Individualized trajectories of upper arm disease progression in Duchenne muscular dystrophy patients

Recruiting

Conditions: Duchenne
Duchenne muscular dystrophy
10029317

Registration Number: NL-OMON52311

Lead Sponsor: eids Universitair Medisch Centrum

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Recruiting

Sex: Not specified

Target Recruitment: 35

Inclusion Criteria

- Confirmed diagnosis of DMD through genetic testing;
- Age five years and older.

Exclusion Criteria

- Lack of confirmed mutation in the dystrophin gene or patients with a
secondary (neuro)muscular or metabolic disease that affect muscle function;
- Intake of investigational medications or exposure to an investigational drug
within 6 months prior to the start of the study;
- Cognitive problems that would make it difficult to follow directions and
participate in testing;
- Contraindications to MRI exposure (such as a metal implant).

Study & Design

Study Type: Observational non invasive

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>The main study parameters at the four different timepoints (0, 6, 12 and 18<br /><br>months) are the mFF of the upper limb flexor muscles, measured by qMRI, the<br /><br>muscle force of the upper limb (elbow flexion strength) and upper arm function<br /><br>as measured with the PUL2.0. These datapoints will be used to construct the<br /><br>individual disease trajectories.</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Secondary endpoints are:<br /><br>- The clinical elbow flexion endpoint, defined as the loss of the ability to<br /><br>bring the hand to the mouth with a 200g weighing cup (supporting elbow on table<br /><br>is allowed).<br /><br>- Upper limb strength: shoulder abduction, elbow flexion and extension strength<br /><br>and wrist flexion- and extension strength measured using hand held dynamometry<br /><br>(HHD; N);<br /><br>- Absolute grip strength (kg), pinch strength (kg), measured using MyoGrip and<br /><br>MyoPinch;<br /><br>- The DMD Upper Limb patient*reported outcome measure (PROM; score 0-64);<br /><br>- The PedsQL3.0 Neuromuscular questionnaire (score 0-100);</p><br>