Neuromuscular Clinical Trials
Amyotrophic Lateral Sclerosis:
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 ABBV-CLS-7262 and DNL343.
Sponsor: Healey Center/Massachusetts General Hospital
Site PI: Michael Weiss, MD
Contact: Gage Sibik (gagsibik@uw.edu) and Kinsey Chapman (kinseyc@uw.edu)
ClinicalTrials.gov Identifier NCT04297683
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)
ClinicalTrials.gov Identifier NCT05137665
Facioscapulohumeral Muscular Dystrophy:
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
Hereditary Transthyretin-Mediated Amyloid Polyneuropathy:
A Study to Assess the Long-Term Safety and Efficacy of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Patients With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
The purpose of this study is to evaluate the safety and tolerability of extended dosing with Eplontersen in participants with hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN).
Sponsor: Ionis Pharmaceuticals, Inc
Site PI: Jane Distad, MD
Contact: Mike Willis (mwillis5@uw.edu)
ClinicalTrials.gov Identifier NCT05071300
Inflammatory Myopathies:
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
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)
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
Painful Peripheral Neuropathies:
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): Max Ma, MD
Contact: Hannah Bauermeister (Bauerh2@uw.edu)
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: Hannah Bauermeister (Bauerh2@uw.edu)
ClinicalTrials.gov Identifier NCT05480228
Spinal Muscular Atrophy:
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: Mike Willis (mwillis5@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), Leslie Vogel (leslie.vogel@ seattlechildrens.org)
ClinicalTrials.gov Identifier NCT04174157
Sporadic Inclusion Body Myositis:
A Study to Evaluate the Efficacy and Safety of ABC008 for Inclusion Body Myositis
A phase II/III randomized, double-blind, placebo-controlled, multicenter study to determine the efficacy and safety of ABC008 in the treatment of subjects with inclusion body myositis.
Sponsor: Abcuro, Inc
Site-PI: Leo Wang, MD
Contact: Dani Dixon (daniedxn@uw.edu)
ClinicalTrials.gov Identifier NCT05721573
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).
Sponsor: NIAMS, NIH
Site-PI: Leo Wang, MD
Contact: Dani Dixon (daniedxn@uw.edu)
ClinicalTrials.gov Identifier NCT05046821