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Completed

NCT Number: NCT05109637

A 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.

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Key information

Conditions

Spinal Muscular Atrophy Central Nervous System Diseases Motor Neuron Disease Muscular Atrophy, Spinal Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases Spinal Cord Diseases

Age range

18 year–64 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

Research Site, Essen, North Rhine-Westphalia, Germany

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Konectom NMD Application

Device

Administered as specified in the treatment arm.

Primary outcomes

  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 outcomes

  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.

Sponsors and collaborators

Lead sponsor

Biogen

Industry

Registry information

Official study title

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

Acronym: DigiNOA

Important dates

Study start
2022
Primary completion
2023
Study completion
2023
First posted
Nov 5, 2021
Registry last updated
Feb 6, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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