Archive for clinical trials
Two drugs join forces against COVID-19
Two drugs, including one developed by a researcher at Boston Children’s Hospital, inhibit the SARS-CoV-2 virus that causes COVID-19 in tests of human cells. Both drugs, vacuolin-1 and apilimod, originally developed years ago, target a large enzyme called PIKfyve kinase. Prior to this study, little was known about this enzyme’s role in COVID-19 infection. Details ... Read More
Trial tests dornase alfa, a cystic fibrosis drug, for severe COVID pneumonia
Boston Children’s Hospital and Brigham and Women’s Hospital will soon begin testing an existing drug, dornase alfa, in patients with severe COVID-19 pneumonia and respiratory failure. The randomized, controlled clinical trial aims to enroll 60 adults and children over age 3 who require mechanical ventilation. Key takeaways:· A randomized trial is testing whether a cystic ... Read More
Hydroxychloroquine and COVID-19: What we know so far
Despite the current hype, infectious disease experts and others are cautious about using chloroquine drugs in the current COVID-19 pandemic. While the urgent need for COVID-19 treatments justifies the rapid pace of trials, several concerns have been raised related to the limitations of studies published so far. Kristin Moffitt, MD The drugs chloroquine and hydroxychloroquine ... Read More
Could plasma from recovered COVID-19 patients help others?
As new cases of COVID-19 mount daily, treatment revolves around supportive therapy to reduce symptoms, meaning there are no treatments shown to slow down or kill the SARS-CoV-2 virus. One new idea actually isn’t so new: transfusing blood plasma from recovered COVID-19 patients into patients currently sick with the disease. Last week, the Food and Drug ... Read More
Migraine prevention in children and teens: Weak support for medication
Several medications successfully treat an active migraine. But a review of the medical literature from Boston Children’s Hospital finds only weak evidence that drug treatment prevents migraine in children and adolescents. Only two medications — topiramate and propranolol — showed any difference in preventing migraine in this age group compared with a placebo. Joe Kossowsky ... Read More
New clinical trial hopes to improve long-term outcomes for heart transplant patients
If your child or teenager has had a heart transplant in the last six months, they may be eligible to participate in a clinical trial developed to test a new anti-rejection therapy. In the process, they can play an important part in building a base of evidence that will help advance care for future generations. ... Read More
Gene therapy to boost fetal hemoglobin continues to do well in sickle cell trial
A pilot gene therapy treatment for sickle cell disease, restoring patients’ ability to make fetal hemoglobin, has produced good results in the first three patients to receive it. Investigators at Boston Children’s Hospital reported the findings of their ongoing clinical trial this week at the American Society of Hematology (ASH) annual meeting. The three adult ... Read More
Low enrollment stymies completion of rare disease clinical trials
Rare diseases are anything but rare when taken together. In the United States alone, they affect nearly 30 million people. Yet clinical trials for rare diseases are frequently incomplete or go unpublished in the medical literature, finds a study published today in the journal PLOS Medicine. Analyzing 659 trials, researchers at Boston Children’s Hospital found ... Read More
Diet trials often amend their outcome measures as they go
Ever wonder why science can’t settle simple diet-related questions? In part it’s because most clinical trials involving diet, even those published in good journals, lack the rigor of most drug trials. They’re often small, of short duration, and unable to rigorously control subjects’ adherence to the test diets. Moreover, a study published today in JAMA ... Read More
While enjoying college, Ewing sarcoma survivor looks to her past
It lacks sunsets and skylines, but Maddie Carlson loves the view from her dorm room at Emmanuel College in Boston. If the 20-year-old junior sticks her head out the window at just the right angle, she can almost make out a portion of the place she credits for saving her life after she was diagnosed ... Read More