Department of Neurology

Neuromuscular Clinical Trials

ALS Healey Trial Platform

These are multi-drug phase 3 ALS studies using a unique trial design that utilizes the same placebo population – current therapeutics trehalose

Sponsor: Healey Center/MGH
Site PI: Weiss
Contact: Kassie Chu (kasanac@uw.edu)
ClinicalTrials.gov Identifier NCT04297683

ALS Healey Trial Platform Open Label Extension

These are open label extension studies for the Healey drug regimens – current therapeutics CNM-AU8, trehalose, and pridopidine

Sponsor: Healey Center/MGH
Site PI: Weiss
Contact: Amos Sahu (adsahu@uw.edu)
ClinicalTrials.gov Identifier NCT04297683

ALS Oral Edaravone (A02 Study)

This is a phase 3 study for ALS comparing two oral dosing regimens of edaravone.

Sponsor: Mitsubishi Tanabe Pharma
Site PI: Weiss
Contact: Amos Sahu (adsahu@uw.edu)
ClinicalTrials.gov Identifier NCT04569084

RAISE-XT

This is the open label extension of the phase 3 zilucoplan study in subjects with generalized myasthenia gravis.

Sponsor: UCB-Ra Pharma
Site PI: Weiss
Contact: Amos Sahu (adsahu@uw.edu)
ClinicalTrials.gov Identifier NCT04225871

Charcot-Marie-Tooth disease Type 1A PREMIER Study

This is a phase 3 study assessing the efficacy of the medication PXT3003 for the treatment of Charcot-Marie-Tooth disease type 1A.

Sponsor: Pharnext
Site PI: Weiss
Contact: Amos Sahu (adsahu@uw.edu)
ClinicalTrials.gov Identifier NCT04762758

Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD)

This is a prospective study designed to collect motor and functional outcomes specific to FSHD over time

Sponsor: University of Kansas
Site PI: Wang
Contact: Aliya Shabbir (aliya15@uw.edu)
ClinicalTrials.gov Identifier NCT04635891

Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients with Myotonic Dystrophy Type 1

The primary objective of this study is to evaluate the safety and efficacy of pitolisant compared with placebo in treating excessive daytime sleepiness (EDS) in patients with Myotonic Dystrophy Type 1 ages 18 to 65 years.                  

Sponsor: Harmony Biosciences
Site-PI: Wang
Contact: Aliya Shabbir (aliya15@uw.edu)
ClinicalTrials.gov Identifier NCT04886518

Sporadic Inclusion Body Myositis Natural History Study (INSPIRE-IBM)

This is a prospective natural history study on patients with clinically defined sIBM. Participants will be assessed every 6 months over two years (five visits total).

Site-PI: Wang
Contact: Aliya Shabbir (aliya15@uw.edu)
ClinicalTrials.gov Identifier NCT05046821

IONIS | ION-682884-CS3 [Neuro] TTRansform

This is a phase 3 global, open-label, randomized study to evaluate the efficacy and safety of ION-682884 in patients with hereditary transthyretin-mediated amyloid polyneuropathy.

Sponsor: Ionis
Site-PI: Distad, sub-I: Preston
Contact: Aliya Shabbir (aliya15@uw.edu)
ClinicalTrials.gov Identifier NCT04136184

Spinraza in Adult Spinal Muscular Atrophy (SMA)

This is an observational study of adult SMA patients on Spinraza.

Sponsor: Washington University
Site-PI: Rad
Contact: Aliya Shabbir (aliya15@uw.edu)
ClinicalTrials.gov Identifier NCT03709784  

Pamrevlumab or Placebo in Combination with Systemic Corticosteroids in Subjects with Non-Ambulatory Duchenne Muscular Dystrophy (DMD)

This is a phase 3 study of the monoclonal antibody pamrevlumab for the treatment of DMD.

Sponsor: FibroGen
Site PI (Seattle Children’s hospital): Perlman
ClinicalTrials.gov Identifier NCT04371666

Registry of Patients with a Diagnosis of Spinal Muscular Atrophy

This is a registry of SMA patients to assess long-term outcomes with and without medications.

Sponsor: AveXis
Site PI (Seattle Children’s hospital): Perlman
ClinicalTrials.gov Identifier NCT04174157

Painful and Autonomic Idiopathic Neuropathy (PAIN) Genetic Study

This is a prospective study to determine genetic causes of idiopathic neuropathies with associated pain and associated autonomic neuropathy

Sponsor: Aksea
Site PI (Puget Sound VA): Ma
Contact: Justine Marecaux (marecj@uw.edu)

Efficacy and Safety of Losmapimod in Treating Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) (Reach) 

Sponsor: Fulcrum Therapeutics
Site PI: Leo Wang
Contact: Aliya Shabbir (aliya15@uw.edu)
ClinicalTrials.gov Identifier NCT05397470

A Study to Evaluate the Safety of LION-101 in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1) 

Sponsor: Asklepios BioPharmaceutical, Inc.
Site PI: Jane Distad
Contact: Aliya Shabbir (aliya15@uw.edu)
ClinicalTrials.gov Identifier NCT05230459

Argenx: A Study to Investigate the Efficacy and Safety of Efgartigimod PH20 SC in Adult Participants With Active Idiopathic Inflammatory Myopathy. (ALKIVIA) 

Sponsor: Argenx
Site PI: Leo Wang
Contact: Aliya Shabbir (aliya15@uw.edu)
ClinicalTrials.gov Identifier NCT05523167

A Phase 1/2 Study of AOC 1020 in Adults With Facioscapulohumeral Muscular Dystrophy (FSHD) (FORTITUDE)

Sponsor: Avidity Biosciences, Inc.
Site PI: Leo Wang; Sub-I: Nassim Rad 
Contact: Aliya Shabbir (aliya15@uw.edu)
ClinicalTrials.gov Identifier NCT05747924
Study Website: https://fortitude-study.com/