Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA) (WeSMA)

Genentech

Status and phase: Active, not recruiting; Phase 4

Conditions: Spinal Muscular Atrophy

Treatments: Drug: Risdiplam; Device: SMA-DAT Application and ADAM Sensor

Study type: Interventional

Funder types: Industry

Identifiers

NCT05232929

ML43702

Details and patient eligibility

About

A multi-center, longitudinal, prospective, non-comparative study to investigate the long-term safety and effectiveness of risdiplam, prescribed based on clinician judgment as per the Evrysdi® U.S. Package Insert (USPI) in adult and pediatric participants with SMA. In this study, participants will be followed for the duration of the study or until withdrawal of consent, loss to follow-up, or death. Participants who discontinue risdiplam may still remain in the study, if they agree to continue participating in the follow-up assessments.

An optional sub study will assess the feasibility, acceptability, and adherence of remote assessment of motor and bulbar functions in participants with SMA using wearable and smartphone-based biosensors. Approximately 39 participants from the main study are planned to be enrolled in the sub study.

Enrollment: 402 patients

Sex: All

Volunteers: No Healthy Volunteers

Inclusion criteria

• Clinical diagnosis of SMA
• Prescribed or continued risdiplam based on clinical judgment of prescriber, as per the Evrysdi® USPI, after U.S. FDA approval (07 August 2020)

Sub study:

Participants in the main study (ML43702) are eligible to be included in the sub study only if all of the following criteria apply:

• Age ≥ 10 years at the time of signing Informed Consent Form
• Willingness and ability to use smartphone technology
• Fluency in English (written and spoken as per the judgment of the investigator)
• Willingness and ability to complete all aspects of the sub study, including respiration and swallowing measurements using respiratory inductance plethysmography (RIP) belts and surface electromyography (sEMGs)
• Hammersmith Functional Motor Scale-Expanded (HFMSE) > 10
• Functional Oral Intake Scale (FOIS) >1
• Willingness to be video recorded during in-clinic SMA-DAT tasks and ADAM sensor assessments and training
• Availability of a caregiver who is willing to participate throughout this sub study

Exclusion criteria

• Hypersensitivity to risdiplam
• Participated in a registrational trial for risdiplam (i.e., Firefish [NCT02913482], Sunfish [NCT02908685], Jewelfish [NCT03032172], and Rainbowfish [NCT03779334])

Sub study:

Potential participants will be excluded from the sub study if they meet any of the following criteria:

• Current respiratory infection that, in the opinion of the investigator, would interfere with the conduct of the sub study
• History or known presence of any significant psychiatric disorder such as schizophrenia, bipolar disorder, or substance use disorders
• Current active clinically significant anxiety or depressive disorder, as judged by the investigator, that is likely to impede a participant's ability to participate in the sub study
• Wearing a pacemaker (due to incompatibility with the ADAM sensor)
• Inability to tolerate the performance of sub study procedures

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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402 participants in 2 patient groups

Risdiplam

Experimental group

Description:

Participants who are taking risdiplam prescribed based on clinician judgment, as per the Evrysdi® USPI are enrolled in this arm.

Treatment:

Drug: Risdiplam

Substudy Cohort

Experimental group

Description:

Participants from the main study will be enrolled in three sub-groups based on their Functional Oral Intake Scale (FOIS) score. Participants will be provided with a preconfigured study smartphone with the spinal muscular atrophy digital assessment tool (SMA-DAT) application and an ADvanced Acousto-Mechanic (ADAM) sensor. Participants will be asked to perform a programmed selection of SMA-DAT tasks for 24 days of the 27-day remote monitoring period.

Treatment:

Device: SMA-DAT Application and ADAM Sensor