Department of Neurology

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

Amyotrophic Lateral Sclerosis (ALS)

ALL ASSESS ALS
Sponsor: St. Joseph’s Hospital and Medical Center, Phoenix
Site PI: Michael Weiss, MD
Contact: Kinsey Chapman (kinseyc@uw.edu), Lila Brisk (lbrisk@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), Lila Brisk (lbrisk@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.

Charcot-Marie-Tooth Disease (CMT)

Natural History Evaluation of Charcot Marie Tooth Disease (CMT) Types CMT1B, CMT2A, CMT4A, CMT4C, and Others
Site PI: Seth Perlman, MD
Contact: Marissa Robertson (Marissa.Robertson@seattlechildrens.org) and Christa Rocco (Christa.Rocco@seattlechildrens.org)
Sponsor: Michael Shy
ClinicalTrials.gov ID: NCT01193075
The purpose of this registry is to determine the natural history of CMT1B, CMT2A, CMT4A, CMT4C, and other types of CMT in order to refine the overall picture of disease for use in future clinical trials. Participant’s strength, sensation and how well the peripheral nerves (nerves going to the hands and feet) conduct a signal will be assessed annually. The study is also collecting data to develop a new scoring scale to measure progression of symptoms in children

Chronic Inflammatory Demyelinating Polyneuropathy

A PHASE 3 RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF DNTH103 IN ADULTS WITH CHRONIC INFLAMMATORY DEMYELINATING POLYNEUROPATHY (CAPTIVATE)
Sponsor: Dianthus Therapeutics
Site PI: Michael Weiss, MD
Contact: Kinsey Chapman (kinseyc@uw.edu)
ClinicalTrials.gov Identifier: NCT06858579
The goal of the study is to demonstrate the efficacy of DNTH103 compared to placebo based on the time to relapse. This will be done by maintaining double-blinded protocol for a placebo and DNTH103 (study drug) administered via infusions.
A Phase 2, Open-label, Proof-of-Concept Study to Investigate the Efficacy, Safety, and Tolerability of TAK-411 in Adult Subjects With Chronic Inflammatory Demyelinating Polyradiculoneuropathy (The CASCA Study)
Sponsor: Takeda Development Center Americas, Inc.
Site PI: Michael Weiss, MD
Contact: Ipshita Tripathi (itripa@uw.edu)
ClinicalTrials.gov Identifier: NCT06798012
The goal of the study is to determine the effect of TAK-411 compared with that expected under placebo. As this is a phase 3 study, all participants who are eligible will receive TAK-411(study drug) administered via infusions.

Duchenne Muscular Dystrophy (DMD)

A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice (EVOLVE)
Sponsor: Sarepta Therapeutics, Inc.
Site PI: Seth Perlman, MD
Contact: Marissa 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).
A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
Sponsor Solid Biosciences Inc.
Site PI: Alicia Henriquez, MD, MSCR 
Contact: Marissa Robertson (Marissa.Robertson@seattlechildrens.org) and Janaki O’Brien (Janaki.OBrien@seattlechildrens.org)
ClinicalTrials.gov ID: NCT06138639
INSPIRE DUCHENNE is a Phase 1/2 study to investigate the safety, tolerability, and efficacy of a single IV infusion of Solid Bioscience’s gene therapy (SGT-003) in
pediatric male participants with DMD. This is an open-label study in which all participants receive the study drug. The study is enrolling children 0-12 years old, with follow-up through five years after receiving dosing.
Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients with Duchenne Muscular Dystrophy
Sponsor Catalyst Pharmaceuticals, Inc.
Site PI: Seth Perlman, MD
Contact: Marissa Robertson (Marissa.Robertson@seattlechildrens.org) and Christa Rocco (Christa.Rocco@seattlechildrens.org)
ClinicalTrials.gov ID NCT06564974
 This registry collects additional information on the safety of long-term treatment with AGAMREE® in male patients aged 2 years and older with Duchenne muscular dystrophy (DMD). Patients will be followed for approximately 5 years in the registry and will return to the site for Yearly Follow-up Visits (+/- 30 days) for registry assessments. Information on standard of care treatment and procedures for management of DMD will also be collected. Patients and/or their parents/legal guardians will be asked to complete paper QoL questionnaires at enrollment and at each Yearly Follow-up Visit.
Registry Study to Observe Long-term Safety and Efficacy of delandistrogene moxeparvovec (Elevidys) in Patients with Duchenne Muscular Dystrophy (ENDURE)
Sponsor: Sarepta Therapeutics
Site PI: Alicia Henriquez, MD
Contact: Marissa Robertson (Marissa.Robertson@seattlechildrens.org) and Macarena Racciatti (Macarena.Racciatti@seattlechildrens.org)
ClinicalTrials.gov ID: NCT06270719
This is a multicenter, prospective, observational Phase 4 study designed to collect both medical history and prospective data on DMD treatment outcomes in participants receiving delandistrogene moxeparvovec as part of clinical care, compared to participants with DMD receiving or prescribed to start chronic glucocorticoid treatment at baseline in routine clinical practice. Planned visits will occur at Baseline/Enrollment and every 6 months (± 60 days) for 10 years. Participants who are enrolled prior to administration of delandistrogene moxeparvovec will be followed for up to 10 years from the date of infusion.

