Study to Assess the Clinical Validity of Konectom™ in Adults Living With Neuromuscular Disorders
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- Biogen
- 入组人数
- 93
- 试验地点
- 7
- 主要终点
- Type of Correlation of Konectom DOAs Versus Hammersmith Functional Motor Scale-Expanded (HFMSE) Total Score in PwSMA
研究概览
简要总结
The primary objective of the study is to explore the convergent validity of smartphone-based Konectom DOAs against in-clinic standard assessments.
The secondary objectives of this study are to evaluate the test-retest reliability of smartphone-based Konectom Digital Outcome Assessments (DOAs); to determine the relationship between Konectom upper limb DOAs and conventional upper limb assessments in clinical environments; to determine the relationship between Konectom lower limb DOAs and status of ambulation in clinical environments; to evaluate group differences in smartphone-based Konectom DOAs [self-administered at home and in-clinic] between person with spinal muscular atrophy (PwSMA) and healthy subjects (HS); to evaluate the variability of Konectom DOAs self-administered in everyday environment in HS and PwSMA; to compare Konectom DOAs between in-clinic supervised administration versus self-assessments in everyday environment in HS, PwSMA groups; to evaluate the relationship of Konectom DOAs against patient-reported outcomes (PROs) in PwSMA and to evaluate the clinical safety of Konectom in PwSMA.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Other
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 64 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Genetic documentation of 5q SMA (homozygous gene deletion, mutation, or compound heterozygote).
- •For Healthy Participants
- •Age group matched with SMA participants
排除标准
- •Change of disease modifying treatment (DMT) in the last 1 month.
- •Recent history of bacterial meningitis, viral encephalitis, or hydrocephalus.
- •Addiction (alcohol or another drug abuse).
- •Presence of an implanted shunt for the drainage of cerebrospinal fluid (CSF) or of an implanted central nervous system (CNS) catheter.
- •Hospitalization for surgery (i.e., scoliosis surgery or other surgery), pulmonary event, or nutritional support in the previous 2 months or planned within the study duration.
- •Known pregnancy.
- •NOTE: Other protocol defined inclusion/ exclusion criteria may apply.
结局指标
主要结局
Type of Correlation of Konectom DOAs Versus Hammersmith Functional Motor Scale-Expanded (HFMSE) Total Score in PwSMA
时间窗: Up to 28 days
This outcome measure will assess the convergent validity of smartphone-based Konectom DOAs against in-clinic standard assessments.
Strength of Correlation of Konectom DOAs Versus HFMSE Total Score in PwSMA
时间窗: Up to 28 days
This outcome measure will assess the convergent validity of smartphone-based Konectom DOAs against in-clinic standard assessments.
次要结局
- Strength of Correlation of Upper Limb Konectom DOAs Versus RULM in PwSMA(Up to 28 days)
- Type of Correlation of Upper Limb Konectom DOAs Versus Revised Upper Limb Module (RULM) in PwSMA(Up to 28 days)
- Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) Related to Konectom NMD Use(Up to 43 days)
- Interclass Correlation Coefficient (ICC) of the Konectom Digital Outcome Assessment (DOA) Scores(Up to 28 days)
- Type of Correlation of Upper Limb Konectom DOAs Versus 9-Hole Peg test (9HPT) in PwSMA(Up to 28 days)
- Type of Correlation of Lower Limb Konectom DOAs Versus 6-Minute Walk Test (MWT) Total Distance in Ambulatory PwSMA(Up to 28 days)
- Standard Deviation of Each Participant's Raw Konectom DOA Scores Over the At-Home Period(Up to 28 days)
- Paired-Comparisons of Konectom DOA Scores Between In-Clinic Supervised Administration and Self-Assessment In Everyday Environment, Separately for HS and PwSMA Groups(Up to 28 days)
- Type of Correlation of Konectom DOA Scores Versus Fatigue Severity Scale (FSS) Total Scores in PwSMA(Up to 28 days)
- Strength of Correlation of Upper Limb Konectom DOAs Versus 9HPT in PwSMA(Up to 28 days)
- Strength of Correlation of Lower Limb Konectom DOAs Versus 6-MWT Total Distance in Ambulatory PwSMA(Up to 28 days)
- Type of Correlation of Konectom DOA Scores Versus Neuro-Quality of Life (QoL) Total Scores in PwSMA(Up to 28 days)
- Strength of Correlation of Konectom DOA Scores Versus Neuro-QoL Total Scores in PwSMA(Up to 28 days)
- Strength of Correlation of Konectom DOA Scores Versus FSS Total Scores in PwSMA(Up to 28 days)
- Differences Between PwSMA and HS in the Konectom DOA Scores During Each Testing Condition(Up to 28 days)