Recruiting

Observational Study

Sponsor:

Medstar Health Research Institute

Code:

NCT07223489

Conditions

Spinal Muscular Atrophy (SMA)

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

Telephone Interview

Study Details

Brief summary:

Evaluate the diagnostic journey, patient experience, and disparities in the treatment of Spinal Muscular Atrophy (SMA) in the MedStar Health System.

Conditions

Spinal Muscular Atrophy (SMA)

Study ID

NCT07223489

Start date

Oct 10, 2025

Status verified date

Jan, 2026

Completion date

Dec 30, 2026

Anticipated

Primary completion date

Jun 30, 2026

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • SMA diagnosis, age greater than or equal to 18 years

Exclusion Criteria:

  • Deceased

Study Design

Enrollment

200 participants

Anticipated

Interventions and Outcome Measures

Interventions

Telephone Interview

Telephone interviews to gather qualitative data on SMA patients' diagnostic journey, access to treatment and barriers encountered, understanding of their disease, and quality of life measures

Primary outcome measure

  • Proportion of SMA patients not receiving disease-modifying therapy and categorical causes for non-treatment based on structured patient/caregiver survey responses [ Time Frame: 2020-2025 ]

Central Contacts and Locations

Central contacts

Gabrielle Edwards, BS

2022219998gabrielle.edwards@medstar.net

Locations

MedStar Health Research Institute

Recruiting

Washinton, District of Columbia, United States, 20010

Contacts

Gabrielle Edwards, BS

2022219998gabrielle.edwards@medstar.net

Principal Investigator:

Nicholas Streicher, MD

More Information

Sponsor

Medstar Health Research Institute

Last update posted

Jan 16, 2026

Last verified

Jan, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Medstar Health Research Institute on 2026-01-16.