Neuromuscular Clinical Trials

List of Current Trials

ARTHREX

The goal of this clinical trial is to test ATX-01 in participants with myotonic dystrophy type 1 (DM1). The main question it aims to answer is if ATX-01 is…

Investigator
Sankarasubramoney Subramony
Protocol ID
NCT06300307
Ages
18 Years – 64 Years
Sexes
All
Location
Gainesville

Avidity FSHD

A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory…

Investigator
Sankarasubramoney Subramony
Protocol ID
NCT05747924
Ages
16 Years – 70 Years
Sexes
All
Location
Gainesville

COYA

The ALSTARS trial will be conducted across 20-25 sites in the US and Canada, and will evaluate the safety and efficacy of an investigational treatment called…

Investigator
James Wymer
Protocol ID
NCT07161999
Ages
18 Years – 80 Years
Sexes
All
Location
Gainesville

Fortitude OLE (AOC 1020-CS2)

A Phase 2 Open-label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of AOC 1020 Administered Intravenously to Participants with…

Investigator
Sankarasubramoney Subramony
Protocol ID
NCT06547216
Ages
16 Years – 72 Years
Sexes
All
Location
Gainesville

HARBOR

A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous Delpacibart Etedesiran (abbreviated…

Investigator
Sankarasubramoney Subramony
Protocol ID
NCT06411288
Ages
16 Years – 65 Years
Sexes
All
Location
Gainesville

HEALEY ALS Platform Trial

The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the…

Investigator
James Wymer
Protocol ID
NCT04297683
Ages
18 Years – N/A
Sexes
All
Location
Gainesville

LUMINA

This study is a placebo-controlled Phase I study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of the antisense oligonucleotide…

Investigator
James Wymer
Protocol ID
NCT06665165
Ages
18 Years – N/A
Sexes
All
Location
Gainesville

PASS

In this study, researchers will learn more about the safety of BIIB141, also known as omaveloxolone or SKYCLARYS®. This is a drug available for doctors to…

Investigator
Sankarasubramoney Subramony
Protocol ID
NCT06623890
Ages
16 Years – N/A
Sexes
All
Location
Gainesville