Researchers from St. Louis Children’s Hospital at Washington University School of Medicine are enrolling infants 0-6 months with diagnosed NPC disease and evidence of liver disease. The purpose of the study is to learn how well adrabetadex can reduce liver damage in infants diagnosed with NPC disease. The study has an initial 6-week phase during which the study participant will receive twice-weekly intravenous administration of the drug, adrabetadex. If there is evidence of clinical response, the participant is eligible to continue treatment for an additional 6 months. The National Institute of Child Health and Human Development of the National Institute of Health is funding this study.
More information please contact Patricia Dickson, MD: 314-273-2943, pdickson@wustl.edu, Barbara Warner, MD: 314-454-2683, warnerbb@wustl.edu, or Laura Linneman, RN: 314-454-4950, llinneman@wustl.edu. More information can be found on https://clinicaltrials.gov/ct2/show/NCT03471143?cond=VTS-270&rank=2
Here are more details on the trial:
PURPOSE OF THE STUDY:
The purpose of the study is to learn how well Adrabetadex can reduce liver damage in infants diagnosed with NPC disease.
WHO WILL BE ENROLLED:
Infants 6 months old or less with diagnosed NPC disease and evidence of liver disease are eligible to be screened for the trial.
WHAT IS INVOLVED IF I PARTICIPATE?
The study has an initial 6-week phase during which the study participant will receive twice-weekly intravenous administration of the Adrabetadex drug. If there is evidence of clinical response, the participant is eligible to continue treatment for an additional 6 months.
WHO IS FUNDING THE STUDY?
The National Institute of Child Health and Human Development of the National Institutes of Health
WHO IS THE SPONSOR?
Patricia Dickson, MD
WHO IS THE PRINCIPAL INVESTIGATOR (PI)?
Barbara Warner, MD
WHERE WILL THE STUDY TAKE PLACE?
St. Louis Children’s Hospital at Washington University Medical Center
WHO DO I CONTACT FOR MORE INFORMATION?
Patricia Dickson, MD
314-273-2943
Barbara Warner, MD
314-454-2683
Laura Linneman, RN
Research Nurse Coordinator
314-454-4950
More information can be found on https://clinicaltrials.gov
