Clinical trials: Myasthenic Syndrome
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.
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
Myasthenic Crisis, Upper Respiratory Infection
Incidence and Risk of Influenza in Myasthenic Patients
Upper respiratory infection , including influenza, may exacerbate the symptoms of myasthenia gravis (MG), which is an autoimmune disease that causes muscle weakness and, in some cases, can precipitate a myasthenic crisis In the…
- Trial name
- NCT03884777
- Volunteers
- 400 people
- Sponsor
- Centre d'Investigation Clinique et Technologique 805
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
Lambert-Eaton Myasthenic Syndrome
Phase 3 Study to Evaluate Efficacy of Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS)
This study evaluates the effect of withdrawing amifampridine phosphate treatment from patients with LEMS. One half of the patients will continue to receive amifampridine phosphate and the other half will receive placebo, during…
- Trial name
- NCT02970162
- Testing
- Amifampridine Phosphate
- Where
- United States
- Volunteers
- 26 people
- Sponsor
- Catalyst Pharmaceuticals, Inc.
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.
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
Autoimmune Myasthenia Gravis, Lambert-Eaton Myasthenic Syndrome
Exercise in Autoimmune Myasthenia Gravis and Myasthenic Syndromes
A few recent observational studies show that despite the lack of clear recommendations, many individuals with myasthenia participate in exercise.
- Conditions
- Autoimmune Myasthenia Gravis, Lambert-Eaton Myasthenic Syndrome, Congenital Myasthenic Syndrome, Myasthenia Gravis
- Trial name
- MYaEX (NCT05408702)
- Where
- France
- Volunteers
- 500 people
- Sponsor
- Institut de Myologie, France
Myasthenia Gravis
Follow-up of a Cohort of Patients With Myasthenic Syndrome and COVID-19 Infection
Coronavirus disease 2019 (COVID-19), declared by the World Health Organization (WHO) as a "public health emergency of international concern" (January 31, 2020), has posed a significant threat to global health.
- Trial name
- CO-MY-COVID (NCT04695379)
- Where
- 10 sites in France
- Volunteers
- 34 people
- Sponsor
- University Hospital, Bordeaux
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 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 Myasthenia Syndrome
Evaluating the Safety and Effects of AMP-101 in People With DOK7 Congenital Myasthenia Syndrome
This study is evaluating the safety and potential effects of AMP-101 in people with DOK7 Congenital Myasthenia Syndrome (CMS). Participants will receive a single dose of the study treatment and will be monitored to assess their…
- Trial name
- NCT07830563
- Testing
- AMP-101
- Volunteers
- 4 people
- Sponsor
- Amplo Biotechnology
Myasthenic Syndromes, Congenital
Ephedrine for the Treatment of Congenital Myasthenia
Previous research has demonstrated possible efficacy of Ephedrine in the treatment of congenital myasthenia caused by end-plate acetylcholinesterase deficiency.
- Trial name
- NCT00541216
- Testing
- Ephedrine
- Where
- Israel
- Volunteers
- 15 people
- Sponsor
- Hadassah Medical Organization
Congenital Myasthenic Syndrome
Pregnancy Outcomes in Congenital Myasthenie Syndrome
This is a retrospective study that follows the clinical evolution, the pregnancy and the post-partum perioad of female patients with Congenital Myasthenic Syndrome.
- Trial name
- POCoMS (NCT01474980)
- Volunteers
- 25 people
- Sponsor
- Institut de Myologie, France
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
Lambert-Eaton Myasthenic Syndrome
Use Of 3,4-Diaminopyridine (3,4-DAP) In The Treatment Of Lambert Eaton Myasthenic Syndrome
Compassionate use of orphan drug 3,4-Diaminopyridine(DAP) in Treatment of Lambert Eaton Myasthenic Syndrome (LEMS). 3,4-DAP is used to decrease the muscle weakness associated with LEMS and hopefully will decrease the need for…
- Trial name
- 34-DAP (NCT01373333)
- Testing
- 3,4 DAP
- Where
- United States
- Sponsor
- The Cleveland Clinic
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.
Myasthenia Gravis, Myasthenic Crisis
Study of Myasthenic Crisis in China
This study is a multicenter, prospective, observational research. It will register the basic demographic information, medical history characteristics, clinical features, auxiliary examinations, treatment, outcomes of tracheal…
- Trial name
- NCT04837625
- Testing
- no interventions
- Where
- China
- Volunteers
- 300 people
- Sponsor
- Huashan Hospital
Lambert-Eaton Myasthenic Syndrome
Treatment of Lambert-Eaton Syndrome With 3,4 Diaminopyridine
The purpose of the study is to provide expanded access (compassionate use) of 3,4 diaminopyridine to patients with Lambert-Eaton myasthenic syndrome (LEMS).
- Trial name
- NCT00994916
- Testing
- 3,4 diaminopyridine
- Sponsor
- David Lacomis, MD
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