A Multi-Center Study of Riociguat in Patients With Sickle Cell Diseases



Status:Recruiting
Conditions:Anemia
Therapuetic Areas:Hematology
Healthy:No
Age Range:18 - Any
Updated:2/2/2019
Start Date:April 11, 2017
End Date:December 2019
Contact:Gregory Kato, MD
Email:katogj@upmc.edu
Phone:412-648-3017

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A Phase II Randomized, Double-Blind, Placebo-Controlled Multi-Center Study to Assess the Safety, Tolerability, and Efficacy of Riociguat in Patients With Sickle Cell Diseases

The proposed study is a Phase 2 multi-center, randomized, double-blind, placebo-controlled,
parallel groups study aimed to evaluate the safety, tolerability and the efficacy of
riociguat compared with placebo in patients with sickle cell disease (SCD).

This randomized study involves 12 weeks of treatment with riociguat pills or placebo pills,
and a follow-up period of 30 days after treatment. The dose is adjusted every 2 weeks based
on systolic 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 Criteria:

- Age ≥ 18 years

- Sickling disorder (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.

- Females of reproductive potential (FRP) must have a negative, pre-treatment pregnancy
test. Post-menopausal women (defined as no menses for at least 1 year or post-surgical
from bilateral oophorectomy) are not required to undergo a pregnancy test.

- Females of reproductive potential must agree to use reliable contraception when
sexually active. Adequate contraception is defined as any combination of at least 2
effective methods of birth control, of which at least one is a physical barrier (e.g.
condoms with hormonal contraception or implants or combined oral contraceptives,
certain intrauterine devices). Adequate contraception is required beginning at the
signing of the informed consent form until one month after the last dose of riociguat.

- Patients must be willing to provide a blood sample for DNA analysis.

Exclusion Criteria:

- Pregnant or breast feeding women

- Patients with severe hepatic impairment defined as Child Pugh C

- End stage renal disease requiring dialysis

- Patients with eGFR <30 mL/min/1.73m, where GFR is estimated based on CKD-epi equation

- Patients on phosphodiesterase type 5 inhibitors (PDE-5) (such as sildenafil,
tadalafil, vardenafil) and nonspecific PDE inhibitors (such as dipyridamole or
theophylline) or nitrates

- Patients on strong cytochrome P450 (CYP) and P-glycoprotein 1(P-gp)/BCRP inhibitors
such as systemic azole antimycotics (eg: ketoconazole, itraconazole), or HIV protease
inhibitors (such as ritonavir)

- Patients on St. John's Wort

- If patients are taking antihypertensive drugs, hydroxyurea, or L-glutamine prior to
enrollment, they are excluded until the dose level is stable for at least three months

- Systolic blood pressure <95 mm Hg at Screening Visit 1 or 2 or Week 0 before
randomization

- Current enrollment in an investigational new drug trial. Patients are eligible for
enrollment 30 days after the last dose of an investigational drug has been received

- Evidence of qualitative urine drug test at screening for cocaine, phencyclidine (PCP),
heroin, or amphetamines within three months prior to enrollment

- Patients who have recently (last six months) experienced serious bleeding from the
lung or have undergone a bronchial arterial embolization procedure.

- Pulmonary hypertension associated with Idiopathic Interstitial Pneumonias

- Medical disorder, condition, or history that in the investigator's judgment would
impair the patient's ability to participate or complete this study or render the
patient to be inappropriate for enrollment
We found this trial at
11
sites
Durham, North Carolina 27710
(919) 684-8111
Principal Investigator: Marilyn Telen, MD
Phone: 919-684-5378
Duke University Younger than most other prestigious U.S. research universities, Duke University consistently ranks among...
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1648 Pierce Dr NE
Atlanta, Georgia 30322
(404) 727-5640
Principal Investigator: Claudia Morris, MD
Phone: 404-727-5500
Emory University School of Medicine Emory University School of Medicine has 2,359 full- and part-time...
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Atlanta, GA
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3400 N Charles St
Baltimore, Maryland 21205
410-516-8000
Principal Investigator: Sophie Lanzkron, MD, MHS
Phone: 410-502-8642
Johns Hopkins University The Johns Hopkins University opened in 1876, with the inauguration of its...
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Baltimore, MD
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1 Boston Medical Center Place
Boston, Massachusetts 02118
617.638.8000
Principal Investigator: Elizabeth Klings, MD
Phone: 617-638-8679
Boston University Medical Center Boston Medical Center is an extraordinary community of health care providers...
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1825 Eastchester Road
Bronx, New York 10461
Principal Investigator: Caterina Minniti, MD
Phone: 718-920-4137
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Bronx, NY
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450 West Drive
Chapel Hill, North Carolina 27599
Principal Investigator: James Ford, MD
Phone: 919-966-2531
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Chapel Hill, NC
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171 Ashley Avenue
Charleston, South Carolina 29425
843-792-1414
Principal Investigator: Temia Martin, MD
Phone: 843-876-8614
Medical University of South Carolina The Medical University of South Carolina (MUSC) has grown from...
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Charleston, SC
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2035 W Taylor St
Chicago, Illinois
(312) 996-4350
Principal Investigator: Victor R Gordeuk, MD
Phone: 312-996-5680
University of Illinois at Chicago A major research university in the heart of one of...
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Chicago, IL
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281 W. Lane Ave
Columbus, Ohio 43210
(614) 292-6446
Principal Investigator: Payal Desai, MD
Phone: 614-293-2887
Ohio State University The Ohio State University’s main Columbus campus is one of America’s largest...
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Columbus, OH
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Pittsburgh, Pennsylvania 15233
Principal Investigator: Laura DeCastro, MD
Phone: 412-623-7062
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1250 E. Marshall St.
Richmond, Virginia 23298
(804) 828-9000
Principal Investigator: Wally Smith, M.D.
Phone: 804-828-6938
Virginia Commonwealth University Medical Center The Virginia Commonwealth University Health System is an urban, comprehensive...
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Richmond, VA
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