The Data Coordinating Center (DCC)

The Data Coordinating Center (DCC) at Rho, Inc performs all the statistical and data management for the Treatment of Pulmonary Hypertension and Sickle Cell Disease with Sildenafil Treatment (walk-PHaSST) study.

• Operational Support activities include:
  • Facilitating communication between study sites, study collaborators, and the NHLBI
  • Supporting the conduct, logistics, and compliance of studies
  • Overseeing the collection and maintenance of study documentation, including the maintenance of a project website
  • Developing study documents, such as the protocol and Manual of Operations
  • Preparing reports for the NHLBI, the Protocol Review Committee (PRC), and the Data & Safety Monitoring Board (DSMB)
• Statistical Analysis activities include:
  • Participating as collaborative scientists in all the study's stages, from concept development to study design to manuscript development
  • Providing routine analyses of study progress
  • Conducting interim analyses in support of the DSMB's activities
  • Conducting final analyses and preparing final study reports
• Data Management activities include:
  • Developing Case Report Forms (CRFs) for the accurate and efficient collection of study data
  • Developing, maintaining, and validating study databases, including the Electronic Data Capture (EDC) system for the various studies.
• Regulatory Support activities include:
  • Ensuring that all regulatory documentation is properly collected, reviewed, and maintained
  • Assisting in the development and submission of Investigational New Drug applications and other FDA reports and applications
  • Monitoring sites to ensure compliance with the protocol and with Good Clinical Practice (GCP), NIH, and FDA requirements
  • Facilitating safety communication between sites and the NIH

walk-PHaSST Overview

walk-PHaSSST is a double-blind, placebo-controlled phase II/III trial. The primary efficacy hypothesis is that 16 weeks of sildenafil therapy provides greater improvement in six-minute walk (6MW) distance than does placebo in patients with sickle cell disease (SCD) and pulmonary hypertension. The primary hypothesis test will be based on a test that the average change differs between the two treatment groups, with baseline 6MW distance and TRV stratum used as covariates.

Up to 1000 patients will be screened based on medical history, physical examination, laboratory testing, transthoracic Doppler-echocardiography, and 6MW test. All consented subjects in this cohort will provide plasma and serum for a biomarker and DNA repository and will be followed prospectively in the Observational Follow-up Study. Subjects found to have a screening TRV of > 2.7 m/s will be invited to participate in the Main Interventional Trial. Upon repeat echocardiogram at baseline, subjects with TRV > 2.7 m/s who meet all inclusion/exclusion criteria will be enrolled in the 16 week, randomized, double-blind, placebo-controlled trial of sildenafil versus placebo. The study will randomize 132 subjects in the double-blind phase of the MIT. Subjects will be stratified and randomized based on TRVs. The primary endpoint is the change in 6MW distance across this study phase. Subjects who complete the double-blind phase will be eligible to participate in the Open-label Follow-up Phase and will be treated with sildenafil for up to 1 additional year.

Please contact Nancy Yovetich for questions or comments.
Last modified: 8October2007.