Spinal Muscular AtrophyDigiNOANCT05109637
A Study to Assess the Clinical Validity of Konectom™ in Adults Living With Neuromuscular Disorders
A study testing Konectom NMD Application in people with Spinal Muscular Atrophy, run by Biogen at 5 sites in Germany.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 64 years
- Conditions the study is for
- Spinal Muscular Atrophy
- What is being tested?
- Konectom NMD Application
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 93 participants
- Where
- 5 sitesGermany
- Study dates
- 10 Feb 2022to 25 Jul 2023
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 64 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- For PwSMA
- Genetic documentation of 5q SMA (homozygous gene deletion, mutation, or compound heterozygote).
- For Healthy Participants
- Age group matched with SMA participants
Who cannot join
- 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.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
Germany5 sites
Research Site
Dresden, 01307
Research Site
Essen, North Rhine-Westphalia, 45147
Research Site
Hanover, 30625
Research Site
Heidelberg, 69120
LMU Klinikum Friedrich-Baur-Institute, Dept. of Neurology
Munich, 80336
About this study
Official title: 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.
- Conditions
- Spinal Muscular Atrophy
- Being tested
- Konectom NMD Application
- Keywords
- Neuromuscular Diseases, Spinal Muscular Atrophy, Digital technology, Digital outcome assessments, Smartphone, Digital Biomarkers
- Medical subject headings
- Muscular Atrophy, Spinal, Neuromuscular Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Konectom NMD ApplicationDevice
Administered as specified in the treatment arm.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: 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. Receives: Device: Konectom NMD Application |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: Up to 28 days
Secondary outcomes (15)
Interclass Correlation Coefficient (ICC) of the Konectom Digital Outcome Assessment (DOA) Scores
This outcome measure will assess test-retest reliability of smartphone-based Konectom DOAs.
Measured: Up to 28 days
Type of Correlation of Upper Limb Konectom DOAs Versus Revised Upper Limb Module (RULM) in PwSMA
This outcome measure will assess the relationship between Konectom Upper Limb DOAs and conventional upper limb assessments in clinical environments.
Measured: Up to 28 days
Strength of Correlation of Upper Limb Konectom DOAs Versus RULM in PwSMA
This outcome measure will assess the relationship between Konectom Upper Limb DOAs and conventional upper limb assessments in clinical environments.
Measured: Up to 28 days
Type of Correlation of Upper Limb Konectom DOAs Versus 9-Hole Peg test (9HPT) in PwSMA
This outcome measure will assess the relationship between Konectom Upper Limb DOAs and conventional upper limb assessments in clinical environments.
Measured: Up to 28 days
Strength of Correlation of Upper Limb Konectom DOAs Versus 9HPT in PwSMA
This outcome measure will assess the relationship between Konectom Upper Limb DOAs and conventional upper limb assessments in clinical environments.
Measured: Up to 28 days
Type of Correlation of Lower Limb Konectom DOAs Versus 6-Minute Walk Test (MWT) Total Distance in Ambulatory PwSMA
This outcome measure will assess the relationship between Konectom Lower Limb DOAs and status of ambulation in clinical environments.
Measured: Up to 28 days
Strength of Correlation of Lower Limb Konectom DOAs Versus 6-MWT Total Distance in Ambulatory PwSMA
This outcome measure will assess the relationship between Konectom Lower Limb DOAs and status of ambulation in clinical environments.
Measured: Up to 28 days
Differences Between PwSMA and HS in the Konectom DOA Scores During Each Testing Condition
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).
Measured: Up to 28 days
Standard Deviation of Each Participant's Raw Konectom DOA Scores Over the At-Home Period
This outcome measure will assess the variability of Konectom DOAs self-administered in everyday environment in HS and PwSMA.
Measured: 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
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.
Measured: Up to 28 days
Type of Correlation of Konectom DOA Scores Versus Neuro-Quality of Life (QoL) Total Scores in PwSMA
This outcome measure will assess the relationship of Konectom DOAs against patient-reported outcomes (PROs) in PwSMA.
Measured: Up to 28 days
Strength of Correlation of Konectom DOA Scores Versus Neuro-QoL Total Scores in PwSMA
This outcome measure will assess the relationship of Konectom DOAs against PROs in PwSMA.
