Medical Device Daily Washington Editor
GAITHERSBURG, Maryland – Roughly one out of 1,000 babies is born deprived of oxygen.
Now, a new device from Olympic Medical (Seattle) called Cool-Cap is a step closer to giving physicians a treatment option where few choices previously existed.
On Friday, the neurological devices panel of the FDA’s medical devices advisory committee voted 5-1, with one abstention, for approval with conditions.
The device is designed for infants younger than 36 weeks who suffer brain trauma from a loss of oxygen during delivery – called severe hypoxic-ischemic encephalopathy (HIE) – which can happen if the umbilical cord is in the wrong position, if the placenta comes loose from the uterus or if the uterus tears.
Cooling, or mild hypothermia, is thought to help prevent the damage that occurs when brain cells are deprived of oxygen and begin to die, especially within the critical six hours following birth.
But at present, there are few options open to doctors treating babies who suffer from HIE, and it is a problem most obstetricians are unable to predict or prevent.
“For this population, we have very little to offer and anything that would benefit this group of patients is a welcome addition,” said panel member Annapurni Jayam-Trouth, professor of neurology at Howard University (Washington).
Three families with newborns who received successful Cool-Cap treatment also testified on Friday. Accompanying them were their children as jumping, crying, laughing, examples of healthy, well-developed toddlers given a second chance by the device.
Pam Davies, MD, an obstetrician whose daughter Felicity was one of the Cool-Cap trial patients, said that the decision to participate in the study was an easy one.
“When you’re looking at a 50/50 chance of survival, with a number of possible complications,” she told Medical Device Daily, “the decision seemed obvious. Our daughter has developed normally; she’s healthy, happy and we may not have been having this conversation otherwise.”
Researchers believe a small window of opportunity exists after birth to prevent physical or mental disability later in life, including such conditions as cerebral palsy. The Cool-Cap cooling system is intended to reduce the brain’s need for oxygen and slow the process that continues to kill brain cells once blood flow resumes.
The device consists of a small hat strapped to the baby’s head with water channels running through it. Cold water circulates through the cap for a total of 72 hours, bringing the baby’s body temperature down to 94 degrees. The baby is then warmed up again slowly. In addition to the cap, other system components include the device’s computer touch-screen control unit, the cooling unit and a temperature sensor monitor.
For more than two years researchers studied 234 newborns at a total of 25 sites in the U.S., Canada, the UK and New Zealand.
According to results presented by Olympic and the FDA, 45% of babies treated with the Cool-Cap just after birth survived without serious brain damage, compared to 34% of babies treated with standard care. The study also showed, however, that the device was not as helpful for patients with the most severe brain injuries.
“We believe the benefits clearly outweigh any of the risks,” John Wyatt, professor of neonatal pediatrics and director of the Centre for Neonatology at University College London, told the panel. “We were proud to be associated with this device.”
Wyatt was one of the researchers based in the UK involved with Cool-Cap testing.
He said that before entering the market, the product would be made “more user-friendly,” with more interactive computer displays and a better manual for “how-to” help.
Conditions for approval recommended by the panel include that the device only be used in the kinds of babies studied in the trial, a company-sponsored training program for all Cool-Cap users and establishment of a registry of babies treated with Cool-Cap to continue studying the device’s effects.
Panelists all agreed that the device seemed to show safety and effectiveness. Most questions raised centered around the need for more data supporting efficacy and showing a more substantial positive difference between the patients who received Cool-Cap and those that received current standard of care, which include attempting to prevent asphyxia prior to birth and following birth, supportive treatment such as adequate ventilation and adequate perfusion and treatment of complications.
“I think the company has shown a reasonable assurance of safety and clinical effectiveness in the patient population,” said panelist Mary Jensen, MD, an interventional neuroradiologist at the University of Virginia (Charlottesville). “I look forward to further evidence that this is an effective treatment.”
The abstention came from Thomas Brott, MD, professor of neurology at the Mayo Clinic and Mayo Medical School (Jacksonville, Florida).
“I am very impressed that there is a need for this treatment; and I am very impressed with the very large randomized control trial carried out by the sponsor, which was very admirable,” he said. “However, I am concerned with whether or not it works. From the clinical point of view, I think there is room for bias. I am in the middle, which is why I abstained.”
The lone “no” vote came from Jonas Ellenberg, PhD, a biostatistician at the University of Pennsylvania School of Medicine (Philadelphia), who called the study design “exemplary and extraordinary,” but said he thought the data supporting the device’s effectiveness wasn’t compelling enough.
In late 2003, Olympic released its CFM 6000 device, a cerebral function monitor that received FDA marketing clearance in May of that year. It is designed for long-term patient monitoring in the neonatal intensive care unit and other acute-care departments.