Clinical trials: Lambert-eaton Myasthenic Syndrome
Congenital Myasthenic Syndrome, CMS
A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS)
The purpose of this study is to assess the safety and tolerability of ARGX-119 in adult participants with DOK7- Congenital Myasthenic Syndromes.
- Trial name
- NCT06436742
- Testing
- ARGX-119
- Where
- 9 sites in United States, Canada and 4 more
- Volunteers
- 15 people
- Sponsor
- argenx
Myasthenic Syndromes, Congenital
Treatment Use of 3,4-Diaminopyridine
This protocol provided 3,4 diaminopyridine (DAP) under a treatment-use IND to patients with congenital myasthenic syndrome (CMS). It is now closed.
- Trial name
- NCT01765140
- Testing
- 3,4-diaminopyridine
- Where
- United States
- Sponsor
- Vern C. Juel, M.D.
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
Spinal Muscular Atrophy, Muscular Dystrophy
National Registry for Egyptian Pediatric Neuromuscular Diseases
Our aim is to establish multi-center national Egyptian database of information for inherited and acquired neuromuscular diseases in infants and children from 0 to 18 years of age.
- Conditions
- Spinal Muscular Atrophy, Muscular Dystrophy, Muscle Diseases, Myasthenic Syndromes
- Trial name
- NCT02124616
- Where
- Egypt
- Volunteers
- 200 people
- Sponsor
- Ain Shams University
Lambert Eaton Myasthenic Syndrome
Use of 3,4-Diaminopyridine in the Treatment of Lambert-Eaton Syndrome
To learn more about the effect of 3, 4-Diaminopyridine (3,4-DAP) on patient diagnosed with Lambert-Eaton myasthenic syndrome (LEMS).
- Trial name
- 3 4-DAP (NCT01825395)
- Testing
- 3, 4-Diaminopyridine
- Where
- United States
- Sponsor
- University of Colorado, Denver
Congenital Myasthenic Syndrome, CMS
A Study to Assess Efficacy and Safety of Adimanebart in Adult and Pediatric Participants With DOK7-,MUSK-, AGRN-, or LRP4- Congenital Myasthenic Syndromes (CMS)
The purpose of this study is to assess efficacy and safety of adimanebart in participants at least 12 years of age with DOK7-, MUSK-, AGRN-, or LRP4- Congenital Myasthenic Syndromes (CMS).
- Trial name
- CoMetS (NCT07746089)
- Testing
- Adimanebart IV
- Volunteers
- 105 people
- Sponsor
- argenx
Inclusion Body Myositis, Myotonic Dystrophy 1
The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.
The planned project is an intervention study to assess the risk of falling after adaptation of an assistive gait devices in patients with the following neuromuscular diseases: Inclusion body myositis, myotonic dystrophy, limb…
- Conditions
- Inclusion Body Myositis, Myotonic Dystrophy 1, Myotonic Dystrophy 2, Facio-Scapulo-Humeral Dystrophy
- Trial name
- Risk of fall (NCT07072676)
- Testing
- Assistive gait devices combined with physiotherapy
- Where
- Germany
- Volunteers
- 30 people
- Sponsor
- LMU Klinikum
Lambert Eaton Myasthenic Syndrome
Treatment of Lambert-Eaton Syndrome With 3,4 DAP
A new drug called 3,4-Diaminopyridine (3,4-DAP) is currently under investigation for treatment of the symptoms of Lambert-Eaton Myasthenic Syndrome (LEMS).
- Trial name
- NCT00704925
- Testing
- 3, 4 DAP
- Where
- United States
- Sponsor
- David P. Richman, MD
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
CoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux Falls, South Dakota. It provides researchers with a centralized, international patient registry for all rare diseases.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- 2 sites in United States, Australia
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Lambert-Eaton Myasthenic Syndrome (LEMS), Congenital Myasthenia (CM)
3,4-Diaminopyridine for Lambert-Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenia (CM)
Lambert-Eaton Myasthenic Syndrome (LEMS) is a rare autoimmune disorder which affects the nerve-muscle junction. The major symptoms of LEMS are progressive muscle weakness.
- Trial name
- NCT02012933
- Testing
- 3,4-diaminopyridine
- Where
- United States
- Sponsor
- Oregon Health and Science University
Congenital Myasthenic Syndrome
A Natural History Study in Participants With Congenital Myasthenic Syndromes (CMS) Due to Mutations in DOK7, MUSK, AGRN, or LRP4
Participants will attend up to 4 study visits to collect clinical assessments. The assessments will evaluate participants' symptoms and quality of life to understand disease activity in patients with CMS due to mutations in DOK7…
- Trial name
- NCT06078553
- Where
- 33 sites in United States, Austria and 10 more
- Volunteers
- 100 people
- Sponsor
- argenx
Myasthenia Gravis, Myasthenic Syndrome
Patient Observation With Environmental and Wearable Sensors in Myasthenia Gravis
This observational study seeks to investigate the underlying processes of myasthenia gravis by employing multimodal monitoring techniques.
