Natural History Study of Patients With Limb-Girdle Muscular Dystrophy 2I (NCT03842878) | Clinical Trial Compass
CompletedNot Applicable
Natural History Study of Patients With Limb-Girdle Muscular Dystrophy 2I
Denmark, France, United Kingdom52 participantsStarted 2020-02-19
Plain-language summary
Prospective, longitudinal, interventional, single-group, multicenter natural history study to better know the LGMD2I disease physiopathology. The duration of participation for each patient will be up to 24 months.
Who can participate
Age range
16 Years – 99 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Female and male patients
. Patients ≥ 16 years old
. Clinical diagnosis of LGMD2I and gene testing demonstrating two pathogenic mutations in fukutin-related protein gene, FKRP)
. Ambulant patients
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
6-Minute Walk Test
Timeframe: Baseline through 24 months
2
10-Meter Walk test (10MWT)
Timeframe: Baseline through 24 months
3
Timed Up and Go (TUG) test
Timeframe: Baseline through 24 months
4
Four-stair climb test
Timeframe: Baseline through 24 months
5
North Star Assessment for Neuromuscular Disorders (NSAD)
Timeframe: Baseline through 24 months
6
Upper limb assessment via the Performance of the Upper Limb (PUL) tool version 2.0