Clinical Research

Actively Enrolling Natural History Studies

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Actively Enrolling Natural History Studies

Defining Clinical Endpoints in Limb Girdle Muscular Dystrophy (LGMD) (GRASP-01-001)

Study Type

Virginia Commonwealth University - Natural History 

Disease

Sarcoglycanopathies

Age Range

4 - 65

Clinical Research Coordinator

Allison Johnston

Principal Investigator

Peter B. Kang

Brief Summary

Limb Girdle Muscular Dystrophy comprise a group of disorders made up of over 30 mutations which share a common phenotype of progressive weakness of the shoulder and hip girdle muscles. While the individual genetic mutations are rare, as a cohort, LGMDs are one of the four most common muscular dystrophies. The overall goal of project 1 is to define the key phenotypes as measured by standard clinical outcome assessments (COAs) for limb girdle muscular dystrophies (LGMD) to hasten therapeutic development.

Defining Clinical Endpoints in Becker Muscular Dystrophy (BMD) (GRASP-01-002)

Study Type

Virginia Commonwealth University - Natural History 

Disease

Becker Muscular Dystrophy (BMD)

Age Range

8+

Clinical Research Coordinator

Allison Johnston

Principal Investigator

Peter B. Kang

Brief Summary

This is a 24-month, observational study of 150 participants with Becker muscular dystrophy (BMD)

Actively Enrolling Interventional Studies

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Actively Enrolling Interventional Studies

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

Study Type

Harmony Biosciences - Business & Industry

Disease

Myotonic (DM)

Age Range

18 - 65

Clinical Research Coordinator

John Martone

Principal Investigator

Peter Karachunski

Brief Summary

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.

The secondary objectives of this study are to assess the impact of pitolisant on fatigue, cognitive function and the burden of disease along with assessing the long-term safety and effectiveness of pitolisant in patients with Myotonic Dystrophy Type 1 ages 18 to 65 years.

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

Study Type

Avidity Biosciences - Business & Industry 

Disease

Facioscapulohumeral Muscular Dystrophy (FSHD)

Age Range

18 - 65

Clinical Research Coordinator

Erin Aguero

Principal Investigator

George Manousakis

Brief Summary

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)

Study to Evaluate the Efficacy and Safety of BBP-418 (Ribitol) in Patients With Limb Girdle Muscular Dystrophy 2I (LGMD2I) (Fortify)

Study Type

ML Bio Solutions - Industry & Business

Disease

LGMD2I/R9

Age Range

12 - 60

Clinical Research Coordinator

Allison Johnston

Principal Investigator

Peter B. Kang

Brief Summary

This study will evaluate the safety and efficacy of long-term administration of BBP-418 in patients with LGMD2I/R9. The study will include patients ages 12 to 60, consistent with the existing preclinical toxicology profile. This will encompass the significant majority of existing diagnosed patients based upon the established edpidemiology of the disease.