- Conditions
- Myasthenia Gravis, Myasthenic Syndrome, Lambert-Eaton Myasthenic Syndrome
- Trial name
- POWER-MG (NCT06441825)
- Testing
- Increased multimodal observation including digital monitoring
- Where
- Germany
- Volunteers
- 20 people
- Sponsor
- Heinrich-Heine University, Duesseldorf
Lambert-Eaton Myasthenic Syndrome, Eaton-Lambert Myasthenic Syndrome
Effectiveness of 3,4-Diaminopyridine in Lambert-Eaton Myasthenic Syndrome
Hypothesis: 3,4-Diaminopyridine base (3,4-DAP) improves Lambert-Eaton Myasthenic Syndrome (LEMS)-related weakness.
- Trial name
- DAPPER (NCT01511978)
- Testing
- Continuous 3,4-DAP
- Where
- 7 sites in United States
- Volunteers
- 32 people
- Sponsor
- Jacobus Pharmaceutical
Congenital Myasthenic Syndrome, Lambert-Eaton Myasthenic Syndrome
Expanded Access Study Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS),Congenital Myasthenic Syndrome
Primary: The primary objective of this study under the original protocol was to provide neuromuscular specialists and neurologists access to amifampridine phosphate therapy for their patients with LEMS, CMS or downbeat nystagmus…
- Conditions
- Congenital Myasthenic Syndrome, Lambert-Eaton Myasthenic Syndrome, Nystagmus, Acquired
- Trial name
- EAP-001 (NCT02189720)
- Testing
- Amifampridine Phosphate
- Where
- United States
- Sponsor
- Catalyst Pharmaceuticals, Inc.
Small Cell Lung Cancer ( SCLC ), Lambert Eaton Myasthenic Syndrome (LEMS)
A Study to Evaluate the Incidence of Clinically Suspicious Lambert-Eaton Myasthenic Syndrome (LEMS) in Subjects Diagnosed With Small Cell Lung Cancer (SCLC)
This is a multi-center observational study in subjects with a diagnosis of small cell lung cancer. Screening and/or medical record review for symptoms associated with Lambert Eaton Myasthenic Syndrome (LEMS) will be conducted.
- Trial name
- NCT07075627
- Where
- United States
- Sponsor
- Addario Lung Cancer Medical Institute
Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1
Baby Detect : Genomic Newborn Screening
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions.
- Conditions
- Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1, Phosphoglucomutase 1 Deficiency, Maturity Onset Diabetes of the Young
- Trial name
- BabyDetect (NCT05687474)
- Where
- Belgium
- Volunteers
- 6,824 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
Congenital Myasthenic Syndrome
Patients With Congenital Myasthenic Syndrome Will be Treated With Umbilical or Placental Derived Mesenchymal Stem Cell Exosome Solution
Patients with Congenital Myasthenic Syndrome will be treated with Umbilical or Placental Derived Mesenchymal Stem Cell Exosome solution.
- Trial name
- NCT07226726
- Testing
- AlloEx exosomes
- Where
- 2 sites in United States, Greece
- Volunteers
- 20 people
- Sponsor
- The Foundation for Orthopaedics and Regenerative Medicine
Autoimmune Disease, Neurologic Autoimmune Disease
Autologous Stem Cell Transplant for Neurologic Autoimmune Diseases
This phase II trial studies the side effects and how well carmustine, etoposide, cytarabine and melphalan together with antithymocyte globulin before a stem cell transplant works in treating patients with autoimmune neurologic…
- Conditions
- Autoimmune Disease, Neurologic Autoimmune Disease, Autologous Transplant Autoimmune, Multiple Sclerosis Transplant
- Trial name
- NCT00716066
- Testing
- Anti-Thymocyte Globulin, Autologous Hematopoietic Stem Cell Transplantation, Carmustine
- Where
- 3 sites in United States
- Volunteers
- 53 people
- Sponsor
- Fred Hutchinson Cancer Center
Lambert Eaton Myasthenic Syndrome
A Phase 3 Study of Amifampridine Phosphate in Patients With Lambert Eaton Myasthenic Syndrome (LEMS)
A Phase 3 study to evaluate the efficacy and safety of Amifampridine Phosphate in patients with Lambert-Eaton Myasthenic Syndrome (LEMS).
- Trial name
- NCT01377922
- Testing
- Amifampridine Phosphate
- Where
- 14 sites in United States, France and 6 more
- Volunteers
- 38 people
- Sponsor
- Catalyst Pharmaceuticals, Inc.
Congenital Myasthenic Syndrome
Efficacy of Albuterol in the Treatment of Congenital Myasthenic Syndromes
The study tests the notion that patients suffering from certain types of congenital myasthenic syndromes are benefitted by the use of Albuterol at doses used in clinical practice.
- Trial name
- NCT01203592
- Testing
- Albuterol
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Mayo Clinic