Clinical trial NCT05109637: A Study to Assess the Clinical Validity of Konectom™ in Adults Living With Neuromuscular Disorders
Official title: Study to Assess the Clinical Validity of Konectom™ in Adults Living With Neuromuscular Disorders
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Spinal Muscular Atrophy
Keywords: Neuromuscular Diseases
Spinal Muscular Atrophy
Digital technology
Digital outcome assessments
Smartphone
Digital Biomarkers
Lead sponsor: Biogen
Sponsor class: INDUSTRY
First posted: 2021-11-05
Enrollment: 93
Sex: ALL
Minimum age (years): 18
Maximum age (years): 64
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: OTHER

Brief summary: 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.


Eligibility criteria: Key Inclusion Criteria:

For PwSMA

* Genetic documentation of 5q SMA (homozygous gene deletion, mutation, or compound heterozygote).

For Healthy Participants

* Age group matched with SMA participants

Key Exclusion Criteria:

For PwSMA

* 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.

Primary outcomes:
measure: Type of Correlation of Konectom DOAs Versus Hammersmith Functional Motor Scale-Expanded (HFMSE) Total Score in PwSMA
timeFrame: Up to 28 days
measure: Strength of Correlation of Konectom DOAs Versus HFMSE Total Score in PwSMA
timeFrame: Up to 28 days
Secondary outcomes:
measure: Interclass Correlation Coefficient (ICC) of the Konectom Digital Outcome Assessment (DOA) Scores
timeFrame: Up to 28 days
measure: Type of Correlation of Upper Limb Konectom DOAs Versus Revised Upper Limb Module (RULM) in PwSMA
timeFrame: Up to 28 days
measure: Strength of Correlation of Upper Limb Konectom DOAs Versus RULM in PwSMA
timeFrame: Up to 28 days
measure: Type of Correlation of Upper Limb Konectom DOAs Versus 9-Hole Peg test (9HPT) in PwSMA
timeFrame: Up to 28 days
measure: Strength of Correlation of Upper Limb Konectom DOAs Versus 9HPT in PwSMA
timeFrame: Up to 28 days
measure: Type of Correlation of Lower Limb Konectom DOAs Versus 6-Minute Walk Test (MWT) Total Distance in Ambulatory PwSMA
timeFrame: Up to 28 days
measure: Strength of Correlation of Lower Limb Konectom DOAs Versus 6-MWT Total Distance in Ambulatory PwSMA
timeFrame: Up to 28 days
measure: Differences Between PwSMA and HS in the Konectom DOA Scores During Each Testing Condition
timeFrame: Up to 28 days
measure: Standard Deviation of Each Participant's Raw Konectom DOA Scores Over the At-Home Period
timeFrame: Up to 28 days
measure: Paired-Comparisons of Konectom DOA Scores Between In-Clinic Supervised Administration and Self-Assessment In Everyday Environment, Separately for HS and PwSMA Groups
timeFrame: Up to 28 days
measure: Type of Correlation of Konectom DOA Scores Versus Neuro-Quality of Life (QoL) Total Scores in PwSMA
timeFrame: Up to 28 days
measure: Strength of Correlation of Konectom DOA Scores Versus Neuro-QoL Total Scores in PwSMA
timeFrame: Up to 28 days
measure: Type of Correlation of Konectom DOA Scores Versus Fatigue Severity Scale (FSS) Total Scores in PwSMA
timeFrame: Up to 28 days
measure: Strength of Correlation of Konectom DOA Scores Versus FSS Total Scores in PwSMA
timeFrame: Up to 28 days
measure: Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) Related to Konectom NMD Use
timeFrame: Up to 43 days
Other outcomes:
Locations:
city: Essen
country: Germany
facility: Research Site
state: North Rhine-Westphalia
city: Dresden
country: Germany
facility: Research Site
city: Hannover
country: Germany
facility: Research Site
city: Heidelberg
country: Germany
facility: Research Site
city: Munich
country: Germany
facility: LMU Klinikum Friedrich-Baur-Institute, Dept. of Neurology
Study officials:
affiliation: Biogen
name: Medical Director
role: STUDY_DIRECTOR
References: