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

NCT05109637 | Completed

• 2 other identifiers: DE-SMG-11894CIV-21-06-036845
• Study Type: interventional
• Target: 93
• Locations: 1 country
• Sites: 5

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.

Conditions

Spinal Muscular Atrophy

Keywords

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

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (15)

• 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 9-Hole Peg test (9HPT) in PwSMA

  Up to 28 days

• Strength of Correlation of Upper Limb Konectom DOAs Versus 9HPT in PwSMA

  Up to 28 days

Study Arms (3)

Participants with SMA (PwSMA)

EXPERIMENTAL

Participants with SMA will have their motor functions assessed using the Konectom NMD smartphone-based application up to Day 28.

Device: Konectom NMD Application

Participants with ALS (PwALS)

EXPERIMENTAL

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

Healthy Participants

EXPERIMENTAL

Healthy participants will have their cognitive and motor functions assessed using the Konectom NMD smartphone-based application for 28 days.

Device: Konectom NMD Application

Interventions

Konectom NMD Application DEVICE

Administered as specified in the treatment arm.

Healthy Participants; Participants with ALS (PwALS); Participants with SMA (PwSMA)

Eligibility Criteria

• Age: 18 Years - 64 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• For PwSMA
• Genetic documentation of 5q SMA (homozygous gene deletion, mutation, or compound heterozygote).
• For Healthy Participants
• Age group matched with SMA participants

You may not qualify if:

• 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.

Sponsors & Collaborators

• Biogen: lead

Study Sites (5)

Research Site

Essen, North Rhine-Westphalia, 45147, Germany

Location

Research Site

Dresden, 01307, Germany

Location

Research Site

Hanover, 30625, Germany

Location

Research Site

Heidelberg, 69120, Germany

Location

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

Munich, 80336, Germany

Location

MeSH Terms

Conditions

Muscular Atrophy, Spinal; Neuromuscular Diseases

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases

Study Officials

• Medical Director

  Biogen

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: OTHER
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 27, 2021
• First Posted: November 5, 2021
• Study Start: February 10, 2022
• Primary Completion: July 10, 2023
• Study Completion: July 25, 2023
• Last Updated: February 6, 2024

Record last verified: 2024-02

Data Sharing

• IPD Sharing: Will share

Locations

DE (5)