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

Biogen

Status: Completed

Conditions: Spinal Muscular Atrophy

Treatments: Device: Konectom NMD Application

Study type: Interventional

Funder types: Industry

Identifiers

NCT05109637

CIV-21-06-036845 (Registry Identifier)

DE-SMG-11894

Details and patient eligibility

About

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.

Enrollment: 93 patients

Sex: All

Ages: 18 to 64 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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.

Trial design

Primary purpose: Other

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

---

93 participants in 3 patient groups

Participants with SMA (PwSMA)

Experimental group

Description:

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

Treatment:

Device: Konectom NMD Application

Participants with ALS (PwALS)

Experimental group

Description:

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.

Treatment:

Device: Konectom NMD Application

Healthy Participants

Experimental group

Description:

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

Treatment:

Device: Konectom NMD Application