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

• ClinicalTrials.gov Identifier: NCT05109637
• Recruitment Status: Completed
• First Posted: November 5, 2021
• Last Update Posted: February 6, 2024
• Sponsor: Biogen
• Information provided by (Responsible Party): Biogen

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.

Condition or disease	Intervention/treatment	Phase
Spinal Muscular Atrophy	Device: Konectom NMD Application	Not Applicable

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 93 participants
• Allocation: Non-Randomized
• Intervention Model: Parallel Assignment
• Masking: None (Open Label)
• Primary Purpose: Other
• Official Title: Study to Assess the Clinical Validity of Konectom™ in Adults Living With Neuromuscular Disorders
• Actual Study Start Date: February 10, 2022
• Actual Primary Completion Date: July 10, 2023
• Actual Study Completion Date: July 25, 2023

Arms and Interventions

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: 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.
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.

Outcome Measures

Primary Outcome Measures :

1. Type of Correlation of Konectom DOAs Versus Hammersmith Functional Motor Scale-Expanded (HFMSE) Total Score in PwSMA [ Time Frame: Up to 28 days ]
   This outcome measure will assess the convergent validity of smartphone-based Konectom DOAs against in-clinic standard assessments.
2. Strength of Correlation of Konectom DOAs Versus HFMSE Total Score in PwSMA [ Time Frame: Up to 28 days ]
   This outcome measure will assess the convergent validity of smartphone-based Konectom DOAs against in-clinic standard assessments.

Secondary Outcome Measures :

1. Interclass Correlation Coefficient (ICC) of the Konectom Digital Outcome Assessment (DOA) Scores [ Time Frame: Up to 28 days ]
   This outcome measure will assess test-retest reliability of smartphone-based Konectom DOAs.
2. Type of Correlation of Upper Limb Konectom DOAs Versus Revised Upper Limb Module (RULM) in PwSMA [ Time Frame: Up to 28 days ]
   This outcome measure will assess the relationship between Konectom Upper Limb DOAs and conventional upper limb assessments in clinical environments.
3. Strength of Correlation of Upper Limb Konectom DOAs Versus RULM in PwSMA [ Time Frame: Up to 28 days ]
   This outcome measure will assess the relationship between Konectom Upper Limb DOAs and conventional upper limb assessments in clinical environments.
4. Type of Correlation of Upper Limb Konectom DOAs Versus 9-Hole Peg test (9HPT) in PwSMA [ Time Frame: Up to 28 days ]
   This outcome measure will assess the relationship between Konectom Upper Limb DOAs and conventional upper limb assessments in clinical environments.
5. Strength of Correlation of Upper Limb Konectom DOAs Versus 9HPT in PwSMA [ Time Frame: Up to 28 days ]
   This outcome measure will assess the relationship between Konectom Upper Limb DOAs and conventional upper limb assessments in clinical environments.
6. Type of Correlation of Lower Limb Konectom DOAs Versus 6-Minute Walk Test (MWT) Total Distance in Ambulatory PwSMA [ Time Frame: Up to 28 days ]
   This outcome measure will assess the relationship between Konectom Lower Limb DOAs and status of ambulation in clinical environments.
7. Strength of Correlation of Lower Limb Konectom DOAs Versus 6-MWT Total Distance in Ambulatory PwSMA [ Time Frame: Up to 28 days ]
   This outcome measure will assess the relationship between Konectom Lower Limb DOAs and status of ambulation in clinical environments.
8. Differences Between PwSMA and HS in the Konectom DOA Scores During Each Testing Condition [ Time Frame: 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).
9. Standard Deviation of Each Participant's Raw Konectom DOA Scores Over the At-Home Period [ Time Frame: Up to 28 days ]
   This outcome measure will assess the variability of Konectom DOAs self-administered in everyday environment in HS and PwSMA.
10. Paired-Comparisons of Konectom DOA Scores Between In-Clinic Supervised Administration and Self-Assessment In Everyday Environment, Separately for HS and PwSMA Groups [ Time Frame: 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.
11. Type of Correlation of Konectom DOA Scores Versus Neuro-Quality of Life (QoL) Total Scores in PwSMA [ Time Frame: Up to 28 days ]
    This outcome measure will assess the relationship of Konectom DOAs against patient-reported outcomes (PROs) in PwSMA.
12. Strength of Correlation of Konectom DOA Scores Versus Neuro-QoL Total Scores in PwSMA [ Time Frame: Up to 28 days ]
    This outcome measure will assess the relationship of Konectom DOAs against PROs in PwSMA.
13. Type of Correlation of Konectom DOA Scores Versus Fatigue Severity Scale (FSS) Total Scores in PwSMA [ Time Frame: Up to 28 days ]
    This outcome measure will assess the relationship of Konectom DOAs against PROs in PwSMA.
14. Strength of Correlation of Konectom DOA Scores Versus FSS Total Scores in PwSMA [ Time Frame: Up to 28 days ]
    This outcome measure will assess the relationship of Konectom DOAs against PROs in PwSMA.
15. Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) Related to Konectom NMD Use [ Time Frame: 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.

Eligibility Criteria

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

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.

Contacts and Locations

Locations

Germany

Research Site

Essen, North Rhine-Westphalia, Germany, 45147

Research Site

Dresden, Germany, 01307

Research Site

Hannover, Germany, 30625

Research Site

Heidelberg, Germany, 69120

LMU Klinikum Friedrich-Baur-Institute, Dept. of Neurology

Munich, Germany, 80336

Sponsors and Collaborators

Biogen

Investigators

• Study Director: Medical Director; Biogen

More Information

• Responsible Party: Biogen
• ClinicalTrials.gov Identifier: NCT05109637
• Other Study ID Numbers: DE-SMG-11894; CIV-21-06-036845 ( Registry Identifier: EUDAMED )
• First Posted: November 5, 2021
• Last Update Posted: February 6, 2024
• Last Verified: February 2024

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: Yes
• Plan Description: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/
• URL: https://vivli.org/

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Biogen:

Neuromuscular Diseases; Spinal Muscular Atrophy; Digital technology; Digital outcome assessments; Smartphone; Digital Biomarkers

Additional relevant MeSH terms:

Muscular Atrophy; Muscular Atrophy, Spinal; Neuromuscular Diseases; Atrophy; Pathological Conditions, Anatomical; Neuromuscular Manifestations; Neurologic Manifestations; Nervous System Diseases; Spinal Cord Diseases; Central Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases