Children's Hospital is part of the UPMC family.
Be safe anytime, anywhere.
To find a pediatrician or pediatric specialist, please call 412-692-7337 or search our directory.
A resource for our network of referring physicians.
For more information about research, please call our main office at 412-692-6438.
Ranked #6 Nationally by U.S. News & World Report.
This multi-center international study tests the effects of Trappsol Cyclo combined with standard-of-care medication for people with Nieman-Pick disease, type C1 (NPC1). Standard of care may include other approved therapeutic drugs. NPC1 is a rare genetic disorder causing a buildup of lipids (fats) in the liver, brain, lungs, and spleen, which can lead to a decline in cognitive and motor function (movement), as well as a difficulty swallowing, sleep disturbances, and pneumonia.
Subject to certain exclusion criteria, this study is accepting people with NPC1, ages 3 and older.
Males and Females: Ages 3 years and older
Based on random selection, study participants will be divided into groups that will receive either the study drug or a placebo administered by intravenous infusion every 2 weeks at the study clinic while still receiving standard of care. During visits, participants will receive physical examinations and other tests that may include blood and urine sampling, electrocardiogram, and respiratory function tests among others.
Visits: At least 52
Duration: 23 months
Cyclo Therapeutics, Inc.
Study Description at National Institutes of Health
Genetic and Genomic Medicine Research
Damara Ortiz, MD
For more information about the study or enrollment, please contact:
Nadene Henderson, MS, LCGC
Children's Hospital's main campus is located in the Lawrenceville neighborhood. Our main hospital address is:
UPMC Children’s Hospital of Pittsburgh
One Children’s Hospital Way
4401 Penn Ave.
Pittsburgh, PA 15224
In addition to the main hospital, Children's has many convenient locations in other neighborhoods throughout the greater Pittsburgh region.
With myCHP, you can request appointments, review test results, and more.
For questions about a hospital bill call:
To pay your bill online, please visit UPMC's online bill payment system.
Interested in giving to Children's Hospital? Support the hospital by making a donation online, joining our Heroes in Healing monthly donor program, or visiting our site to learn about the other ways you can give back.