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Sorafenib for Hepatopulmonary Syndrome (SHPS)

This study is currently recruiting participants.
Verified July 2017 by University of Pennsylvania
Sponsor:
ClinicalTrials.gov Identifier:
NCT02021929
First Posted: December 27, 2013
Last Update Posted: July 6, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Collaborator:
National Heart, Lung, and Blood Institute (NHLBI)
Information provided by (Responsible Party):
University of Pennsylvania
  Purpose
The main purpose of this clinical trial is to determine the safety and effects of the study drug, sorafenib, in adults diagnosed with hepatopulmonary syndrome (HPS). The study will evaluate how well the drug is tolerated and its effect on the level of oxygen in the blood and the function of the lung vessels.

Condition Intervention Phase
Hepatopulmonary Syndrome Drug: Sorafenib Drug: Placebo Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Sorafenib in Patients With Hepatopulmonary Syndrome: A Double-Blind Randomized Clinical Trial

Resource links provided by NLM:


Further study details as provided by University of Pennsylvania:

Primary Outcome Measures:
  • Difference in changes in alveolar-arterial oxygen gradient between sorafenib and placebo groups [ Time Frame: 12 weeks ]

Secondary Outcome Measures:
  • Difference in changes in intrapulmonary shunting between sorafenib and placebo groups [ Time Frame: 12 weeks ]
  • Difference in changes in hematopoietic progenitor cells and other biomarker levels between sorafenib and placebo groups [ Time Frame: 8 weeks ]
  • Difference in changes in hematopoietic progenitor cells and other biomarker levels between sorafenib and placebo groups [ Time Frame: 12 weeks ]

Estimated Enrollment: 30
Study Start Date: March 2014
Estimated Study Completion Date: June 2018
Estimated Primary Completion Date: June 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Sorafenib
400 mg (2 capsules) taken by mouth once a day
Drug: Sorafenib

Sorafenib is a kinase inhibitor indicated for the treatment of:

  • Unresectable hepatocellular carcinoma
  • Advanced renal cell carcinoma
  • Locally recurrent or metastatic, progressive, differentiated thyroid carcinoma refractory to radioactive iodine treatment
Other Name: Nexavar
Placebo Comparator: Placebo
2 capsules taken by mouth once a day
Drug: Placebo

  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   21 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Diagnosis of HPS:

    1. AaPO2 ≥ 15 mm Hg (≥ 20 mm Hg for age > 64 yrs)
    2. Intrapulmonary shunting
    3. Absence of significant restriction (TLC < 70%) or obstruction (FEV1 < 80% & FEV1/FVC < 70%)
    4. Presence of cirrhosis/hepatic fibrosis and/or portal hypertension
  • Child-Pugh class A or B liver disease
  • Platelet count ≥ 30 ×10e9 per liter
  • Hemoglobin ≥ 8.5 g per deciliter
  • International normalized ratio ≤ 2.3
  • Albumin ≥ 2.8 g per deciliter
  • Total bilirubin ≤ 5 mg per deciliter
  • Alanine aminotransferase and aspartate aminotransferase ≤ 5 times the upper limit of the normal range
  • Serum creatinine ≤ 1.5 times the upper limit of the normal range and not receiving dialysis
  • Negative pregnancy test (for women of childbearing potential) at both screening and baseline visits. Post-menopausal women (defined as no menses for one year) and surgically sterilized women are not required to undergo a pregnancy test.
  • Subjects (men and women) of childbearing potential must agree to use medically acceptable contraception beginning at the signing of the Informed Consent Form until at least 14 days after the last dose of study drug.
  • Age ≥ 21 years
  • Ability to provide informed consent

Exclusion Criteria:

  • Recent chronic heavy alcohol consumption
  • Enrollment in a clinical trial or concurrent use of another investigational drug or device therapy (i.e., outside of study treatment) during, or within 28 days of screening visit
  • Current hepatic encephalopathy
  • Active infection
  • Diagnosis of portopulmonary hypertension
  • WHO Class IV functional status
  • Congenital long-QT syndrome
  • Subjects who have used strong CYP3A4 inducers (e.g., phenytoin, carbamazepine, phenobarbital, St. John's Wort [Hypericum perforatum], dexamethasone at a dose of greater than 16 mg daily, or rifampin [rifampicin], and/or rifabutin) within 28 days before randomization
  • Subjects who are currently taking Coumadin®(warfarin)
  • Active or clinically significant cardiac disease, including:

    1. Active coronary artery disease
    2. Unstable angina (anginal symptoms at rest), new-onset angina within 12 weeks before randomization, or myocardial infarction within 24 weeks before randomization
  • Liver or other solid organ transplant recipients
  • Expectation of liver transplant within four months of randomization
  • Hepatocellular carcinoma that does not meet all of the following criteria:

