Case Study: Highest-Enrolling U.S. Site in Generalized Myasthenia Gravis Clinical Trial
How direct EMR access, targeted pre-screening, and an engaged Investigator helped the site reach 200% of its enrollment goal
Recruiting for a rare and complex neuromuscular disease study can present significant challenges, particularly when strict eligibility criteria leave only a small subset of patients who qualify. For one generalized myasthenia gravis (gMG) clinical trial, Profound Research needed to identify patients who met strict clinical, serologic, and disease-severity thresholds. Background treatment stability requirements and washout periods for several commonly used MG therapies narrowed eligibility further.
Profound combined direct electronic medical record (EMR) access, targeted pre-screening, and a highly engaged Investigator to create a highly focused recruitment pathway.
The result: the site enrolled 4 participants in just over 6 months, achieved 200% of its enrollment goal, and finished as the highest-enrolling U.S. site.
The Challenge: Finding a Small Subset of Eligible gMG Patients
Enrollment required identifying a small subset of patients with gMG who met strict clinical, serologic, and disease-severity thresholds. Eligibility was further narrowed by background treatment stability requirements and washout periods for several commonly used MG therapies. Finding patients who qualified meant looking closely at both diagnosis and treatment history.
The Solution: EMR-Driven Pre-Screening and Hands-On Investigator Involvement
With direct access to the neurology practice’s EMR, the research team proactively identified patients with established MG diagnoses and reviewed relevant treatment history before outreach. A highly engaged Investigator worked closely with the team to assess potential candidates, confirm clinical fit, and identify additional patients from the practice. Together, EMR-driven pre-screening and hands-on Investigator involvement created a highly focused recruitment pathway.
The Outcome: Highest-Enrolling U.S. Site at 200% of Goal
The site enrolled 4 participants in just over 6 months, achieving 200% of its enrollment goal and finishing as the highest-enrolling U.S. site.
Key Study Achievements:
- Highest-enrolling U.S. site
- Achieved 200% of enrollment goal
- Site Greenlight: April 9, 2026
- Enrollment Closed: October 12, 2026
What This Means for Sponsors
In a rare disease study with strict eligibility requirements, the number of patients who qualify can be very small. Reaching them depends on knowing which patients to look at and confirming their fit before outreach. In this gMG trial, direct EMR access allowed the research team to review diagnoses and treatment history up front, and an engaged Investigator helped confirm clinical fit and identify additional patients from the practice.
For sponsors running rare and complex neuromuscular disease studies, this case highlights the impact of direct EMR access, Investigator engagement, and targeted pre-screening.
Looking Ahead
Strict serologic, disease-severity, and treatment-history criteria will continue to shape recruitment in rare disease research. This case shows how focused pre-screening and close Investigator collaboration can help a site find and enroll the right patients for these studies. At Profound Research, we remain focused on strengthening Investigator relationships and integrating recruitment and retention efforts to help sponsors navigate enrollment challenges, support study participation, and advance clinical research.