Ignite Creation Date:
2025-12-26 @ 11:11 AM
Ignite Modification Date:
2026-01-01 @ 1:19 AM
Study NCT ID:
NCT03930628
Status:
UNKNOWN
Last Update Posted:
2022-11-07
First Post:
2019-04-15
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Limb-Girdle Muscular Dystrophy Type 2I in Norway
Sponsor:
University Hospital of North Norway
Organization:
Study Overview
Official Title:
Limb-Girdle Muscular Dystrophy Type 2I in Norway - a Cohort Study
Status:
UNKNOWN
Status Verified Date:
2022-11
Last Known Status:
ACTIVE_NOT_RECRUITING
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:
Key goals are to establish the natural history of limb-girdle muscular dystrophy type 2I (LGMD 2I) and identify feasible and sensitive tools and biomarkers to measure disease affection and progression, determine the Norwegian LGMD 2I prevalence, carrier frequency and genotypes, and to assess health-related quality of life in the Norwegian LGMD 2I population.
Main aims are to facilitate future clinical trials and contribute to good clinical practice with suitable methodology and to complete health and social care in order to optimize the function and quality of daily living of the patient group.
Main aims are to facilitate future clinical trials and contribute to good clinical practice with suitable methodology and to complete health and social care in order to optimize the function and quality of daily living of the patient group.
Detailed Description:
A single-center study with Norwegian nationwide enrollment. Data is based on questionnaires, patient journals, clinical examination, a set of functional tests and biomarkers, and patient reported outcomes. Clinical/ paraclinical tests are repeated after 2-years in order to measure disease progression. Both skeletal muscle, heart and respiratory function will be examined. At baseline there will also be performed a sleep study in order to find if they are prone to sleep-disordered breathing.
Study Oversight
Has Oversight DMC:
False
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: