Where are all the pediatric clinical trials?
Two researchers at Boston Children’s Hospital found a disturbing trend when they searched for data on registered trails involving children.
Natalie Pica, MD and and Florence Bourgeois, MD, MPH took a retrospective, cross-sectional study of pediatric randomized clinical trials (RCTs) registered on ClinicalTrials.gov from 2008 to 2010 and found that 19 percent of were discontinued early. Of those that were actually completed, two thirds had still not published results after an average of 58 months.
Many studies do not publish data because technically, they are only required to report on Clinicaltrials.gov. However, without undergoing appropriate analysis, that data isn’t useful to others.
Our sister blog Vector spoke with Pica and Bourgeois to further investigate this disturbing trend and report on the various roadblocks facing pediatric RTCs.
Read Vector‘s coverage of unpublished pediatric trials.
About 1 in 10 Americans have some form of liver disease. One rare, under-recognized disorder, lysosomal acid lipase (LAL) deficiency—which prompts a build-up of fat and cholesterol in the liver, spleen and other parts of the body—can fly under the radar until it becomes life-threatening, often requiring a liver transplant.
“LAL deficiency, to some degree, begins at birth and slowly damages the liver to the point where patients who were asymptomatic no longer have enough liver function and drop off the edge,” Edward Neilan, MD, PhD, of Boston Children’s Hospital’s Metabolism Program recently told the Vector blog. Symptoms, he adds, “might not appear until you are 30 years old.”
Neilan is the Boston Children’s principal investigator for an international clinical trial for children with LAL deficiency. Learn more about LAL deficiency and the trial on Vector.