logo Hematology/Oncology Research Studies: Open for Enrollment Date: 12/12/18

Category: SICKLE CELL DISEASE

Randomized, Double-Blind, Placebo-Controlled Multi-Center Study to Assess the Safety, Tolerability, and Efficacy of Riocigut in Patients with Sickle Cell Diseases

NCT#/Phase: NCT02633397
Phase 2b
IRB#: 2017-064
Description: The purpose of this research study is to look at what doses of riociguat are safe in sickle cell disease (SCD) and to find out if riociguat can prevent or reduce clinical complications of SCD. Subjects in this randomized study will take either riociguat pills or placebo pills for 12 weeks and then have a follow-up visit 30 days after stopping the drug. The dose is adjusted every 2 weeks based on blood pressure (SBP) and well-being assessed at that visit. Physical examinations, vital signs, blood tests and questionnaires will be performed at 2 week intervals during the double blinded study treatment. Echocardiogram, urine testing, six-minute walk distance and questionnaires will be assessed at the beginning and end of the treatment phase.
Inclusion: Individuals must be 18 years or older with HbSS, HbSC, HbSbeta-thalassemia, HbSD, HbSO-Arab documented by hemoglobin electrophoresis or HPLC fractionation. At least one of the following findings: a. Systolic blood pressure >= 130 mm Hg on at least two occasions at least 1 day apart (one of these may be by history), b. Macroalbuminuria as manifested by urine albumin to creatinine ratio > 300 mg/g, c. Tricuspid regurgitant velocity (TRV) > 2.9 m/sec measured by echocardiography d. NT-proBNP level >= 160 pg/mL e. Urinalysis protein 1 + or higher
Enrollment Status: Open for enrollment
Sponsor: University of Pittsburgh
Principal Investigator(s): Ward Hagar, MD
Contact(s): Mariam Hameed (510-428-3885 x4151)

Study Link: https://clinicaltrials.gov/ct2/show/NCT02633397

UCSF Benioff Children's Hospital Oakland • 747 52nd Street, Oakland, California 94609 &bull Phone: (510) 428-3000