    1. Single lesion ≤ 3 cm documented by LIRADS criteria
    2. Complete response to ablative therapy (TACE, RFA, alcohol ablation) using the modified RECIST criteria one month after therapy with no more than two treatments
    3. No other lesions develop after initiation of HCC therapy
  • Uncontrolled hypertension (systolic pressure >140 mm Hg or diastolic pressure > 90 mm Hg on repeated measurement) despite optimal medical management.
  • Any hemorrhage/bleeding event of NCI-Common Toxicity Criteria for Adverse Effects v4.0 Grade 3 or higher within 4 weeks before randomization
  • Presence of a non-healing wound, non-healing ulcer, or bone fracture
  • Women who are pregnant or breast-feeding
  • Major surgery 28 days prior to randomization
  • Subjects with any previously untreated or concurrent cancer except cervical cancer in-situ, treated basal cell carcinoma, or superficial bladder tumor. Subjects surviving a cancer that was curatively treated and without evidence of disease for more than 3 years before randomization are allowed. All cancer treatments (chemotherapy, radiation therapy, surgery, immunotherapy, biologic therapy, or tumor embolization) must be completed at least 3 years prior to study entry (i.e., signature date of the informed consent form).
  • Inability to comply with the protocol and/or not willing or not available for follow-up assessments
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02021929


Contacts
Contact: Tiffany Sharkoski 215-746-4186 sharkosk@upenn.edu

Locations
United States, Arizona
Mayo Clinic Arizona Recruiting
Phoenix, Arizona, United States, 85054
Contact: Kelly Clark    480-342-2536    clark.kelly2@mayo.edu   
Contact: Barbara Knight    480-342-2545    knight.barbara@mayo.edu   
Principal Investigator: Hugo Vargas, MD         
United States, Illinois
Northwestern University Feinberg School of Medicine Recruiting
Chicago, Illinois, United States, 60611
Contact: Patrice Al-Saden, RN    312-694-0232    palsaden@northwestern.edu   
Contact: Ojoma Agbo    312-694-0253    ojoma.agbo@northwestern.edu   
Principal Investigator: Josh Levitsky, MD         
United States, Minnesota
Mayo Clinic - Rochester Recruiting
Rochester, Minnesota, United States, 55905
Contact: Britten Block    507-293-4773    block.britten@mayo.edu   
Principal Investigator: Michael Krowka, MD         
United States, New York
Columbia University-NewYork-Presbyterian Hospital Recruiting
New York, New York, United States, 10032
Contact: Samina Munir    212-305-3839    sm3417@cumc.columbia.edu   
Contact: Moury Minhaz    212-305-3839    mm3597@cumc.columbia.edu   
Principal Investigator: Alyson Fox, MD         
United States, Pennsylvania
University of Pennsylvania - Perelman Center Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Tiffany Sharkoski, 15    215-746-4186    sharkosk@upenn.edu   
Principal Investigator: Steven M Kawut, MD, MS         
Sub-Investigator: David Goldberg, MD, MSCE         
United States, South Carolina
Medical University of South Carolina Recruiting
Charleston, South Carolina, United States, 29424
Contact: Frank Beylotte    843-876-4273    beylott@musc.edu   
Principal Investigator: David Koch, MD         
United States, Texas
University of Texas Health Science Center at Houston Medical School Recruiting
Houston, Texas, United States, 77030
Contact: Jordan Varing    713-500-6676    Jordan.P.Varing@uth.tmc.edu   
Principal Investigator: Moises Nevah, MD         
Sponsors and Collaborators
University of Pennsylvania
National Heart, Lung, and Blood Institute (NHLBI)
Investigators
Principal Investigator: Steven M Kawut, MD, MS University of Pennsylvania
  More Information

Responsible Party: University of Pennsylvania
ClinicalTrials.gov Identifier: NCT02021929     History of Changes
Other Study ID Numbers: 819185
UM1HL116886 ( U.S. NIH Grant/Contract )
First Submitted: December 20, 2013
First Posted: December 27, 2013
Last Update Posted: July 6, 2017
Last Verified: July 2017

Keywords provided by University of Pennsylvania:
Randomized Controlled Trial
Clinical Trial
sorafenib
Hepatopulmonary Syndrome

Additional relevant MeSH terms:
Syndrome
Hepatopulmonary Syndrome
Disease
Pathologic Processes
Liver Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Sorafenib
Niacinamide
Antineoplastic Agents
Protein Kinase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Vitamin B Complex
Vitamins
Micronutrients
Growth Substances
Physiological Effects of Drugs