An Observational Study in Participants with Ryanodine Receptor 1-Related Myopathies (RYR1-RM) to Determine Optimal Endpoint Measurements. - ARMGO RYR1-RM Endpoints

ARMGO Pharma, Inc.0 个研究点目标入组 5 人开始时间: 待定最近更新: 2024年8月19日

适应症

试验速览

暂无简介。

•- Males and females aged 18 years and older at screening;
•- Confirmed genetic diagnosis of RYR1-RM with autosomal dominant mutation and
•supporting clinical phenotype with demonstrable proximal weakness on at least
•one of the baseline study assessments;
•- Evidence of at least one demonstrable muscle/motor function deficit assessed
•through Manual muscle Testing (MMT) and scored using the medical Research
•Council (MRC) Scale for muscle strength on physical examination;
•- Able to walk 10 meters, with or without assistance - e.g. with a cane
•(assessed using the 10 Meter Walk Test);
•- Willingness and ability to comply with scheduled visits and study procedures;
•- Willingness to be fitted with the Syde device at Screening Visit (for
•inclusion in the exploratory objective analysis only); and
•- Able to provide written informed consent and understand the study procedures
•in the informed consent form (ICF).

•- Severe pulmonary dysfunction at Screening (FVC < 40% predicted) or evidence
•of pulmonary exacerbation (note that pulmonary exacerbations refer to acute
•worsening respiratory symptoms resulting from a decline in lung function);
•- Significant cognitive impairment in the judgement of the investigator who
•will be unable to follow the protocol;
•- Patients with progressive neurological conditions (e.g. Parkinson's disease);
•- Non-ambulant patients; or
•- Pregnant woman.

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