- NCT00313677
- Where
- United States
- Volunteers
- 190 people
- Sponsor
- Katherine Mathews
Central Nervous System/Leptomeningeal Neoplasms
131I-omburtamab for the Treatment of Central Nervous System/Leptomeningeal Neoplasms in Children and Young Adults
The researchers are doing this study to provide access to treatment with 131I-omburtamab for children and young adults who have CNS/leptomeningeal neoplasms.
- Trial name
- NCT05064306
- Testing
- 131I-omburtamab
- Where
- United States
- Sponsor
- Memorial Sloan Kettering Cancer Center
Autoimmune Diseases of the Nervous System, Neuromyelitis Optica Spectrum Disorder
Biomarkers in Autoimmune Disease of Nervous System
Neurological autoimmune diseases are a group of disorders characterized by the abnormal immune response attacking the nervous system, including the brain, spinal cord and peripheral nerves.
- Conditions
- Autoimmune Diseases of the Nervous System, Neuromyelitis Optica Spectrum Disorder, Multiple Sclerosis, Guillain-Barre Syndrome
- Trial name
- NCT06502015
- Testing
- biomaker levels
- Where
- China
- Volunteers
- 50,000 people, healthy volunteers welcome
- Sponsor
- Tongji Hospital
Stroke, Ischemic, Stroke Hemorrhagic
Exploring Accessible Beauty for Individuals With Upper Extremity Deficits
The purpose of this study is to investigate the accessibility of beauty products for individuals with upper extremity disabilities.
- Conditions
- Stroke, Ischemic, Stroke Hemorrhagic, Stroke (CVA) or TIA, Stroke, Acute
- Trial name
- NCT05945784
- Testing
- Use of Rare Beauty makeup products
- Where
- United States
- Volunteers
- 57 people, healthy volunteers welcome
- Sponsor
- Casa Colina Hospital and Centers for Healthcare