HEALEY ALS Platform Trial (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
• Key Eligibility Criteria: diagnosis: ALS, onset of ALS symptoms less than 36 months ago, able to swallow pills.
  

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)

FORTITUDE (22-2179)

• Investigator: Dianna Quan, MD
• Purpose: to determine whether an investigational drug is safe and effective (works well) for the treatment of Facioscapulohumeral Muscular Dystrophy (FSHD).
• Intervention: investigational drug or placebo (chance of receiving investigational drug will vary between Parts).
• Timeline:
  • Screening period (up to 6 weeks)
  • Baseline visit(s) (up to 21 days before starting treatment period)
  • Treatment period (39 weeks). This study has 3 parts, Part A, Part B, and Part C. The different parts of the study will test increasing dose levels of AOC 1020 to see how safe it is, how well the body handles taking it (tolerability), what the body does to the study drug (pharmacokinetics), and what the study drug does to the body (pharmacodynamics). The part each participant is assigned to will depend on when they join the study
  • Post-treatment Follow-up period (13 weeks)
• ClinicalTrials.gov Identifier: NCT05747924
• Key Eligibility Criteria: age: 18 to 65 years old (inclusive) at time of informed consent, diagnosis: Facioscapulohumeral Muscular Dystrophy (FSHD) confirmed by documented genetic diagnosis, ambulatory and able to walk 10 meters (with or without assistive devices such as one cane, walking stick or braces)
  

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.
  

ROCHE (22-1495)

• Investigator: Stacy Dixon, MD, PhD
• Purpose: is to compare the effects, good or bad, of RO7204239 versus placebo on patients with FSHD.
• Intervention: RO7204239 (50% chance) or placebo (50% chance) for first 52 weeks, everyone will receive RO7204239 for final 52 weeks.
• Timeline: approximately 26 in-person visits over 52 weeks.
• ClinicalTrials.gov Identifier: NCT05548556
• Key Eligibility Criteria: age: 18-65 years old, diagnosis: FSHD1 or FSHD2, able to walk unassisted.
  

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.
  

GRASP LGMD (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).
  

FLEX (22-2391)

• Investigator: Thomas Ragole, MD
• Purpose: to learn how well batoclimab works and how safe it is, when compared with placebo (an inactive material that looks like batoclimab but does not have any active drug).
• Intervention: batoclimab or placebo (chance of receiving batoclimab will vary between Periods).
• Timeline: Screening Period of up to 4 weeks, two 12-week Treatment Periods (referred to as Period 1 and Period 2), a 52-week Long-Term Extension Period (referred to as Period 3), and a 4 week Follow-up Period. Total study duration: approximately 84 weeks (1 ½ years), approximately 34 study visits.
• ClinicalTrials.gov Identifier: NCT05403541
• Key Eligibility Criteria: age: ≥ 18 years, diagnosis: mild to severe Generalized Myasthenia Gravis (gMG), have diagnostic autoantibody (AChRAb+, anti-MuSK+, or anti-LRP4+) confirmation of gMG at the Screening Visit.
  

Prevail (22-1330)

• Investigator: Thomas Ragole, MD
• Purpose: to determine if the study medication (ALXN1720) is safe and effective in the treatment of generalized Myasthenia Gravis (gMG).

• Intervention: ALXN1720 (50% chance) or placebo (50% chance).
• Timeline: 24 in-person visits over 2.5 years.
• ClinicalTrials.gov Identifier: NCT05556096
• Key Eligibility Criteria: age: 18+ years old, diagnosis: generalized Myasthenia Gravis (gMG)
  

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).

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.