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

Spinal Muscular Atrophy Clinical Trial

— DigiNOA  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT05109637
• Other study ID #: DE-SMG-11894
• Secondary ID: CIV-21-06-036845
• Status: Completed
• Phase: N/A
• Start date: February 10, 2022
• Est. completion date: July 25, 2023

Study information

• Verified date: February 2024
• Source: Biogen
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical 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 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 93
• Est. completion date: July 25, 2023
• Est. primary completion date: July 10, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 64 Years

Eligibility

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.

Related Conditions & MeSH terms

• Atrophy
• Muscular Atrophy
• Muscular Atrophy, Spinal
• Neuromuscular Diseases
• Spinal Muscular Atrophy

Intervention

Device:
• Konectom NMD Application
Administered as specified in the treatment arm.

Locations

Country	Name	City	State
Germany	Research Site	Dresden
Germany	Research Site	Essen	North Rhine-Westphalia
Germany	Research Site	Hannover
Germany	Research Site	Heidelberg
Germany	LMU Klinikum Friedrich-Baur-Institute, Dept. of Neurology	Munich

Sponsors (1)

Lead Sponsor	Collaborator
Biogen

Country where clinical trial is conducted

Germany, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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	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.	Up to 28 days
Secondary	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.	Up to 28 days
Secondary	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.	Up to 28 days
Secondary	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.	Up to 28 days
Secondary	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.	Up to 28 days
Secondary	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.	Up to 28 days
Secondary	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.	Up to 28 days
Secondary	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).	Up to 28 days
Secondary	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.	Up to 28 days
Secondary	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.	Up to 28 days
Secondary	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.	Up to 28 days
Secondary	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.	Up to 28 days
Secondary	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.	Up to 28 days
Secondary	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.	Up to 28 days
Secondary	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.	Up to 43 days