A Roanoke researcher will head up a clinical trial that could lead to establishing a national standard of care for babies who have strokes.
The National Institutes of Health has awarded a $13.5 million grant to Sharon Landesman Ramey of the Fralin Biomedical Research Institute at VTC, and to Warren Lo, a pediatric neurologist at Ohio State University, to determine if intensive bouts of therapy will help babies learn to use arms and legs that have been paralyzed by stroke.
Ramey and Stephanie DeLuca, co-directors of the institute’s Neuromotor Research Clinic, developed the treatment 20 years ago for children with neuromuscular problems, but this is the first time the technique will be used solely on babies and toddlers weakened on one side from stroke.
“We’ve done a lot of Phase 2 studies. We’ve published the results, and children in many places are starting to get our treatment. But the vast majority of children eligible for this are not getting it because there is no Phase 3 trial,” Ramey said.
“We need to learn if it is as good as we think by doing a larger, more rigorous clinical trial,” she said. “Then the results of this will impact what pediatric neurologists, pediatricians, parents, physical therapists, occupational therapists do in the future for all the children who qualify. That’s why it’s so thrilling.”
Ramey and Lo have worked together on Phase 2 trials. They have recruited therapists from 12 research centers across the country, who will come to Roanoke in June to be trained in the technique. Ramey expects the first babies will undergo the therapy starting in August.
About 240 babies will be enrolled in the study over the next few years. One-third will continue with whatever therapy they are undergoing. One-third will be given three hours of therapy, five days a week for four weeks. And the final third will be given six hours of therapy, five days a week for four weeks.
While parents and therapists will know to which group the child is assigned, those assessing the baby before, immediately after and then again at six months will not.
Children in the control group will be offered intensive therapy after the six-month assessment.
Ramey said they want to find out if three hours a day of therapy is as effective as six.
“A big debate has been whether three hours produces benefits as large and as lasting as the six-hour dosage. So this will answer this question,” she said. “It would be great if the three-hour is just as good as the six-hour. Wonderful. If the six-hour is a lot better, then we need to know that. This is the rest of the child’s life, and being able to use an arm and a hand affects balance, crawling and walking.”
Depending on how quickly babies are enrolled, the answer could come in less than four years.
The babies will be as young as 8 months and as old as 24 months.
Usually, by 8 months, babies transfer objects between hands, use their arms to sit up, start to wave and point at objects, and learn to play patty-cake. Babies weakened from stroke can’t do these things.
About 3,000 infants a year in the U.S. are diagnosed with strokes that occur either before birth or within their first month.
“The good news is about half the babies who have strokes don’t have a functional impairment for the rest of their life, or it’s so mild it doesn’t matter,” she said.
The other half may drag a leg when attempting to crawl or are unable to use an arm and hand. They can also have language and cognitive problems.
Ramey said usually children are given the low doses of an hour of physical therapy and an hour of occupational therapy each week, even though it has been shown to not work.
“The field of pediatric rehabilitation has not invested in rigorous scientific studies,” she said.
This study will include 12 centers in Ann Arbor, Michigan; Atlanta; Baltimore; Boston; Chicago; Cincinnati and Columbus, Ohio; Houston; New Haven, Connecticut; Philadelphia and San Diego. When possible, the treatments will be delivered in the child’s home.
Roanoke researchers will most likely work with children whose families travel here for the therapy. The treatment will be delivered without charge, but families will need to pay for their travel and living expenses.
Parents will also be trained in what they should do following the therapy, and they might be required to come for follow-up visits.
Ramey said most children who have participated in Phase 2 trials have shown dramatic improvement.
“We had children who were predicted by the neurologist to never use an arm, to never walk, to be cognitively impaired, who are very accomplished, who are walking, who are doing very well in school, who play sports and do fine motor activity,” she said. “It’s just phenomenal.”
She cautions that the therapy is a treatment, not a cure.