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.
  

Healey Platform Trial for ALS (19-2645)

• Investigator: Laura Foster, MD  
• Purpose: to test the safety and effectiveness of multiple treatments in ALS. A regimen is a specific course of treatment, each with a different study drug.
• Intervention: multiple different regimens may be active within the platform trial at the same time. You can find more information about the currently enrolling regimens here.
• Timeline: 6-8 study visits over 30 weeks.
• Clinicaltrials.gov Identifier: NCT04297683 
  

Natural History Evaluation (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.
  

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.
  

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.
  

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: Matthew Wicklund, MD    
• 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 (22-0296)

• Investigator: Matthew Wicklund, 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.

MARINA (21-3032):

• Investigator: Matthew Wicklund, MD  
• Purpose: to determine whether an investigational new drug,  called AOC 1001, is safe and effective for the treatment of myotonic dystrophy type 1 (DM1).  It is hoped that by decreasing the amount of mRNA, AOC 1001 will improve or stabilize muscle strength, and improve or prevent the worsening of breathing and heart problems.
• Intervention: AOC 1001 (75% chance) or placebo (25% chance).  Both will be given via IV infusion.
• Timeline: approximately 16 in-person visits over 7.5 months.
• 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.
  

THAOS (08-0623)   

• Investigator: Dianna Quan, MD  
• Purpose: to follow patients with transthyretin amyloidosis (ATTR) over time and gather information about the disease process to lead to better treatment.
• ClinicalTrials.gov Identifier: NCT00628745  
• Key Eligibility Criteria: age 18+ years old, diagnosis: TTR amyloidosis.