Neuromuscular Clinical Trials

Our Research

Our research division works with rare neuromuscular diseases encompassing several conditions such as amyotrophic lateral sclerosis (ALS), Facioscapulohumeral Muscular Dystrophy (FSHD), spinal muscular atrophy (SMA), idiopathic painful neuropathies (CIAP), CIDP – full, myasthenia gravis, facioscapulohumeral dystrophy, and limb girdle muscular dystrophies. The clinics are housed at both the UW Montlake Medical Center and Seattle Children’s Hospital.

Enrolling Trials

Target ALS biomarker Study; Longitudinal Biofluids, Clinical Measures, and At Home Measures (TALSLB)
The goal of the study is to generate a biorepository of longitudinal blood (plasma and serum), cerebral spinal fluid (CSF) and urine linked to genetics and longitudinal clinical information that are made available to the research community.
Sponsor: Healey Center/Massachusetts General Hospital
Site PI: Michael Weiss, MD
Contact: Kinsey Chapman (kinseyc@uw.edu), Kaycie Opiyo (tkaycie@uw.edu)
ClinicalTrials.gov Identifier NCT05137665

ACCESS ALS
Sponsor: St. Joseph’s Hospital and Medical Center, Phoenix
Site PI: Michael Weiss, MD
Contact: Kinsey Chapman (kinseyc@uw.edu)
ClinicalTrials.gov Identifier: NCT06578195
This is a 24 month observational study of participants with ALS and healthy controls. The goal of
this study is to collect blood, cerebrospinal fluid (optional), data via questionnaires, and physical
assessments at multiple visits over the course of the study.

PREVENT ALS
Sponsor: St. Joseph’s Hospital and Medical Center, Phoenix
Site PI: Michael Weiss, MD
Contact: Kinsey Chapman (kinseyc@uw.edu)
ClinicalTrials.gov Identifier: NCT06581861
This is an observational study in asymptomatic participants at risk of ALS with the goal of
collecting biofluid samples including blood specimens and cerebrospinal fluid with a lumbar
puncture. Participants will be eligible if DNA analysis concludes that they carry gene variants
that may cause ALS or a related disorder, frontotemporal dementia.

A Phase 2b, Multi-center, Randomized, Quadruple-blind, Placebo Controlled Study of Batoclimab Treatment in Adult Participants with Active Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
Sponsor: Immunovant Sciences, GmbH
Site PI: Michael Weiss, MD
Contact: Kinsey Chapman (kinseyc@uw.edu)
ClinicalTrials.gov Identifier: NCT05581199
This is a blinded placebo-controlled 52 week trial of CIDP patients using batoclimab, a self
injectable neonatal Fc receptor inhibitor

A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice (EVOLVE)
Sponsor: Sarepta Therapeutics, Inc.
Site PI: Sean Perlman, MD
Contact: Marrisa Robertson (Marissa.Robertson@seattlechildrens.org) and Macarena Racciatti
(Macarena.Racciatti@seattlechildrens.org)
ClinicalTrials.gov Identifier: NCT06606340
This is an observational study to collect medical history and prospective data on participants
with DMD receiving eteplirsen, golodirsen, and casimersen in clinical practice (prescription
needed before entry to study).

Phase 1/2 Study of AOC 1020 in Adults With Facioscapulohumeral Muscular Dystrophy (FSHD) (FORTITUDE)
A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Adult Participants with Facioscapulohumeral Muscular Dystrophy (FSHD).
Sponsor: Avidity Biosciences, Inc
Site PI: Leo Wang, MD
Contact: Mike Willis (mwillis5@uw.edu)
ClinicalTrials.gov Identifier NCT05747924

Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD)
Sponsor: University of Kansas Medical Center
Site PI: Leo Wang, MD
Contact: Lilly Young
ClinicalTrials.gov Identifier: NCT04635891
This a longitudinal observational study in FSHD-diagnosed participants. Participants will have
blood, physical assessments, and MRI studies as biomarkers to track disease progression.

A Study to Investigate the Efficacy and Safety of Efgartigimod PH20 SC in Adult Participants With Active Idiopathic Inflammatory Myopathy. (ALKIVIA)
This study’s purpose is to measure the treatment response from efgartigimod PH20 SC compared with placebo in participants with Idiopathic Inflammatory Myopathy (IIM). Participants with the IIM subtypes of dermatomyositis (DM), immune-mediated necrotizing myopathy (IMNM), or certain other subtypes of polymyositis (PM; including antisynthetase syndrome [ASyS]) will be included in the study.
Sponsor: Argenx
Site PI: Leo Wang, MD
Contact: Dani Dixon (daniedxn@uw.edu)
ClinicalTrials.gov Identifier NCT05523167

A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)
The purpose of this study is to evaluate the safety and tolerability of a single intravenous infusion of AB-1003 in adults diagnosed with limb girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). Participants will be treated in sequential, dose-level cohorts.
Sponsor: Asklepios Biopharmaceutical, Inc
Site PI: Jane Distad, MD
Contact: Mike Willis (mwillis5@uw.edu)
ClinicalTrials.gov Identifier NCT05230459

EPPIC-Net: Novaremed Painful Diabetic Peripheral Neuropathy ISA (EN21-01
The purpose of this study is to investigate the safety and efficacy of the current hard gelatin capsule formulation of NRD135S.E1 80 mg once daily in the treatment of PDPN when administered for 13 weeks.
Sponsor: NIH
Site PI: Max Ma, MD
Contact: Brianna Zhou (bri1106@uw.edu)
ClinicalTrials.gov Identifier NCT05480228

Spinraza in Adult Spinal Muscular Atrophy (SMA)
This is an observational study of adult SMA patients on Spinraza.
Sponsor: Washington University
Site-PI: Nassim Rad, MD
Contact: Dani Dixon (daniedxn@uw.edu)
ClinicalTrials.gov Identifier NCT03709784

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): Seth Perlman, MD
Contacts: Marissa Robertson (Marissa.Robertson@seattlechildrens.org), Macarena Racciatti
(Macarena.Racciatti@seattlechildrens.org)
ClinicalTrials.gov Identifier NCT04174157