Clinical Trial Details
— Status: Recruiting
Administrative data
| NCT number |
NCT03488784 |
| Other study ID # |
IRB17-01086 |
| Secondary ID |
|
| Status |
Recruiting |
| Phase |
|
| First received |
|
| Last updated |
|
| Start date |
January 5, 2018 |
| Est. completion date |
June 2025 |
Study information
| Verified date |
February 2023 |
| Source |
Nationwide Children's Hospital |
| Contact |
Megan Iammarino |
| Phone |
614 722-6881 |
| Email |
NMDtrialinfo[@]nationwidechildrens.org |
| Is FDA regulated |
No |
| Health authority |
|
| Study type |
Observational
|
Clinical Trial Summary
This is an observational study, no drug (marketed or investigational) will be provided as
part of the study, and the study procedures will have no impact on the medical care delivered
to patients participating in this study. The overall study data collection period is planned
to last up to 5 years with assessments occurring at baseline, and every 6 months thereafter
for a total period of 3 years.
Medical records for enrolled patients will be abstracted at baseline and annually to obtain
clinical information, and data will be recorded for the study. Eligible patients will be
asked to provide informed consent and to complete semi-annual patient surveys and functional
assessments. The patient surveys will include selected PRO instrument(s) along with
additional questions to characterize the patient's perception of disease.
Description:
Neuromuscular disease can be characterized by progressive muscle degeneration, impaired
pulmonary status, and decreased cardiac function.(1-8) Additionally, these neuromuscular
disorders can be rare, and therefore difficult to establish the natural progression of each
disease.The natural history of each neuromuscular disorder provides valuable information
about the specific progression of the disease, which can guide in understanding which
outcomes to measure in order to show change for clinical trials. Experimental treatments for
many of these neuromuscular disorders are currently being assessed in clinical trials with
others in the pipeline for upcoming clinical trials in the near future. Thus, the need to
reliably and objectively detect small, meaningful changes in daily functional activities in
order to serve as a supportive measure of efficacy in clinical trials is of great importance.
Functional and strength measures have been utilized as primary, secondary or exploratory
outcomes in clinical trials studying the efficacy of drug therapies. Many of these outcome
measures have been shown to be reliable and have been validated in neuromuscular disease.
This longitudinal study aims to characterize the clinical progression and functional impact
on patients with neuromuscular disorders over time by evaluating functional and
patient-reported outcomes (PROs). The association between functional impairment and long-term
outcomes, such as loss of mobility, falls, and quality of life, will be examined.
