Spinal Muscular Atrophy
Conditions
Keywords
Neuromuscular Diseases, Spinal Muscular Atrophy, Digital technology, Digital outcome assessments, Smartphone, Digital Biomarkers
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.
Interventions
DEVICEKonectom NMD Application
Administered as specified in the treatment arm.
Sponsors
Biogen
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
OTHER
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 64 Years
Healthy volunteers
No
Inclusion 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/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Type of Correlation of Konectom DOAs Versus Hammersmith Functional Motor Scale-Expanded (HFMSE) Total Score in PwSMA | 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 | Up to 28 days | This outcome measure will assess the convergent validity of smartphone-based Konectom DOAs against in-clinic standard assessments. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Type of Correlation of Upper Limb Konectom DOAs Versus Revised Upper Limb Module (RULM) in PwSMA | Up to 28 days | This outcome measure will assess the relationship between Konectom Upper Limb DOAs and conventional upper limb assessments in clinical environments. |
| Type of Correlation of Upper Limb Konectom DOAs Versus 9-Hole Peg test (9HPT) in PwSMA | Up to 28 days | This outcome measure will assess the relationship between Konectom Upper Limb DOAs and conventional upper limb assessments in clinical environments. |
| Strength of Correlation of Upper Limb Konectom DOAs Versus 9HPT in PwSMA | Up to 28 days | This outcome measure will assess the relationship between Konectom Upper Limb DOAs and conventional upper limb assessments in clinical environments. |
| Type of Correlation of Lower Limb Konectom DOAs Versus 6-Minute Walk Test (MWT) Total Distance in Ambulatory PwSMA | Up to 28 days | This outcome measure will assess the relationship between Konectom Lower Limb DOAs and status of ambulation in clinical environments. |
| Strength of Correlation of Lower Limb Konectom DOAs Versus 6-MWT Total Distance in Ambulatory PwSMA | Up to 28 days | This outcome measure will assess the relationship between Konectom Lower Limb DOAs and status of ambulation in clinical environments. |
| Differences Between PwSMA and HS in the Konectom DOA Scores During Each Testing Condition | Up to 28 days | 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). |
| Strength of Correlation of Upper Limb Konectom DOAs Versus RULM in PwSMA | Up to 28 days | This outcome measure will assess the relationship between Konectom Upper Limb DOAs and conventional upper limb assessments in clinical environments. |
| 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 | 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. |
| Type of Correlation of Konectom DOA Scores Versus Neuro-Quality of Life (QoL) Total Scores in PwSMA | Up to 28 days | This outcome measure will assess the relationship of Konectom DOAs against patient-reported outcomes (PROs) in PwSMA. |
| Strength of Correlation of Konectom DOA Scores Versus Neuro-QoL Total Scores in PwSMA | Up to 28 days | This outcome measure will assess the relationship of Konectom DOAs against PROs in PwSMA. |
| Type of Correlation of Konectom DOA Scores Versus Fatigue Severity Scale (FSS) Total Scores in PwSMA | Up to 28 days | This outcome measure will assess the relationship of Konectom DOAs against PROs in PwSMA. |
| Strength of Correlation of Konectom DOA Scores Versus FSS Total Scores in PwSMA | Up to 28 days | This outcome measure will assess the relationship of Konectom DOAs against PROs in PwSMA. |
| Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) Related to Konectom NMD Use | Up to 43 days | 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. |
| Standard Deviation of Each Participant's Raw Konectom DOA Scores Over the At-Home Period | Up to 28 days | This outcome measure will assess the variability of Konectom DOAs self-administered in everyday environment in HS and PwSMA. |
| Interclass Correlation Coefficient (ICC) of the Konectom Digital Outcome Assessment (DOA) Scores | Up to 28 days | This outcome measure will assess test-retest reliability of smartphone-based Konectom DOAs. |
Countries
Germany
Outcome results
None listed