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

study id #: NCT05109637

condition: Spinal Muscular Atrophy, Amyotrophic Lateral Sclerosis

status: Not yet recruiting

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) or person with amyotrophic lateral sclerosis (PwALS) and healthy subjects (HS); to evaluate the variability of Konectom DOAs self-administered in everyday environment in HS and PwSMA or PwALS; to compare Konectom DOAs between in-clinic supervised administration versus self-assessments in everyday environment in HS, PwSMA and PwALS groups; to evaluate the relationship of Konectom DOAs against patient-reported outcomes (PROs) in PwSMA or PwALS and to evaluate the clinical safety of Konectom in PwSMA and PwALS.

intervention: Konectom NMD Application

results: https://clinicaltrials.gov/ct2/show/results/NCT05109637

last updated: February 04, 2022

start date: December 7, 2021

estimated completion: August 22, 2022

last updated: November 5, 2021

phase of development: Not Applicable

size / enrollment: 160

primary outcomes:

• 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
  Type of Correlation of Konectom DOAs Versus Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) Total Score in PwALS
  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 ALSFRS-R Total Score in PwALS
  This outcome measure will assess the convergent validity of smartphone-based Konectom DOAs against in-clinic standard assessments.
• Up to 28 days
  

secondary outcomes:

• Interclass Correlation Coefficient (ICC) of the Konectom Digital Outcome Assessment (DOA) Scores
  Up to 28 days
• Type of Correlation of Upper Limb Konectom DOAs Versus Revised Upper Limb Module (RULM) in PwSMA
  Up to 28 days
• Strength of Correlation of Upper Limb Konectom DOAs Versus RULM in PwSMA
  Up to 28 days
• Type of Correlation of Upper Limb Konectom DOAs Versus Penn Upper Motor Neuron Score (PUMNS) in PwALS
  Up to 28 days
• Strength of Correlation of Upper Limb Konectom DOAs Versus PUMNS in PwALS
  Up to 28 days
• Type of Correlation of Upper Limb Konectom DOAs Versus 9-Hole Peg test (9HPT) in PwSMA and PwALS
  Up to 28 days
• Strength of Correlation of Upper Limb Konectom DOAs Versus 9HPT in PwSMA and PwALS
  Up to 28 days
• Type of Correlation of Lower Limb Konectom DOAs Versus 6-Minute Walk Test (MWT) Total Distance in Ambulatory PwSMA and PwALS
  Up to 28 days
• Strength of Correlation of Lower Limb Konectom DOAs Versus 6-MWT Total Distance in Ambulatory PwSMA and PwAL
  Up to 28 days
• Differences Between PwSMA or PwALS and HS in the Konectom DOA Scores During Each Testing Condition
  Up to 28 days
• Standard Deviation of Each Participant's Raw Konectom DOA Scores Over the At-Home Period
  Up to 28 days
• Paired-Comparisons of Konectom DOA Scores Between In-Clinic Supervised Administration and Self-Assessment In Everyday Environment, Separately for HS, PwSMA and PwALS Groups
  Up to 28 days
• Type of Correlation of Konectom DOA Scores Versus Neuro-Quality of Life (QoL) Total Scores in PwSMA or PwALS
  Up to 28 days
• Strength of Correlation of Konectom DOA Scores Versus Neuro-QoL Total Scores in PwSMA or PwALS
  Up to 28 days
• Type of Correlation of Konectom DOA Scores Versus Fatigue Severity Scale (FSS) Total Scores in PwSMA or PwALS
  Up to 28 days
• Strength of Correlation of Konectom DOA Scores Versus FSS Total Scores in PwSMA or PwALS
  Up to 28 days
• Type of Correlation of Konectom DOA Scores Versus 5-Item ALS Assessment Questionnaire (ALSAQ-5) Total Scores in PwALS
  Up to 28 days
• Strength of Correlation of Konectom DOA Scores ALSAQ-5 Total Scores in PwALS
  Up to 28 days
• Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) Related to Konectom NMD Use
  Up to 43 days

inclusion criteria:

exclusion criteria: 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.

sponsor: Biogen

contacts: US Biogen Clinical Trial Center, 866-633-4636, [email protected]

investigators: Medical Director,Biogen

trial center locations: Germany

• A Study to Assess the Clinical Validity of Konectom™ in Adults Living With Neuromuscular Disorders
  +49 201 7236513, [email protected]