Trial Name: Oral Treprostinil in Pediatric Patients with Pulmonary Arterial Hypertension (TDE-PH-206)
This study will evaluate the safety, tolerability and pharmacokinetics of oral treprostinil following transition from intravenous remodulin or inhaled prostacyclin or as add-on to current PAH therapy in de novo prostacyclin pediatric patients (age 7-17 years) with pulmonary arterial hypertension.
Enrollment goal: (4-7 patients)
Contact: Mary Mullen, MD, PhD (firstname.lastname@example.org), 617-355-7866
Trial Name: Phase Ii Randomized, Placebo-Controlled, Double Blind Clinical Trial of Valsartan for Attenuating Disease Evolution in Early Sarcomeric Hcm (Vanish)
This is a multicenter, double-blind placebo controlled phase II randomized clinical trial to assess the safety and efficacy of valsartan in attenuating disease evolution in early sarcomeric hypertrophic cardiomyopathy (HCM).Enrollment goal: 15 – 20 at Boston Children’s Hospital, 150 in total.
Contact: Jane Messere, Research Nurse (email@example.com) 857-218-3628
Trial Name: A Novel Non-invasive Approach to Rejection Surveillance in Pediatric Heart Transplant Recipients
We propose to develop an innovative, non-invasive approach based on blood biomarker and echocardiography for non-rejection screening rather than rejection in pediatric heart transplant recipients, using patients’ own baseline biomarker and echocardiographic data as internal control. This approach has the potential to reduce invasive biopsies and health care costs after heart transplantation.
Enrollment goal: We had enrolled about 120 patients with 100% enrollment rate (each approach patient/family agreed to participant in the study. Since summer 2013 we have been enrolling patients until we have the biopsy proven 9 rejection event collected echo-biomarker data (as study was powered).
Contact: Steve Colan, MD (firstname.lastname@example.org CHBOSTON.ORG) 617-355-6429