Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD

Launched by UNIVERSITY OF KANSAS MEDICAL CENTER · Mar 7, 2018

Keywords

ClinConnect Summary

This clinical trial is focused on improving drug development for a condition called facioscapulohumeral muscular dystrophy (FSHD), which affects muscle strength and movement. Researchers have recently identified the main cause of FSHD and are now working to create better ways to measure the effectiveness of potential treatments. The trial will test two new measurement tools and refine the criteria for who can participate, involving 320 patients from 14 sites around the world over the next two years.

To be eligible for the trial, participants must have a confirmed genetic diagnosis of FSHD or show typical signs of the condition and have a family history of it. They should also have noticeable weakness in their limbs and be able to walk 30 feet without help (though they can use canes or braces). However, people with certain heart or lung problems, orthopedic issues that affect muscle testing, or who are pregnant cannot participate. Throughout the study, participants will be asked to keep their supplement routines consistent if they are taking any over-the-counter products. This trial aims to establish clearer pathways for developing new treatments, which could ultimately help improve the lives of those living with FSHD.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Patients with genetically confirmed FSHD1 or clinical diagnosis of FSHD with characteristic findings on exam and an affected parent or offspring
• • Patients with symptomatic limb weakness
• • Patients must be able to walk 30 feet without the support of another person or assistance (canes, walking sticks, and braces allowed; no walker).
• • If taking over the counter supplements, willing to remain consistent with supplement regimen throughout the course of the study
• Exclusion Criteria:
• • Patients with cardiac or respiratory dysfunction (deemed clinically unstable, or would interfere with safe testing, in the opinion of the Investigator)
• • Patients with orthopedic conditions that preclude safe testing of muscle function
• • Patients that regularly use available muscle anabolic/catabolic agents such as corticosteroids, oral testosterone or derivatives, or oral beta agonists
• • Patients that have used an experimental drug in an FSHD clinical trial within the past 30 days
• • Patients that are pregnant