SCCRIP Study: Longitudinal Monitoring of Adult Organ Function in Sickle Cell Disease
Study Objective
SCCRIP is designed to systematically track adult organ damage in patients with sickle cell disease. Prior research has focused on pediatric treatment, but the causes of organ function decline observed in adulthood have not been fully elucidated.
Population and Design
The study commenced on April 15, 2014, and is currently enrolling participants across the United States. Data are collected during routine clinic visits (in‑person or remote) from SCD patients. As an observational study, there is no therapeutic intervention; clinical metrics, imaging studies, and lifestyle information are recorded longitudinally.
Hydroxyurea Pharmacogenomic Sub‑Study
Participants may enroll in an optional sub‑study to undergo hydroxyurea pharmacokinetic assessments. Serial blood samples are taken at predefined intervals to analyze drug concentrations and metabolic pathways, aiming to clarify how individual genetic variants influence treatment response.
Anticipated Impact
Long‑term follow‑up data will enable the development of strategies to reduce disease burden in adult SCD patients. If biomarkers predictive of hydroxyurea responsiveness are identified, personalized therapy could be implemented, substantially improving patients’ quality of life.
This study elucidates the mechanisms of adult organ injury in sickle cell disease, providing essential data assets for building new‑drug pipelines. The expansion of personalized therapeutics leveraging clinical and pharmacokinetic data will create valuable opportunities for those preparing to enter the field.
Source: ClinicalTrials.gov (api_ct)