Johns Hopkins All Children’s Hospital received a $2.8 million grant for a phase 3 clinical trial on treatment of blood clots in children.
The National Institutes of Health (NIH) grant award, one of two recently given to Johns Hopkins All Children’s Hospital, is designed to help determine the length of time infants, children and teens should be treated with anti-clotting medication after a first-time acute venous thromboembolism (VTE), a blood clot in a vein.
Under the grant, the St. Petersburg, Florida pediatric hospital would expand its international clinical trial called Kids-DOTT (Duration of Therapy for Thrombosis) for five years. A separate NIH grant was awarded for Kids-DOTT, to fund the trial's Data Coordinating Center in Colorado.
“The main goal of this clinical trial is to definitively establish how long children and teens who develop a first episode of VTE, and who do not have a medical history suggesting they are at increased risk for developing recurrent blood clots, should be treated with anti-clotting medications,” said Johns Hopkins All Children’s pediatric hematologist Neil Goldenberg, M.D., Ph.D., the grant’s principal investigator.
The trial addresses an important public health need because clots in children are occurring more frequently: approximately 1 in 200 hospitalized children and 1 in 10,000 children overall.
“Because anti-clotting medications increase the risk of serious bleeding, some physicians now recommend only six weeks of treatment for children who are at low risk for developing new blood clots, but there is no scientific evidence to support this approach,” explained Goldenberg. “The main goal for the Kids-DOTT trial is to provide evidence that defines the standard of care by determining whether six weeks of treatment is as safe and effective as three months.”
The shorter treatment also reduces costs and spares patients additional weeks of invasive treatment, often involving twice daily injections of anti-clotting medication or additional blood draws to monitor effectiveness.
The answers provided by the trial will help patients like Kincade, a 14-year-old with cystic fibrosis who has received care at Johns Hopkins All Children’s Hospital for the past decade. In 2015 he had a lung infection that required antibiotics administered through a central venous catheter (also called a port). When the port needed to be replaced, an ultrasound revealed three blood clots in a subclavian vein.
“Kincade was fortunate that the blood clots were discovered before they caused problems like swelling of the arm,” recalls his mother, Jennifer. “The hematology-oncology team explained the treatment options and the risks and benefits of each. They also told us about the Kids-DOTT trial and we decided to enroll. We know Kincade has benefited from the people before us who enrolled in research studies. Now we can help people who have these problems in the future.”
The Kids-DOTT study is one of the largest randomized trials ever performed in the field of pediatric blood clots, with a collaboration of more than 40 children’s hospitals from around the world. More than 800 patients are expected to participate in the trial, with approximately 280 children enrolled to date.
Johns Hopkins All Children’s Hospital also received a second NIH grant as part of a study led by a team of principal investigators at Duke University Clinical Research Institute, Johns Hopkins All Children’s Hospital and Vanderbilt University. Co-principal investigator and Johns Hopkins All Children’s cardiovascular surgeon Jeffrey Jacobs, M.D. explains, “The aim of this study is to leverage existing resources of a clinical, quality and research registry toward the conduct of simple, efficient and low-cost clinical trials in understudied patient populations, such as neonates undergoing heart surgery. The hope is that this approach will accelerate putting new and optimal treatment approaches into clinical practice.”
Both NIH awards are U01 grants, characteristically reserved for multi-center studies bringing researchers together from multiple disciplines and institutions, including NIH itself.
Clinical professionals: Sign up for CDC webinar on venous thromboembolism and blood disorders on Nov. 3, featuring Johns Hopkins All Children's expert, Dr. Neil Goldenberg.