Back to resultsA Study to Assess the Clinical Validity of Konectom™ in Adults Living With Neuromuscular Disorders (DigiNOA)
Primary Purpose
Spinal Muscular Atrophy
Status
Recruiting
Phase
Not Applicable
Locations
Germany
Study Type
Interventional
Intervention
Konectom NMD Application
Sponsored by
About this trial
This is an interventional other trial for Spinal Muscular Atrophy focused on measuring Neuromuscular Diseases, Spinal Muscular Atrophy, Digital technology, Digital outcome assessments, Smartphone, Digital Biomarkers
Eligibility Criteria
Key Inclusion Criteria:
For PwSMA
- Genetic documentation of 5q SMA (homozygous gene deletion, mutation, or compound heterozygote).
For PwALS
- ALS diagnosis by a board-certified neurologist. The minimal level of diagnostic certainty should be "possible ALS" according to El Escorial criteria.
For Healthy Participants
- Age group matched with SMA and ALS participants
Key Exclusion Criteria:
For PwSMA and PwALS
- 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.
Sites / Locations
- Research SiteRecruiting
- Research SiteRecruiting
- Research SiteRecruiting
- Research SiteRecruiting
- LMU Klinikum Friedrich-Baur-Institute, Dept. of NeurologyRecruiting
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm Type
Experimental
Experimental
Experimental
Arm Label
Participants with SMA (PwSMA)
Participants with ALS (PwALS)
Healthy Participants
Arm Description
Participants with SMA will have their motor functions assessed using the Konectom NMD smartphone-based application up to Day 28.
Participants with ALS will have their cognitive and motor functions assessed using the Konectom NMD smartphone-based application up to Day 28. As per protocol version 4.0, enrolment of ALS participants is stopped, and the data of the already enrolled ALS participants would not be analyzed.
Healthy participants will have their cognitive and motor functions assessed using the Konectom NMD smartphone-based application for 28 days.
Outcomes
Primary Outcome Measures
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.
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.
Secondary Outcome Measures
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.
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.
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.
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.
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.
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.
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.
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).
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.
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.
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.
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.
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.
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.
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.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05109637
Brief Title
A Study to Assess the Clinical Validity of Konectom™ in Adults Living With Neuromuscular Disorders
Acronym
DigiNOA
Official Title
Study to Assess the Clinical Validity of Konectom™ in Adults Living With Neuromuscular Disorders
Study Type
Interventional
2. Study Status
Record Verification Date
July 2023
Overall Recruitment Status
Recruiting
Study Start Date
February 10, 2022 (Actual)
Primary Completion Date
December 31, 2023 (Anticipated)
Study Completion Date
December 31, 2023 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Biogen
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Spinal Muscular Atrophy
Keywords
Neuromuscular Diseases, Spinal Muscular Atrophy, Digital technology, Digital outcome assessments, Smartphone, Digital Biomarkers
7. Study Design
Primary Purpose
Other
Study Phase
Not Applicable
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
93 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Participants with SMA (PwSMA)
Arm Type
Experimental
Arm Description
Participants with SMA will have their motor functions assessed using the Konectom NMD smartphone-based application up to Day 28.
Arm Title
Participants with ALS (PwALS)
Arm Type
Experimental
Arm Description
Participants with ALS will have their cognitive and motor functions assessed using the Konectom NMD smartphone-based application up to Day 28.
As per protocol version 4.0, enrolment of ALS participants is stopped, and the data of the already enrolled ALS participants would not be analyzed.
Arm Title
Healthy Participants
Arm Type
Experimental
Arm Description
Healthy participants will have their cognitive and motor functions assessed using the Konectom NMD smartphone-based application for 28 days.
Intervention Type
Device
Intervention Name(s)
Konectom NMD Application
Intervention Description
Administered as specified in the treatment arm.
Primary Outcome Measure Information:
Title
Type of Correlation of Konectom DOAs Versus Hammersmith Functional Motor Scale-Expanded (HFMSE) Total Score in PwSMA
Description
This outcome measure will assess the convergent validity of smartphone-based Konectom DOAs against in-clinic standard assessments.
Time Frame
Up to 28 days
Title
Strength of Correlation of Konectom DOAs Versus HFMSE Total Score in PwSMA
Description
This outcome measure will assess the convergent validity of smartphone-based Konectom DOAs against in-clinic standard assessments.
