NCT02780492

Outcome Measures in Duchenne Muscular Dystrophy: A Natural History Study

Developing Tools for Assessing the Natural History of Ambulant and Non-ambulant DMD Individuals to Assist in Antisense-oligomer Clinical Trials

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 35 (actual)
• Sponsor: University College, London · Academic / Other
• Sex: Male
• Age: 5 Years – 18 Years
• Healthy volunteers: Accepted

Summary

Novel emerging therapies for Duchenne Muscular Dystrophy (DMD) require a deeper understanding of DMD natural history. This study aim to assess the natural history of DMD through a composite assessment tool capable of capturing disease progression linking ambulant and non-ambulant phases of the disease.

Detailed description

Novel emerging therapies for Duchenne Muscular Dystrophy (DMD) require a deeper understanding of DMD natural history. This study aim to assess the natural history of DMD through a composite assessment tool capable of capturing disease progression linking ambulant and non-ambulant phases of the disease. With a recruitment target of 80 DMD patients across 5 centres (London, Newcastle, Paris, Leiden, Nijmegen), subjects are assessed 6 monthly according to a shared protocol. Assessments include 6-minute walk distance (6MWD), North Star Ambulatory Assessment (NSAA), Performance of Upper Limb (PUL) and MyoSet (myogrip, myopinch and moviplate). Both ambulant and non-ambulant subjects undergo upper limb evaluation and respiratory function test including forced vital capacity (FVC), maximum inspiratory and expiratory pressures (MIP/MEP). A subgroup of patients performs annual whole body DEXA scan. An imaging sub-study will aim to characterize muscle (upper/lower limb) and brain MRI. The investigators will analyze the longitudinal data for the different assessment tools and explore correlations among them. This study will offer a comprehensive natural history of DMD including novel outcome measures, allowing to capture disease progression and explore the relationship between different assessment tools.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
OTHER	Set of assessment tools

Timeline

• Start date: 2012-04-11
• Primary completion: 2022-04-28
• Completion: 2022-04-28
• First posted: 2016-05-23
• Last updated: 2024-01-05

Locations

5 sites across 3 countries: France, Netherlands, United Kingdom