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

February 5, 2024 updated by: Biogen

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
93

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Contact Backup

Study Locations

  • Germany
      • Dresden, Germany, 01307
        • Research Site
      • Hannover, Germany, 30625
        • Research Site
      • Heidelberg, Germany, 69120
        • Research Site
      • Munich, Germany, 80336
        • LMU Klinikum Friedrich-Baur-Institute, Dept. of Neurology
    • North Rhine-Westphalia
      • Essen, North Rhine-Westphalia, Germany, 45147
        • Research Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years to 64 years (Adult)

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Other
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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: 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.
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.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
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.
Up to 28 days
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.
Up to 28 days

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
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.
Up to 28 days
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.
Up to 28 days
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.
Up to 28 days
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.
Up to 28 days
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.
Up to 28 days
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.
Up to 28 days
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.
Up to 28 days
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).
Up to 28 days
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.
Up to 28 days
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.
Up to 28 days
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.
Up to 28 days
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.
Up to 28 days
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.
Up to 28 days
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.
Up to 28 days
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.
Up to 43 days

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Biogen

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Medical Director, Biogen

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
February 10, 2022

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
July 10, 2023

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
July 25, 2023

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
October 27, 2021

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
October 27, 2021

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
November 5, 2021

Study Record Updates

Last Update Posted (Estimated)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
February 6, 2024

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 5, 2024

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
February 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • DE-SMG-11894
  • CIV-21-06-036845 (Registry Identifier: EUDAMED)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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