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Investigating NMJ Defects in SMA Following Central and Peripheral SMN Restoration

NCT05219487 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 18

Last updated 2026-02-23

No results posted yet for this study

Summary

This is an observational study to investigate the improvement of NMJ defects in adult patients with SMA following treatment with Risdiplam. Eligible patients will have received treatment with daily oral Risdiplam after receiving approval through their commercial insurance or drug assistance program. All subjects will be evaluated at one visit. Eligible subjects must have been receiving risdiplam for at least 12 months.

Conditions

Interventions

DRUG

Risdiplam

Risdiplam is administered orally once daily and the recommended dosage is determined by age and body weight (see Table 1) \[as outlined in the US Prescribing Information for EVRYSDI™(risdiplam)\]. Table 1 Adult and Pediatric Dosing Regimen by Age and Body Weight: US Prescribing Information Age and Body Weight (Recommended Daily Dosage): 2 months to less than 2 years of age (0.2 mg/kg); 2 years of age and older weighing less than 20 kg (0.25 mg/kg); 2 years of age and older weighing 20 kg or more (5 mg)

Sponsors & Collaborators

  • Genentech, Inc.

    collaborator INDUSTRY

  • Bakri Elsheikh

    lead OTHER

Principal Investigators

  • Bakri Elsheikh, MBBS · Ohio State University

Eligibility

Min Age
18 Years
Max Age
70 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-10-13
Primary Completion
2025-12-31
Completion
2025-12-31

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05219487 on ClinicalTrials.gov