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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
OTHER
Masking
NONE
Enrollment
93
Administered as specified in the treatment arm.
Research Site
Essen, North Rhine-Westphalia, Germany
Research Site
Dresden, Germany
Research Site
Hanover, Germany
Research Site
Heidelberg, Germany
LMU Klinikum Friedrich-Baur-Institute, Dept. of Neurology
Munich, Germany
Type of Correlation of Konectom DOAs Versus Hammersmith Functional Motor Scale-Expanded (HFMSE) Total Score in PwSMA
This outcome measure will assess the convergent validity of smartphone-based Konectom DOAs against in-clinic standard assessments.
Time frame: Up to 28 days
Strength of Correlation of Konectom DOAs Versus HFMSE Total Score in PwSMA
This outcome measure will assess the convergent validity of smartphone-based Konectom DOAs against in-clinic standard assessments.
Time frame: Up to 28 days
Interclass Correlation Coefficient (ICC) of the Konectom Digital Outcome Assessment (DOA) Scores
This outcome measure will assess test-retest reliability of smartphone-based Konectom DOAs.
Time frame: Up to 28 days
Type of Correlation of Upper Limb Konectom DOAs Versus Revised Upper Limb Module (RULM) in PwSMA
This outcome measure will assess the relationship between Konectom Upper Limb DOAs and conventional upper limb assessments in clinical environments.
Time frame: Up to 28 days
Strength of Correlation of Upper Limb Konectom DOAs Versus RULM in PwSMA
This outcome measure will assess the relationship between Konectom Upper Limb DOAs and conventional upper limb assessments in clinical environments.
Time frame: Up to 28 days
Type of Correlation of Upper Limb Konectom DOAs Versus 9-Hole Peg test (9HPT) in PwSMA
This outcome measure will assess the relationship between Konectom Upper Limb DOAs and conventional upper limb assessments in clinical environments.
Time frame: Up to 28 days
Strength of Correlation of Upper Limb Konectom DOAs Versus 9HPT in PwSMA
This outcome measure will assess the relationship between Konectom Upper Limb DOAs and conventional upper limb assessments in clinical environments.
Time frame: Up to 28 days
Type of Correlation of Lower Limb Konectom DOAs Versus 6-Minute Walk Test (MWT) Total Distance in Ambulatory PwSMA
This outcome measure will assess the relationship between Konectom Lower Limb DOAs and status of ambulation in clinical environments.
Time frame: Up to 28 days
Strength of Correlation of Lower Limb Konectom DOAs Versus 6-MWT Total Distance in Ambulatory PwSMA
This outcome measure will assess the relationship between Konectom Lower Limb DOAs and status of ambulation in clinical environments.
Time frame: Up to 28 days
Differences Between PwSMA and HS in the Konectom DOA Scores During Each Testing Condition
This outcome measure will assess the group differences in smartphone-based Konectom DOAs \[self-administered at home and in-clinic\] between PwSMA and healthy subjects (HS).
Time frame: Up to 28 days
Standard Deviation of Each Participant's Raw Konectom DOA Scores Over the At-Home Period
This outcome measure will assess the variability of Konectom DOAs self-administered in everyday environment in HS and PwSMA.
Time frame: 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
This outcome measure will assess the comparison of Konectom DOAs between in-clinic supervised administration versus self-assessments in everyday environment in HS and PwSMA groups.
Time frame: Up to 28 days
Type of Correlation of Konectom DOA Scores Versus Neuro-Quality of Life (QoL) Total Scores in PwSMA
This outcome measure will assess the relationship of Konectom DOAs against patient-reported outcomes (PROs) in PwSMA.
Time frame: Up to 28 days
Strength of Correlation of Konectom DOA Scores Versus Neuro-QoL Total Scores in PwSMA
This outcome measure will assess the relationship of Konectom DOAs against PROs in PwSMA.
Time frame: Up to 28 days
Type of Correlation of Konectom DOA Scores Versus Fatigue Severity Scale (FSS) Total Scores in PwSMA
This outcome measure will assess the relationship of Konectom DOAs against PROs in PwSMA.
Time frame: Up to 28 days
Strength of Correlation of Konectom DOA Scores Versus FSS Total Scores in PwSMA
This outcome measure will assess the relationship of Konectom DOAs against PROs in PwSMA.
Time frame: Up to 28 days
Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) Related to Konectom NMD Use
An AE is any untoward medical occurrence in a participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal assessment such as an abnormal laboratory value), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. An SAE is any untoward medical occurrence that at any dose results in death, in the view of the Investigator, places the participant at immediate risk of death, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results in a birth defect. This outcome measure will assess the clinical safety of Konectom NMD in PwSMA.
Time frame: Up to 43 days
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