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Completed No phase listed Observational

Investigating NMJ Defects in SMA Following Central and Peripheral SMN Restoration

ClinicalTrials.gov ID: NCT05219487

Public ClinicalTrials.gov record NCT05219487. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

ClinicalTrials.gov public records Last synced Nov 4, 2026, 11:07 PM EST

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Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

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.

Study identification

NCT ID
NCT05219487
Recruitment status
Completed
Study type
Observational
Phase
Not listed
Lead sponsor
Bakri Elsheikh
Other
Enrollment
18 participants

Conditions and interventions

Interventions

Drug

Eligibility (public fields only)

Age range
18 Years to 70 Years
Sex
All
Healthy volunteers
Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date
Oct 13, 2021 (Actual)
Primary completion
Dec 31, 2025 (Actual)
Completion
Dec 31, 2025 (Actual)
Last update posted
Feb 23, 2026

2021 – 2025

United States locations

U.S. sites
1
U.S. states
1
U.S. cities
1
Facility City State ZIP Site status
The Ohio State University Wexner Medical Center Columbus Ohio 43210 —

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT05219487, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from
Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh
Last update posted Feb 23, 2026 · Synced Nov 4, 2026

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Open the official record

The complete public registry record, listed eligibility criteria, and available contact information for NCT05219487 live on ClinicalTrials.gov.

View official ClinicalTrials.gov record →