Related Experiment Video
Updated: Feb 6, 2026
Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts
Published on: May 11, 2018
Recruitment & retention program for the NeuroNEXT SMA Biomarker Study: Super Babies for SMA!
Amy Bartlett1, Stephen J Kolb1,2, Allison Kingsley1
1Department of Neurology, The Ohio State University Wexner Medical Center, Columbus, OH, United States.
Insights
Recruiting infants with spinal muscular atrophy (SMA) and healthy controls is feasible with a proactive plan. Understanding participant motivations, like the study's importance, is key for successful enrollment and retention in clinical research.
Area of Science:
- Pediatric Clinical Trials
- Neuromuscular Disorders
- Research Participant Recruitment
Background:
- Recruiting and retaining participants, especially infants with spinal muscular atrophy (SMA), presents significant challenges in clinical trials due to medical fragility and limited therapeutic options.
- Historically, recruiting healthy infants for studies has also been perceived as difficult.
Purpose of the Study:
- To assess the feasibility of recruiting infants with SMA and healthy controls for a longitudinal natural history study.
- To gather data supporting the analysis of planned clinical trials for infants with SMA.
- To understand factors influencing participation and retention in this vulnerable population.
Main Methods:
- A two-year longitudinal study enrolled infants with SMA and age-matched healthy controls, aiming for 27 participants in each group.
- A comprehensive recruitment and retention strategy was implemented.
- A survey was administered to families to identify determinants of study participation.
Main Results:
- The study successfully recruited all healthy infants within the first year and 26 out of 27 targeted infants with SMA within two years.
- Physicians/neurologists were the primary source of study information for 48% of families.
- Understanding the study's importance was the main driver for enrollment (58%) and retention (74%).
- Thematic recruitment tools, including social media and advocacy presentations, positively impacted enrollment decisions.
Conclusions:
- A proactive, inclusive recruitment strategy that emphasizes clear communication and community partnerships is effective for enrolling vulnerable populations.
- Integrating recruitment and retention plans early in clinical trial protocol development is recommended for successful study execution.
Background/Aims:
Recruitment and retention of research participants are challenging and critical components of successful clinical trials and natural history studies. Infants with spinal muscular atrophy (SMA) have been a particularly challenging population to study due to their fragile and complex medical issues, poor prognosis and, until 2016, a lack of effective therapies. Recruitment of healthy infants into clinical trials and natural history studies is also challenging and sometimes assumed to not be feasible.
Methods:
In 2011, our group initiated a two-year, longitudinal natural history study of infants with SMA and healthy infant controls to provide data to assist in the analysis and interpretation of planned clinical trials in infants with SMA. The recruitment goal was to enroll 27 infants less than 6 months of age with SMA and 27 age-matched healthy infants within the two-year enrollment period. A detailed recruitment and retention plan was developed for this purpose. In addition, a survey was administered to participant families to understand the determinants of participation in the study.
Results:
All healthy infants were recruited within the study's first year and 26 SMA infants were recruited within the two-year recruitment period. Thirty-eight participant families responded to the recruitment determinants survey. Nearly half of respondents (18/38, 48%) reported that they first heard of the study from their physician or neurologist. The most common reason to decide to enroll their infant (22/38, 58%) and to remain in the study (28/38, 74%) was their understanding of the importance of the study. Thematic recruitment tools such as a study brochure, video on social media, and presentations at advocacy meetings were reported to positively influence the decision to enroll.
Conclusions:
A proactive, thematic and inclusive recruitment and retention plan that effectively communicates the rationale of a clinical study and partners with patients, advocacy groups and the local communities can effectively recruit participants in vulnerable populations. Recommendations for the proactive integration of recruitment and retention plans into clinical trial protocol development are provided.
More Related Videos
Related Concept Videos
01:20Blood Studies for Cardiovascular System I: Cardiac Biomarkers
The essential diagnostic tools for detecting myocardial necrosis and monitoring individuals suspected of having acute coronary syndrome (ACS) include:
Troponins
Troponins, particularly cardiac troponins I and T, are the most precise and sensitive markers of myocardial injury. They are detectable within 4-6 hours of myocardial injury and remain...
01:37Super-resolution Fluorescence Microscopy
01:05Bioavailability Enhancement: Drug Stability Enhancement and GI Retention
01:22Case Studies
02:41Chromatin Structure Regulates pre-mRNA Processing
The chromatin structure, especially...
01:28Imaging Studies I: Kidney, Ureter, and Bladder Studies