Ignite Creation Date:
2024-05-06 @ 12:18 PM
Last Modification Date:
2024-10-26 @ 12:57 PM
Study NCT ID:
NCT03728803
Status:
COMPLETED
Last Update Posted:
2021-07-07
First Post:
2018-10-15
Brief Title:
Inspiratory Muscle Training in Nemaline Myopathy
Sponsor:
Radboud University Medical Center
Organization:
Radboud University Medical Center
Study Overview
Official Title:
Inspiratory Muscle Training in Patients With Nemaline Myopathy
Status:
COMPLETED
Status Verified Date:
2021-03
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:
NEMTRAIN
Brief Summary:
Nemaline myopathy is a rare congenital myopathy Respiratory failure is the main cause of death in these patients The primary objective of this study is to determine the effect of a 8-week inspiratory muscle training program on respiratory muscle function in nemaline myopathy patients The secondary objective is to determine respiratory muscle function in nemaline myopathy patients and its correlation with clinical severity and general neuromuscular function The nemaline myopathy patients will be included in the first phase for a clinical characterization From this phase patients will be selected for the second phase which is a controlled before-after trial of inspiratory muscle training The primary outcome is the change in maximal inspiratory pressure MIP after active inspiratory muscle training
Detailed Description:
Rationale Nemaline myopathy is a group of congenital hereditary neuromuscular disorders with variable symptoms such as muscle weakness swallowing dysfunction and dysarthria Respiratory failure is the main cause of death in nemaline myopathy and occurs even in ambulant patients who otherwise appear to be only mildly affected respiratory muscle weakness may even be the presenting feature Inspiratory muscle training has shown to increase inspiratory muscle strength in patients with other neuromuscular disorders It is hypothesized that inspiratory muscle training improves respiratory muscle function in nemaline myopathy patients with respiratory muscle weakness
Objective The primary objective is to determine the effect of a 8-week inspiratory muscle training program on respiratory muscle function in nemaline myopathy patients The secondary objective is to determine respiratory muscle function in nemaline myopathy patients and its correlation with clinical severity and general neuromuscular function
Study design The study consist of two phases Phase 1 A screening phase with an open design from which patients will be selected for the second phase Phase 2 A controlled before-after trial of inspiratory muscle training The 2 conditions tested are sham IMT and active IMT
Study population Nemaline myopathy patients from the local neuromuscular database will be recruited Furthermore other centres in the Netherlands will be contacted to expand the database of nemaline myopathy patients Phase 2 requires 23 patients
Intervention Active IMT consists of 15 minutes of IMT twice a day 5 days per week for 8 weeks at a training workload of 30 of MIP using a resistive inspiratory muscle training device Sham IMT consists of similar training regime using a resistive inspiratory muscle training device where the resistance has been removed
Main study parametersendpoints The primary outcome parameter is the change in maximal inspiratory pressure MIP after active inspiratory muscle training
Nature and extent of the burden and risks associated with participation benefit and group relatedness The intervention of inspiratory muscle training is not associated with any risks but can be challenging in patients with respiratory muscle weakness to perform There will be three visits to the hospital in 16 weeks The first visit has a maximal duration of 6 hours including breaks and the other two visits 15 hour During these visits several tests and physical examinations will be performed Some of the tests may cause some physical discomfort but none of them carry any risk Patients may benefit from participating in this study by developing improved respiratory muscle function as a result of the inspiratory muscle training
Objective The primary objective is to determine the effect of a 8-week inspiratory muscle training program on respiratory muscle function in nemaline myopathy patients The secondary objective is to determine respiratory muscle function in nemaline myopathy patients and its correlation with clinical severity and general neuromuscular function
Study design The study consist of two phases Phase 1 A screening phase with an open design from which patients will be selected for the second phase Phase 2 A controlled before-after trial of inspiratory muscle training The 2 conditions tested are sham IMT and active IMT
Study population Nemaline myopathy patients from the local neuromuscular database will be recruited Furthermore other centres in the Netherlands will be contacted to expand the database of nemaline myopathy patients Phase 2 requires 23 patients
Intervention Active IMT consists of 15 minutes of IMT twice a day 5 days per week for 8 weeks at a training workload of 30 of MIP using a resistive inspiratory muscle training device Sham IMT consists of similar training regime using a resistive inspiratory muscle training device where the resistance has been removed
Main study parametersendpoints The primary outcome parameter is the change in maximal inspiratory pressure MIP after active inspiratory muscle training
Nature and extent of the burden and risks associated with participation benefit and group relatedness The intervention of inspiratory muscle training is not associated with any risks but can be challenging in patients with respiratory muscle weakness to perform There will be three visits to the hospital in 16 weeks The first visit has a maximal duration of 6 hours including breaks and the other two visits 15 hour During these visits several tests and physical examinations will be performed Some of the tests may cause some physical discomfort but none of them carry any risk Patients may benefit from participating in this study by developing improved respiratory muscle function as a result of the inspiratory muscle training
Study Oversight
Has Oversight DMC:
None
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?:
None