Ignite Creation Date:
2024-05-06 @ 8:38 AM
Last Modification Date:
2024-10-26 @ 12:02 PM
Study NCT ID:
NCT02780492
Status:
COMPLETED
Last Update Posted:
2024-01-05
First Post:
2016-05-19
Brief Title:
Outcome Measures in Duchenne Muscular Dystrophy A Natural History Study
Sponsor:
University College London
Organization:
University College London
Study Overview
Official Title:
Developing Tools for Assessing the Natural History of Ambulant and Non-ambulant DMD Individuals to Assist in Antisense-oligomer Clinical Trials
Status:
COMPLETED
Status Verified Date:
2024-01
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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 MIPMEP A subgroup of patients performs annual whole body DEXA scan An imaging sub-study will aim to characterize muscle upperlower 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
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 MIPMEP A subgroup of patients performs annual whole body DEXA scan An imaging sub-study will aim to characterize muscle upperlower 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
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None