Key Takeaways
The study was evaluating an investigational therapy for spinal muscular atrophy (SMA) in newborns. To be eligible to participate, newborns had to be genetically diagnosed with SMA, pre-symptomatic and ≤ 6 weeks of age.
Because of the rare nature of the disease and the narrow eligibility criteria, it was important that all potentially eligible patients were screened, and that those enrolled remained enrolled for the entire length of the study.
The study was considered a landmark study for SMA, and the investigational therapy has since been approved as the first treatment for SMA in pediatric patients.
Solution and Impact
Logistical and financial barriers to participation were eliminated with BBK’s TCNTM Arrive travel program and TCNTM Card reimbursement program.
Travel support was customized based on individual needs and included visa procurement, patient ambassadors to assist with translation, international flights and long-term housing accommodations, as needed.
BBK’s concierge specialists ensured that no screening opportunity was missed, often coordinating complex travel support for visits occurring within 24-48 hours.
Reimbursements were immediate, contributing to overall study satisfaction.
-
6
Randomization needed to occur within six weeks of birth.
-
19
At the time of BBK’s engagement, the client had enrolled six patients — BBK enrolled the remaining 19 patients needed to meet the enrollment goal of 25.
-
8
The study enrolled eight months ahead of schedule.
Related Resources
Familial Amyloid Polyneuropathy — Neurology and Gene Therapy — Phase 3
Case Studies
October 12, 2021
Major Depressive Disorder — Mental Health — Pivotal Phase 3 Study
Case Studies
February 5, 2022
Pediatric Boys with Duchenne Muscular Dystrophy — Rare and Gene Therapy — Phase 3B
Case Studies
April 13, 2022
Terms & Conditions Privacy Policy
© 2024 BBK Worldwide. BBK Worldwide is a Publicis Health company.
Terms & Conditions Privacy Policy
© 2024 BBK Worldwide. BBK Worldwide is a Publicis Health company.