Archive for clinical trials
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 Yet clinical trials for rare diseases are frequently incomplete or go unpublished in the medical literature, finds a study published today in the journal PLOS Medicine.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
Racial differences in response to asthma therapies, and other AsthmaNet lessons
African Americans have higher rates of serious asthma attacks, hospitalizations, and asthma-related deaths than whites. Now, a large multicenter study of African Americans with poorly controlled asthma finds that one size doesn’t fit all when it comes to common asthma treatments. Results appear in The New England Journal of Medicine (NEJM). The randomized study had ... Read More
Exploring autism by way of three rare genetic disorders
Rajna Filip-Dhima, MS is a senior project manager for the Translational Neuroscience Center at Boston Children’s Hospital and for the Developmental Synaptopathies Consortium, which just received a new cycle of NIH funding. Autism spectrum disorder (ASD) is now believed to affect 1 in 59 children in the U.S. Over the past five years, Boston Children’s Hospital ... Read More
Following clinical trial, boy with Fanconi anemia transfusion free
Seven-year-old Ervis of Chicago, Illinois, is a model student with a positive attitude and a megawatt smile. His mom Ofelia calls him “un encantador” — “a charmer,” but life as Ervis knows it is not exactly charmed. Born with Fanconi anemia (FA), a rare hereditary blood disorder that can lead to bone marrow failure and cancer, ... Read More
Leading the way: Ava’s experience with teduglutide
Ava Orsini visits Boston Children’s Hospital every two weeks. It’s more time than any kid wants to spend at a hospital, particularly when she lives a state away. But for this 9-year-old and her parents, the trips are worth it: For the past two years, she’s been participating in a clinical trial of a drug ... Read More