Time Frame
Up to 28 days
Secondary Outcome Measure Information:
Title
Interclass Correlation Coefficient (ICC) of the Konectom Digital Outcome Assessment (DOA) Scores
Description
This outcome measure will assess test-retest reliability of smartphone-based Konectom DOAs.
Time Frame
Up to 28 days
Title
Type of Correlation of Upper Limb Konectom DOAs Versus Revised Upper Limb Module (RULM) in PwSMA
Description
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
Title
Strength of Correlation of Upper Limb Konectom DOAs Versus RULM in PwSMA
Description
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
Title
Type of Correlation of Upper Limb Konectom DOAs Versus 9-Hole Peg test (9HPT) in PwSMA
Description
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
Title
Strength of Correlation of Upper Limb Konectom DOAs Versus 9HPT in PwSMA
Description
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
Title
Type of Correlation of Lower Limb Konectom DOAs Versus 6-Minute Walk Test (MWT) Total Distance in Ambulatory PwSMA
Description
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
Title
Strength of Correlation of Lower Limb Konectom DOAs Versus 6-MWT Total Distance in Ambulatory PwSMA
Description
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
Title
Differences Between PwSMA and HS in the Konectom DOA Scores During Each Testing Condition
Description
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
Title
Standard Deviation of Each Participant's Raw Konectom DOA Scores Over the At-Home Period
Description
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
Title
Paired-Comparisons of Konectom DOA Scores Between In-Clinic Supervised Administration and Self-Assessment In Everyday Environment, Separately for HS and PwSMA Groups
Description
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
Title
Type of Correlation of Konectom DOA Scores Versus Neuro-Quality of Life (QoL) Total Scores in PwSMA
Description
This outcome measure will assess the relationship of Konectom DOAs against patient-reported outcomes (PROs) in PwSMA.
Time Frame
Up to 28 days
Title
Strength of Correlation of Konectom DOA Scores Versus Neuro-QoL Total Scores in PwSMA
Description
This outcome measure will assess the relationship of Konectom DOAs against PROs in PwSMA.
Time Frame
Up to 28 days
Title
Type of Correlation of Konectom DOA Scores Versus Fatigue Severity Scale (FSS) Total Scores in PwSMA
Description
This outcome measure will assess the relationship of Konectom DOAs against PROs in PwSMA.
Time Frame
Up to 28 days
Title
Strength of Correlation of Konectom DOA Scores Versus FSS Total Scores in PwSMA
Description
This outcome measure will assess the relationship of Konectom DOAs against PROs in PwSMA.
Time Frame
Up to 28 days
Title
Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) Related to Konectom NMD Use
Description
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
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
64 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
US Biogen Clinical Trial Center
Phone
866-633-4636
Email
clinicaltrials@biogen.com
First Name & Middle Initial & Last Name or Official Title & Degree
Global Biogen Clinical Trial Center
Email
clinicaltrials@biogen.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Director
Organizational Affiliation
Biogen
Official's Role
Study Director
Facility Information:
Facility Name
Research Site
City
Essen
State/Province
North Rhine-Westphalia
ZIP/Postal Code
45147
Country
Germany
Individual Site Status
Recruiting
Facility Contact:
Phone
+49 201 7236513
Email
tim.hagenacker@uk-essen.de
First Name & Middle Initial & Last Name & Degree
Prof. Dr. med. Tim Hagenacker
Facility Name
Research Site
City
Dresden
ZIP/Postal Code
01307
Country
Germany
Individual Site Status
Recruiting
Facility Name
Research Site
City
Hannover
ZIP/Postal Code
30625
Country
Germany
Individual Site Status
Recruiting
Facility Name
Research Site
City
Heidelberg
ZIP/Postal Code
69120
Country
Germany
Individual Site Status
Recruiting
Facility Name
LMU Klinikum Friedrich-Baur-Institute, Dept. of Neurology
City
Munich
ZIP/Postal Code
80336
Country
Germany
Individual Site Status
Recruiting
Facility Contact:
Phone
+49 89 4400 57470
First Name & Middle Initial & Last Name & Degree
Stephan Wenninger
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing Plan Description
In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/
IPD Sharing URL
https://vivli.org/
Learn more about this trial
A Study to Assess the Clinical Validity of Konectom™ in Adults Living With Neuromuscular Disorders
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