The Effect of Sorafenib on Portal Pressure

NCT01714609 | Completed Phase 2 Results DMC

• 1 other identifier: 1002006266
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 6

Brief Summary

Sorafenib is approved by the US FDA for the treatment of unresectable (can not operate) liver cancer and for renal cell carcinoma. Sorafenib is a drug that inhibits the growth of cancer cells and prevents the formation of new blood vessels that would otherwise help the cancer spread. Studies in experimental animals have shown that sorafenib may also lower portal vein pressure (the pressure of the blood passing from the intestine through the liver.) This study seeks to determine if sorafenib lowers the blood pressure in liver blood vessels (portal vein pressure) in patients with cirrhosis who have high portal vein pressure. The study will also obtain information whether sorafenib is safe in this patient population. Half of the patients will be given sorafenib and half will be given a placebo (a pill without any medicine in it.) This allows a comparison of the reactions of people who take sorafenib to those who do not.

Conditions

Clinically Significant Portal Hypertension

Keywords

portal hypertension; sorafenib

Outcome Measures

Primary Outcomes (1)

• Patients With Change in HVPG From Baseline

  Number of participants with a decrease in HPVG that was \> 10% of baseline

  Three Months

Study Arms (2)

Placebo

PLACEBO COMPARATOR

Subjects randomized to placebo will take two tablets of placebo by mouth twice daily.

Drug: Placebo

Sorafenib

EXPERIMENTAL

Subjects randomized to Sorafenib will take Sorafenib 400 mg by mouth twice daily.

Drug: Sorafenib

Interventions

Placebo DRUG

Placebo Comparator: Placebo

Placebo

Sorafenib DRUG

Sorafenib, 400 mg twice daily

Sorafenib

Eligibility Criteria

• Age: 20 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

* Age 20-75 years * Cirrhosis diagnosed by liver biopsy or by imaging studies showing a nodular liver, splenomegaly and/or collaterals * HCC proven histologically or diagnosed following the AASLD criteria if biopsy not feasible or refused by the patient * HCC must be unresectable and within UCSF criteria (single tumor ≤ 6.5 cm diameter, or, if multiple lesions, maximum diameter of the largest lesion ≤ 4.5 and total tumor diameter ≤ 8 cm (23)) * CPT score \<9 (that is all Child A and Child B with a score of 7 or 8) * Complete response to treatment with RFA (including that performed laparoscopically) or TACE or a combination of the above as defined by radiologic criteria (hepatoma protocol MRI or CT scan performed 4-6 weeks after the procedure). * No more than two ablative procedures prior to enrollment * Presence of portal hypertension, as defined by HVPG of \>5 mmHg * EGD for variceal screening performed within 6 months of entry into the study unless the patient is already on a stable dose of a non selective beta-blocker (adjusted to obtain a heart rate of 55-60 bpm) or treated with variceal band ligation. * Women of childbearing potential must have a negative serum pregnancy test performed within 7 days prior to the start of treatment * Women of childbearing potential and men must agree to use adequate contraception (barrier method of birth control) prior to study entry and for the duration of study participation. Men should use adequate birth control for at least three months after the last administration of sorafenib. * Signed informed consent

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• Yale University: lead
• Onyx Therapeutics, Inc.: collaborator

Study Sites (6)

Yale Cancer Center

New Haven, Connecticut, 06520, United States

Location

VA Medical Center West Haven

West Haven, Connecticut, 06516, United States

Location

Brigham & Womens

Boston, Massachusetts, 02115, United States

Location

New York University Langone Medical Center

New York, New York, 10016, United States

Location

University of Pennsylvania School of Medicine

Philadelphia, Pennsylvania, 19104, United States

Location

The University of Texas Health Science Center at Houston

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Hypertension, Portal

Interventions

Sorafenib

Condition Hierarchy (Ancestors)

Liver Diseases; Digestive System Diseases

Intervention Hierarchy (Ancestors)

Phenylurea Compounds; Urea; Amides; Organic Chemicals; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Niacinamide; Nicotinic Acids; Acids, Heterocyclic; Heterocyclic Compounds; Pyridines; Heterocyclic Compounds, 1-Ring

Results Point of Contact

• Title: Dr. Garcia-Tsao
• Organization: Yale University

guadalupe.garcia-tsao@yale.edu

(203) 737-6063

Study Officials

• Guadalupe Garcia-Tsao, MD

  Yale University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 23, 2012
• First Posted: October 26, 2012
• Study Start: August 1, 2011
• Primary Completion: September 1, 2013
• Study Completion: September 1, 2014
• Last Updated: December 29, 2020
• Results First Posted: December 9, 2015

Record last verified: 2020-12

Locations

US (6)