The Cerebral Palsy Research Network (CPRN) submitted a significant multi-center grant application to the National Institute of Neurological Disorders and Stroke (NINDS), one of the National Institutes of Health (NIH), last month. Functional Improvement Related from Spasticity Treatments (FIRST) has been in the planning stages for several years and exemplifies the type of study that CPRN was created to conduct. FIRST, if funded, would engage 25 clinical centers across North America to look at children with ambulatory CP that receive botulinum toxin A (BTA) injections in their lower extremities to reduce spasticity and improve their walking. FIRST seeks to compare children receiving serial BTA injections to those that go on to receive a selective dorsal rhizotomy – a neurosurgery that seeks to permanently reduce or remove spasticity.
Several CPRN investigators worked on the development of FIRST which was submitted by three co-principal investigators including Kristie Bjornson, PT, PhD from Seattle Children’s Hospital, Paul Gross from the University of Utah and Jeffrey Leonard, MD from Nationwide Children’s Hospital. FIRST plans to enroll 1624 subjects across the 25 sites. The study design is observational in nature (patients are not assigned to interventions) and leverages significant expertise at the University of Utah with “causal inference” methodologies. This design is critical because it enables the testing of effectiveness of interventions in the real world setting of clinical practice without compromising the quality of the findings. The particular funding opportunity at NINDS to which this grant was submitted was specifically created to accommodate observational studies for conditions like CP where randomizing children to these interventions is not feasible.
The review process at NIH is thorough but also lengthy. The study team does not expect a funding decision before the Fall of 2020. But the process of assembling the study team, gathering preliminary data from the network and developing an application of this scale was a success in of itself. It demonstrated the deep commitment of the CPRN site investigators to improve outcomes for people with CP.