Clinical trials: Muscle-Eye-Brain Disease
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
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
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
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
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
LGMD2I, LGMDR9
Global FKRP Registry
Mutations in the Fukutin Related Protein (FKRP) gene cause the condition Limb Girdle Muscular Dystrophy type R9 (LGMDR9) also known as LGMD2I, and the rarer conditions Congenital Muscular Dystrophy (MDC1C), Muscle Eye Brain…
- Conditions
- LGMD2I, LGMDR9, Limb Girdle Muscular Dystrophy, Congenital Muscular Dystrophy
- Trial name
- NCT04001595
- Testing
- Patient Registry
- Where
- United Kingdom
- Volunteers
- 1,000 people
- Sponsor
- Newcastle University
Mitochondrial Disorders, Mitochondrial Genetic Disorders
North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)
The North American Mitochondrial Disease Consortium (NAMDC) maintains a patient contact registry and tissue biorepository for patients with mitochondrial disorders.
- Conditions
- Mitochondrial Disorders, Mitochondrial Genetic Disorders, Mitochondrial Diseases, Disorder of Mitochondrial Respiratory Chain Complexes
- Trial name
- NAMDC (NCT01694940)
- Where
- 17 sites in United States, Canada
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Columbia University
Solid Tumor, Adult Central Nervous System Germ Cell Tumor
Busulfan, Melphalan, Topotecan Hydrochloride, and a Stem Cell Transplant in Treating Patients With Newly Diagnosed or Relapsed Solid Tumor
RATIONALE: Giving high-dose chemotherapy before an autologous stem cell transplant stops the growth of tumor cells by stopping them from dividing or killing them.
- Conditions
- Solid Tumor, Adult Central Nervous System Germ Cell Tumor, Adult Rhabdomyosarcoma, Childhood Central Nervous System Germ Cell Tumor
- Trial name
- NCT00638898
- Testing
- busulfan, melphalan, topotecan hydrochloride
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- City of Hope Medical Center
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
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
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
Concussion, Brain, Musculoskeletal Pain
The Impact of Eye and Body Training on the Injury Rate of Concussions in Female Soccer Players
The research project aims to find out how special training influences the frequency and severity of injuries among female footballers in the top Swiss league.
- Trial name
- NCT06473883
- Testing
- Training recommendation
- Volunteers
- 209 people, healthy volunteers welcome
- Sponsor
- BrainCare Medical Group
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
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
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
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
Brain and Central Nervous System Tumors, Intraocular Melanoma
Iodine I 131 Monoclonal Antibody 3F8 in Treating Patients With Central Nervous System Cancer or Leptomeningeal Cancer
RATIONALE: Radiolabeled monoclonal antibodies, such as iodine I 131 monoclonal antibody 3F8, can find tumor cells and carry tumor-killing substances to them without harming normal cells.
- Conditions
- Brain and Central Nervous System Tumors, Intraocular Melanoma, Lung Cancer, Melanoma (Skin)
- Trial name
- NCT00445965
- Testing
- DNA analysis, immunologic technique, pharmacological study
- Where
- United States
- Volunteers
- 78 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
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
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