A Phase 2 interventional study of Placebo and Sorafenib in Clinically Significant Portal Hypertension, sponsored by Yale University. Completed at 6 sites in United States. Open to participants aged 20 Years to 75 Years. Per ClinicalTrials.gov, last updated 2020-12-29.
Sponsored by Yale University · Phase 2, Interventional, and Treatment
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.
This is a pilot proof-of-concept study that investigates the effect of sorafenib on portal pressure, as determined by the hepatic venous pressure gradient (HVPG), in patients with liver cirrhosis, portal hypertension and unresectable hepatocellular carcinoma (HCC) that has successfully responded to radiofrequency ablation and/or transarterial chemoembolization, and have obtained a complete response.
The primary end-point of the study is the change in HVPG observed from baseline to three months after starting treatment with sorafenib. Secondary end-point is safety of sorafenib.
The trial is structured as a randomized double blind placebo controlled study. After a three-month period of therapy with sorafenib or placebo (double-blind phase), patients will be given open-label sorafenib for an additional 3-month period (open-label phase). A total of 44 patients will be randomized (in the initial phase) on a 1:1 ratio to sorafenib or placebo. Patients will be followed monthly and HCC follow-up will be according to standards of care. The study will be sponsored by Onyx, who will also provide the treatment medication (sorafenib and placebo)
332 studies on the registry are indexed under Hypertension, Portal; 106 are open to participants now.
This study's enrollment of 10 is below the median of 60 across 206 interventional studies indexed under Hypertension, Portal.
Browse Hypertension, Portal studies →Yale University is the lead sponsor of 1,724 studies on the registry; 298 are open to participants now.
Of its 210 completed or terminated interventional studies of FDA-regulated products, 126 (60%) have results posted.
Counted across the registry records on this site, refreshed daily.
Subjects randomized to placebo will take two tablets of placebo by mouth twice daily.
Drug: Placebo
Subjects randomized to Sorafenib will take Sorafenib 400 mg by mouth twice daily.
Drug: Sorafenib
Placebo Comparator: Placebo
Sorafenib, 400 mg twice daily
Patients With Change in HVPG From Baseline
Number of participants with a decrease in HPVG that was \> 10% of baseline
Time frame: Three Months
| Milestone | Placebo | Sorafenib |
|---|---|---|
| Started | 5 | 5 |
| Completed | 4 | 5 |
| Not completed | 1 | 0 |
| Withdrew: Protocol violation | 1 | 0 |
Number of participants with a decrease in HPVG that was \> 10% of baseline
| participants | Placebo | Sorafenib |
|---|---|---|
| Patients With Change in HVPG From Baseline | 1 | 1 |
Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Placebo | — | 1/5 (20%) | 5/5 (100%) |
| Sorafenib | — | 0/5 (0%) | 5/5 (100%) |
| Event | Placebo | Sorafenib |
|---|---|---|
| FeverGeneral disorders | 1/5 | 0/5 |
| Event | Placebo | Sorafenib |
|---|---|---|
| gastrointestinal disordersGastrointestinal disorders | 5/5 | 5/5 |
| Skin and subcutaneous tissue disordersSkin and subcutaneous tissue disorders | 4/5 | 5/5 |
| General DisordersGeneral disorders | 4/5 | 4/5 |
| Nervous System DisordersNervous system disorders | 3/5 | 4/5 |
| Metabolism and nutrition disordersMetabolism and nutrition disorders | 3/5 | 2/5 |
| Muskoloskeletal and connective tissueMusculoskeletal and connective tissue disorders | 2/5 | 1/5 |
| Psychiatric disordersPsychiatric disorders | 2/5 | 1/5 |
| Respitory, thoracic and medistinalRespiratory, thoracic and mediastinal disorders | 2/5 | 2/5 |
| Vascular disordersVascular disorders | 2/5 | 0/5 |
| Alanine aminostranferaseHepatobiliary disorders | 1/5 | 0/5 |
| Age, Continuous(years) | Placebo | Sorafenib | Total |
|---|---|---|---|
| Mean | 55.98 ± 4.7 | 58.78 ± 4.25 | 57.38 ± 4.48 |
| Sex: Female, Male(Participants) | Placebo | Sorafenib | Total |
|---|---|---|---|
| Female | 0 | 0 | 0 |
| Male | 4 | 5 | 9 |
| Region of Enrollment(participants) | Placebo | Sorafenib | Total |
|---|---|---|---|
| United States | 4 | 5 | 9 |
This study is completed, as verified in Dec 2020. You cannot join it, but the record below documents what was studied.
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