COURAGE–ALS (21-2598)   

• Investigator: Dianna Quan, MD  
• Purpose: to evaluate the efficacy and safety of reldesemtiv as a possible treatment for the improvement of skeletal muscle function in conditions associated with muscle weakness or fatigue such as ALS.
• Intervention: reldesemtiv (66% chance) or placebo (33% chance) for first 24 weeks but everyone will receive reldesemtiv for the next 24 weeks.
• Timeline: 8 in-person study visits over 52 weeks
• ClinicalTrials.gov Identifier: NCT04944784
• Key Eligibility Criteria: age 18-80 years old, diagnosis: ALS, onset of ALS symptoms less than 24 months ago, able to swallow pills.
  

Himalaya (22-0037)

• Investigator: Thomas Ragole, MD
• Purpose: to evaluate the investigational drug SAR443820 compared to placebo (which has no active medication) in addition to the standard of care, (e.g. riluzole or edaravone) for ALS. The study will evaluate the effectiveness (how well it works) and the possible risks of taking SAR443820.
• Intervention: SAR443820 (66% chance) or placebo (33% chance) during first 24 weeks but then everyone will receive SAR443820 during next approximately 1.5 years.
• Timeline: 14 in-person visits over 2 years.
• ClinicalTrials.gov Identifier: NCT05237284
• Key Eligibility Criteria: age: 18-80 years old, diagnosis: ALS, Onset of ALS symptoms less than 24 months ago, able to swallow pills.
  

Oral Edaravone (20-2340)

• Investigator: Stacy Dixon, MD, PhD  
• Purpose: to test if an experimental drug called edaravone (Radicava) is effective and safe and to compare its effects when it is given in 2 different ways.
• Intervention: oral form of edaravone, taken daily for 28 days (50% chance) or taken daily for 14 days followed by 14 days of placebo (50% chance).
• Timeline: 8 in-person visits over 58 weeks.
• ClinicalTrials.gov Identifier: NCT04569084  
• Key Eligibility Criteria: age 18-75 years old, diagnosis: ALS, onset of ALS symptoms less than 2 years ago, no previous treatment with edaravone.
  

REFINE-ALS (19-1560)

• Investigator: Laura Foster, MD  
• Purpose: to learn what changes happen in patients with Amyotrophic Lateral Sclerosis (ALS) that can be seen in the blood and urine when they take edaravone (Radicava). These changes are called biomarkers.  
• Timeline: 8 study visits over 24 weeks.
• ClinicalTrials.gov Identifier: NCT04259255 
• Key Eligibility Criteria: age 18+ years old, diagnosis: ALS, planning to start edaravone (Radicava) or received first does within last 28 days.
  

CANYON (22-0354)

• Investigator: Matthew Wicklund, MD
• Purpose: to learn about the safety, and effectiveness of EDG-5506 when compared to placebo in individuals diagnosed with Becker muscular dystrophy.
• Intervention: EDG-5506 (at least 66% chance) or placebo (no greater than 33% chance).
• Timeline: 10 in-person study visits over 14 months.
• ClinicalTrials.gov Identifier: NCT05291091
• Key Eligibility Criteria: age: 12-50 years old, diagnosis: Becker muscular dystrophy, gender: male at birth, able to walk 100m without assistance.
  

GRASP BMD (22-1278)

• Investigator: Stacy Dixon, MD, PhD
• Purpose: to learn more about Becker Muscular Dystrophy by measuring how your muscles change over time. This information will help plan future studies and drug development for people with BMD.
• Timeline: 5 in-person study visits over 24 months.
• ClinicalTrials.gov Identifier: NCT05257473
• Key Eligibility Criteria: age: 8+ years old, diagnosis: Becker Muscular Dystrophy.
  

Genetics of CMT (20-1525)

• Investigator: Vera Fridman, MD  
• Purpose: to look for new genes that cause Charcot Marie Tooth disease (CMT) and to look for genes that don’t cause CMT, but may modify the symptoms a person has.
• ClinicalTrials.gov Identifier: NCT01193088 
• Key Eligibility Criteria: diagnosis: CMT or have a relative with CMT.

Natural History of CMT (18-2537)

• Investigator: Vera Fridman, MD  
• Purpose: observational, longitudinal study to determine natural history and genotype-phenotype correlations of disease causing mutations in Charcot Marie Tooth disease (CMT).
• ClinicalTrials.gov Identifier: NCT01193075  
• Key Eligibility Criteria: diagnosis: CMT or have a relative with CMT.
  

DN (19-3004)

• Study Title: Participants Needed for Research Study About Diabetes and Neuropathy.
• Investigator: Vera Fridman, MD
• Purpose: to learn more about the underlying causes of diabetic neuropathy by looking for the presence of abnormal molecules in the blood and skin and comparing them in people with and without Type 2 Diabetes.
• Procedures: blood draw, skin biopsy, neurological and physical exam, autonomic nervous system testing, nerve conduction study, questionnaires and balance, coordination, and agility tests.
• Timeline: 2 study visits which will occur within 30 days of each other.
• Key Eligibility Criteria: age: 45-65 years old, diagnosis: Type 2 Diabetes (T2D)

MOVE FSHD (20-0405)

• Investigator: Matthew Wicklund, MD  
• Purpose: to collect motor and functional outcomes specific to facioscapulohumeral muscular dystrophy (FSHD) over time to ensure the best level of clinical care and to speed up drug development by gaining a better understanding of how having FSHD impacts motor function and other health outcomes.
• Timeline: 1 study visit per year for 3 years.
• ClinicalTrials.gov Identifier: NCT04635891 
• Key Eligibility Criteria: diagnosis: FSHD Type 1 or 2, genetically confirmed or family history and functionally affected.
  