Facioscapulohumeral Muscular Dystrophy

Clenbuterol to Target DUX4 in FSHD (Target FSHD): A Phase 1 Open label Safety and Tolerability Study of 3 Doses of Clenbuterol
Sponsor:  University of Kansas Medical Center
Site PI: Leo Wang, MD, PhD
Contact: Lilly Young (eyoung8@uw.edu)
ClinicalTrials.gov Identifier: NCT06721299
The goal of the study is to determine the best dose for a future trial of the efficacy of Clenbuterol. The study is a 6-month open-label multiple ascending dose study of clenbuterol for safety and tolerability.
Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD)
Sponsor: University of Kansas Medical Center
Site PI: Leo Wang, MD
Contact: Lilly Young (eyoung8@uw.edu)
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.

Inflammatory Myopathies

No current work in progress.

Limb Girdle Muscular Dystrophy

A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/​R9 (Part1)
Sponsor: Asklepios Biopharmaceutical, Inc
Site PI: Jane Distad, MD
Contact: Piya Modalavalasa (piyam@uw.edu)
ClinicalTrials.gov Identifier: NCT05230459
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.

Myotonic Dystrophy

A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multiple-Ascending Dose Study of PGN-EDODM1 in Adult Participants with Myotonic Dystrophy Type 1 (FREEDOM2-DM1)
Sponsor: PepGen Inc.
Site PI: Leo Wang, MD, PhD
Contact: Kaycie Opiyo (tkaycie@uw.edu)
ClinicalTrials.gov Identifier: NCT06667453
The purpose of this study is to learn about the effects of an investigational medicine, PGN-EDODM1, to see how safe and tolerable multiple administrations of PGN-EDODM1 are for people with myotonic dystrophy type 1 (DM1) compared to placebo over a period of 16 weeks.

Myasthenia Gravis

A Randomized, Double-Blind, Placebo-Controlled Phase 3 Trial of Descartes-08 in Patients With Acetylcholine Receptor Antibody-Positive Generalized Myasthenia Gravis (AURORA)
Sponsor: Cartesian Therapeutics, Inc.
Site PI: Michael Weiss, MD
Contact: Kaycie Opiyo (tkaycie@uw.edu)
ClinicalTrials.gov Identifier: NCT06799247
The AURORA Study is evaluating the safety, tolerability, and efficacy of an investigational mRNA CAR T-cell therapy known as Descartes-08 in adults with acetylcholine receptor autoantibody -positive generalized myasthenia gravis. The study will have a 6 month double-blinded treatment period, followed by an 8 month open-label extension period.
A Randomized, Double-blind, Placebo-controlled Phase III Study to Evaluate the Efficacy, Safety, and Tolerability of Remibrutinib in Patients With Generalized Myasthenia Gravis, Followed by an Open-label Extension Phase (RELIEVE)
Sponsor: Novartis Pharma AG
Site PI: B. Jane Distad, MD
Contact: Kaycie Opiyo (tkaycie@uw.edu)
ClinicalTrials.gov Identifier: NCT06744920
The goal of the study is to investigate the efficacy, safety, and tolerability of remibrutinib versus placebo in patients with generalized Myasthenia Gravis. Eligible participants will be randomized in a ratio of 1:1 to receive either remibrutinib or a matching placebo for 6 months, followed by an open-label extension of up to 5 years. Learn more about the study here: https://www.iths.org/participate/relieve-a-clinical-trial-for-myasthenia-gravis/

Spinal Muscular Atrophy

Spinraza in Adult Spinal Muscular Atrophy (SMA)
Sponsor: Washington University
Site-PI: Nassim Rad, MD
Contact: Dani Dixon (daniedxn@uw.edu)
ClinicalTrials.gov Identifier: NCT03709784 
This is an observational study of adult SMA patients on Spinraza.
Registry of Patients with a Diagnosis of Spinal Muscular Atrophy
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
This is a registry of SMA patients to assess long-term outcomes with and without medications.

Principal Investigators

Research Coordinators