The classic website will no longer be available as of June 25, 2024. Please use the modernized ClinicalTrials.gov.
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05109637
Recruitment Status : Completed
First Posted : November 5, 2021
Last Update Posted : February 6, 2024
Sponsor:
Information provided by (Responsible Party):
Biogen

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

Layout table for study information
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


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.




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.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
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.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05109637


Locations
Layout table for location information
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
Layout table for investigator information
Study Director: Medical Director Biogen
Layout table for additonal 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    Key Record Dates
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/

Layout table for additional information
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:
Layout table for 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