A Study to Assess the Clinical Validity of Konectom™ in Adults Living With Neuromuscular Disorders (DigiNOA)

Study to Assess the Clinical Validity of Konectom™ in Adults Living With Neuromuscular Disorders

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 Overview

• Status: Completed
• Conditions: Spinal Muscular Atrophy
• Intervention / Treatment: Device: Konectom NMD Application

• Study Type: Interventional
• Enrollment (Actual): 93
• Phase: Not Applicable

Contacts and Locations

Study Locations

• Germany
  • Dresden, Germany, 01307
    • Research Site
  • Hannover, Germany, 30625
    • Research Site
  • Heidelberg, Germany, 69120
    • Research Site
  • Munich, Germany, 80336
    • LMU Klinikum Friedrich-Baur-Institute, Dept. of Neurology
  • North Rhine-Westphalia
    • Essen, North Rhine-Westphalia, Germany, 45147
      • Research Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 64 years (Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Participants with SMA (PwSMA); Participants with SMA will have their motor functions assessed using the Konectom NMD smartphone-based application up to Day 28.	Device: Konectom NMD Application; Administered as specified in the treatment arm.
Experimental: Healthy Participants; Healthy participants will have their cognitive and motor functions assessed using the Konectom NMD smartphone-based application for 28 days.	Device: Konectom NMD Application; Administered as specified in the treatment arm.
Experimental: Participants with ALS (PwALS); 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.	Device: Konectom NMD Application; Administered as specified in the treatment arm.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	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.	Up to 28 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	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.	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.	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.	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.	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.	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.	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).	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.	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.	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.	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.	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.	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.	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.	Up to 43 days

Collaborators and Investigators

• Sponsor: Biogen
• Investigators: Study Director: Medical Director, Biogen

Study record dates

Study Major Dates

• Study Start (Actual): February 10, 2022
• Primary Completion (Actual): July 10, 2023
• Study Completion (Actual): July 25, 2023

Study Registration Dates

• First Submitted: October 27, 2021
• First Submitted That Met QC Criteria: October 27, 2021
• First Posted (Actual): November 5, 2021

Study Record Updates

• Last Update Posted (Estimated): February 6, 2024
• Last Update Submitted That Met QC Criteria: February 5, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Keywords: Smartphone; Neuromuscular Diseases; Spinal Muscular Atrophy; Digital Biomarkers; Digital technology; Digital outcome assessments
• Additional Relevant MeSH Terms: Central Nervous System Diseases; Nervous System Diseases; Neurologic Manifestations; Neurodegenerative Diseases; Neuromuscular Manifestations; Pathological Conditions, Anatomical; Spinal Cord Diseases; Motor Neuron Disease; Muscular Atrophy; Atrophy; Muscular Atrophy, Spinal; Neuromuscular Diseases
• Other Study ID Numbers: DE-SMG-11894; CIV-21-06-036845 (Registry Identifier: EUDAMED)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No