Cure SMA Provides Funding and Recruitment for the NeuroNEXT SMA Biomarker Study: Super Babies for SMA!
A recent paper published in the Journal of Contemporary Clinical Trials Communications titled, “Recruitment & retention program for the NeuroNEXT SMA Biomarker Study: Super Babies for SMA!” highlights the challenges faced when recruiting infants with SMA into the NeuroNEXT study and how the study leaders were able to overcome those challenges with the help of advocacy groups, such as Cure SMA.
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) are a particularly challenging population to study due to their fragile and complex medical issues 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.
In 2011, the NeuroNEXT 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.
Prior to the finalization of the clinical protocol, the study collaborated with the Cure SMA. Cure SMA representatives provided feedback on the draft recruitment materials prior to the study going live. Once enrollment was open, Cure SMA actively worked to share and inform the SMA community of the recruitment materials and messages.
Every new patient registered with Cure SMA received a welcome packet of general SMA information that included the SMA Biomarker study brochure during the enrollment period. Cure SMA also circulated quarterly updates to ensure that people did not forget about the study. Furthermore, Dr. Stephen Kolb, the study’s Principal Investigator attended the annual, international Cure SMA family meeting as an outlet to share study data directly with the families affected with SMA.
To help reduce the financial burden of participation in the trial for families and optimize reliability of study measures, Cure SMA provided funds to reimburse families for travel expenses and as well as general funding for the study.
Enhancing Our Understanding of SMA Natural History
The NeuroNEXT study is an example of how development of 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. The success of the study has contributed to the interpretation of clinical trials in this population and to the approval of the ﬁrst FDA-approved medication for SMA and promises to contribute to numerous clinical trials involving the study of motor function in infants.
This study is critical because of its definitive and well-controlled data on the natural history of SMA type I. It showed that that motor function decreased rapidly in SMA infants, whereas it rapidly increased in the unaffected infants. In addition, by eight months of age, babies with two copies of SMN2 had typically reached an endpoint of either death or usage of ventilation (BiPap or a tracheotomy) for more than 16 hours a day.
This further reinforces the fact that early intervention is especially critical in SMA type I. It will also support continued efforts to add SMA to state newborn screening panels, as that represents the most effective way to ensure early treatment for those affected by SMA.