Measured: Up to 28 days
Type of Correlation of Konectom DOA Scores Versus Fatigue Severity Scale (FSS) Total Scores in PwSMA
This outcome measure will assess the relationship of Konectom DOAs against PROs in PwSMA.
Measured: Up to 28 days
Strength of Correlation of Konectom DOA Scores Versus FSS Total Scores in PwSMA
This outcome measure will assess the relationship of Konectom DOAs against PROs in PwSMA.
Measured: Up to 28 days
Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) Related to Konectom NMD Use
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.
Measured: Up to 43 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Nov 2021
- Study start10 Feb 2022
- Primary completion10 Jul 2023
- Study completion25 Jul 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Spinal Muscular AtrophyStudies of the same condition, open and under way.
- More trials run by BiogenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 93 participants (actual)
Sponsor and investigators
- Lead sponsor
- Biogen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Biogen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05109637
- Sponsor's protocol ID
- DE-SMG-11894
- Other IDs
- CIV-21-06-036845
- Acronym
- DigiNOA
- Status last verified
- Feb 2024
- First submitted
- 27 Oct 2021
- Last updated
- 6 Feb 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/
Study at a glance
From the registry record, last updated by the sponsor on 6 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study to Assess the Clinical Validity of Konectom™ in Adults Living With Neuromuscular Disorders
- ClinicalTrials.gov ID
- NCT05109637
- Acronym / sponsor ID
- DigiNOA / DE-SMG-11894
- Conditions
- Spinal Muscular Atrophy
- Keywords
- Neuromuscular Diseases, Spinal Muscular Atrophy, Digital technology, Digital outcome assessments, Smartphone, Digital Biomarkers
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 93 participants (actual)
- Lead sponsor
- BiogenIndustry (pharma, biotech, devices)
- Study start
- 10 Feb 2022 (actual)
- Primary completion
- 10 Jul 2023 (actual)
- Study completion
- 25 Jul 2023 (actual)
- First posted
- 5 Nov 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Participants with SMA (PwSMA) | Experimental | Device: Konectom NMD ApplicationParticipants with SMA will have their motor functions assessed using the Konectom NMD smartphone-based application up to Day 28. |
| Participants with ALS (PwALS) | Experimental | Device: Konectom NMD ApplicationParticipants 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. |
| Healthy Participants | Experimental | Device: Konectom NMD ApplicationHealthy participants will have their cognitive and motor functions assessed using the Konectom NMD smartphone-based application for 28 days. |
Who may qualify
- Ages
- 18 to 64 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (4 listed)
- 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 (8 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Konectom NMD ApplicationAdministered as specified in the treatment arm. | Device | Healthy Participants, Participants with ALS (PwALS), Participants with SMA (PwSMA) |
Primary outcome
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.
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.
15 secondary outcomes
Interclass Correlation Coefficient (ICC) of the Konectom Digital Outcome Assessment (DOA) Scores
Time frame: 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
Strength of Correlation of Upper Limb Konectom DOAs Versus RULM in PwSMA
Time frame: 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
Strength of Correlation of Upper Limb Konectom DOAs Versus 9HPT in PwSMA
Time frame: 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
Strength of Correlation of Lower Limb Konectom DOAs Versus 6-MWT Total Distance in Ambulatory PwSMA
Time frame: Up to 28 days
Differences Between PwSMA and HS in the Konectom DOA Scores During Each Testing Condition
Time frame: 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
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
Type of Correlation of Konectom DOA Scores Versus Neuro-Quality of Life (QoL) Total Scores in PwSMA
Time frame: Up to 28 days
Strength of Correlation of Konectom DOA Scores Versus Neuro-QoL Total Scores in PwSMA
Time frame: 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
Strength of Correlation of Konectom DOA Scores Versus FSS Total Scores in PwSMA
Time frame: 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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Feb 2024.
- Individual participant dataSharing planned: Yes. In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director study director
Biogen
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Konectom NMD Application in people with Spinal Muscular Atrophy, run by Biogen at 5 sites in Germany.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 6 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Type of Correlation of Konectom DOAs Versus Hammersmith Functional Motor Scale-Expanded (HFMSE) Total Score in PwSMA (time frame: Up to 28 days). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Feb 2024.