REACH (22-1143)

• Investigator: Matthew Wicklund, MD
• Purpose: to evaluate whether losmapimod may slow down the progression of the disease in individuals with FSHD1 and FSHD2.
• Intervention: losmapimod (50% chance) or placebo (50% chance).
• Timeline: 8 in-person study visits over 53 weeks.
• ClinicalTrials.gov Identifier:  NCT05397470
• Key Eligibility Criteria: age: 18-65 years old, diagnosis: FSHD1 or FSHD2 verified by genetic testing, not dependent upon wheelchair or walker.
  

INSPIRE IBM (21-3383)

• Investigator: Matthew Wicklund, MD
  
• Purpose: to follow and assess people with diagnosed sporadic inclusion body myositis over time to further understand and characterize the disease with the intent to improve future clinical trials. We are also trying to see if there are specific biomarkers that can help predict disease severity.
• Timeline: 5-hour visit every 6 months for approximately 2 years.
• ClinicalTrials.gov Identifier: NCT05046821
• Key Eligibility Criteria: age: 40+ years old, diagnosis: Sporadic Inclusion Body Myositis (sIBM), disease onset is within the past 10 years of the time of Baseline Visit.
  

LGMD GRASP (19-0506)

• Investigator: Stacy Dixon, MD, PhD
• Purpose: to learn more about limb girdle muscular dystrophy (LGMD) by measuring how your muscles change over time.
• Timeline: 4-5 study visits over 12 months.
• ClinicalTrials.gov Identifier: NCT03981289
• Key Eligibility Criteria: age 4-65 years old, diagnosis: clinically affected LGMD (weakness in limb girdle pattern or in distal extremity).
  

MINT (20-1787)

• Investigator: Dianna Quan, MD 
• Purpose: to determine whether the investigational drug inebilizumab is safe and effective in reducing the symptoms of myasthenia gravis.
• Intervention: inebilizumab (50% chance) or placebo (50% chance).
• Timeline: up to 12 visits over up to 13 months (depends on what type of MG you have), open label extension available.
• Clinicaltrials.gov Identifier: NCT04524273  
• Key Eligibility Criteria: age 18+ years old, diagnosis: AChR or MuSK myasthenia gravis, currently taking (1) corticosteroids (2) non-steroidal IST or a combination of the 2.
  

Vivacity MG3 (21-3289)

• Investigator: Dianna Quan, MD 
• Purpose: to see if nipocalimab is safe and useful for treating adults with generalized myasthenia gravis.
• Intervention: nipocalimab infusion (50% chance) or placebo (50% chance).
• Timeline: 13 visits over 24 weeks, (includes infusion every 2 weeks) open label extension available.
• Clinicaltrials.gov Identifier: NCT04951622
• Key Eligibility Criteria: age 18+ years old, diagnosis: myasthenia gravis with generalized muscle weakness.
  

END-DM1 (21-4907)

• Investigator: Thomas Ragole, MD
  
• Purpose: to determine the best ways to assess how people are affected by DM1.  The study will examine the effects of DM1 on your muscles, heart, blood, and nervous system.
• Timeline: up to 4 in person study visits over 2 years.
• ClinicalTrials.gov Identifier: NCT03981575 
• Key Eligibility Criteria: age: 18-70 years old, diagnosis: type 1 myotonic dystrophy (DM1).

Harmony (21-3397)

• Investigator: Matthew Wicklund, MD  
• Purpose: to study pitolisant, an investigational medication, for the treatment of excessive daytime sleepiness, fatigue and cognitive dysfunction in adults with type 1 myotonic dystrophy.
• Intervention: you have a 1 in 3 chance of receiving either low dose pitolisant, high dose pitolisant, or placebo during the initial 11 weeks of the study, but everyone who participates in the final 55 weeks of the study will receive pitolisant.
• Timeline: 8 in-person visits over approximately 1.5 years.
• ClinicalTrials.gov Identifier: NCT04886518
• Key Eligibility Criteria: age: 18-65 years old, diagnosis: genetically confirmed diagnosis of type 1 myotonic dystrophy (DM1), able to walk independently for at least 10 meters.

WeSMA (22-0296)

• Investigator: Stacy Dixon, MD, PhD
• Purpose: to follow patients receiving Evrysdi (risdiplam) for a long time.  This will add to the body of evidence about the safety and effectiveness of Evrysdi.
• Intervention: Evrysdi.
• Timeline: visits every 6 months for up to 5 years.
• ClinicalTrials.gov Identifier:  NCT05232929 
• Key Eligibility Criteria: diagnosis: spinal muscular atrophy, currently prescribed Evrysdi.