NCT04038138: ReSOLVE_France

Clinical Trial Readiness Network FSHD France: Prospective 24 Months MRI Study

Active, enrolled NA Last updated 3 December 2025

What this trial tests

NA trial testing Validation of new COA for FSHD patients in Muscular Dystrophy in 100 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline

16 September 2019

Primary endpoint
3 September 2025

3 September 2026

Quick facts

Lead sponsor	Centre Hospitalier Universitaire de Nice
Phase	NA
Status	Active, enrolled
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	other
Enrollment	100
Start date	16 September 2019
Primary completion	3 September 2025
Estimated completion	3 September 2026
Sites	3 locations across France

Drugs / interventions tested

• Validation of new COA for FSHD patients

Conditions studied

• Muscular Dystrophy — all drugs for Muscular Dystrophy →
• Facioscapulohumeral — all drugs for Facioscapulohumeral →

Sponsor

Centre Hospitalier Universitaire de Nice

Who can join

Adults 18 to 75, any sex, with Muscular Dystrophy or Facioscapulohumeral. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

The overall aim of this study is to hasten drug development for facioscapulohumeral muscular dystrophy (FSHD). Recent breakthroughs in FSHD research have identified the primary disease mechanism as the aberrant expression of a normally silenced gene, DUX4, resulting in a toxic gain-of-function. This disease mechanism is particularly amenable to knock-down of DUX4 using epigenetic strategies or RNA therapies, as well as to other interventions targeting the downstream effects of DUX4 expression. There are many drug companies actively working towards disease-targeted therapies, and two clinical trials either under way now, or planned to start in early Fall 2016. However, meetings with industry, advocacy groups, and FSHD researchers have identified several gaps in the clinical trial arsenal, and clinical trial planning as a major goal for the community. Consequently, there is an urgent need to establish the tools necessary for the conduct of currently planned and expected therapeutic trials in FSHD.

To this end, the researchers propose to develop two novel clinical outcome assessments (COA), a composite functional outcome measure (FSH-COM) and skeletal muscle biomarker, electrical impedance myography (EIM). In addition there is broad consensus a better understanding of the relationship of genetic and demographic features to disease progression will be necessary for enumerating eligibility criteria.

The specific aims are to: 1. Determine the multi-site validity of the COAs, 2. Compare the responsiveness of new COAs to other FSHD outcomes and determine the minimal clinically meaningful changes, and 3. establish FSHD cohort characteristics useful for determining clinical trial eligibility criteria. To achieve these aims, the Nice University Hospital is conducting a monocentric, prospective, 18 month study on 30 subjects.

Publications & conference data

1 peer-reviewed publication reference this trial (live from Europe PMC):

1. Interleukin-6 as a Key Biomarker in Facioscapulohumeral Dystrophy: Evidence From Longitudinal Analyses.
   Pini J, Martinuzzi E, Dhifallah S, Slioui A, et al · · 2026 · cited 1× · PMID 41058127 · DOI 10.1002/acn3.70210