Experimental Drug Improves Colorectal Cancer Survival Time

Colorectal cancer patients in a phase III clinical trial who took the oral drug regorafenib lived longer than those who got the best supportive care along with a placebo.

In fact, when the independent Data Monitoring Committee analyzed the progress of the trial, they recommended it be unblinded so that patients who had been randomly assigned to supportive care and a placebo pill could receive the new medicine.

The survival benefit was statistically significant, although how much time was gained isn’t yet available. Overall survival benefits are expected to be reported at an upcoming scientific meeting.

Patients enrolled in the randomized Phase III CORRECT trial had already had their cancer get worse on all standard approved treatments.

 What’s Regorafenib?

Regorafenib is an oral drug called a multi-targeted kinase inhibitor. A kinase inhibitor targets certain key proteins that are essential for the survival of a cancer cell. By specificallyblocking those proteins, regorafenib may stop cancer growth. Also known as BAY 73-4506, it is manufactured by Bayer Healthcare, who sponsored the CORRECT clinical trial.

CORRECT randomly assigned 760 patients with metastatic colorectal cancer whose disease had progressed on all approved standard therapies to either regorafenib plus the best supportive care or a placebo and supportive care.  The study was blinded so that neither the patients nor their doctors knew if they were getting the drug or the placebo.

The trial was conducted at many sites in North America, Europe, China, Japan and Australia.

Why was the trial stopped early?

In randomized clinical trials, no one can see the results as they emerge except for a data monitoring committee (DMC), who with its statisticians, periodically review the progress of the trial.  The DMC also tracks serious side effects.

Keeping clinical trial information secret from everyone involved in randomly assigning patients to trial arms and providing treatment reduces bias and ensures that results will be fair and accurate.

In the CORRECT trial, a planned review by the DMC discovered that the patients on regorafenib were living longer than patients getting a placebo.  The result was statistically significant and met the primary objective for overall survival set in the trial plan.

The DMC recommended the trial be unblinded so that doctors and patients knew whether they were getting regorafenib or a placebo. Patients on regorafenib will continue to get that drug, and patients on placebo will be offered a switch to regorafenib.

What’s next for regorafenib?

Bayer Healthcare will discuss

• details of the trial results at a scientific meeting.  A source tells Fight Colorectal Cancer that might be in January at the Gastrointestinal Cancers Symposium.
• the next steps for approving regorafenib for the treatment of colorectal cancer with European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA).

Clinical trials of regorafenib in combination with FOLFIRI and FOLFOX for colorectal cancer are underway, along with trials for other cancer types.

Can I get regorafenib now?  Is it on the market?

No, the drug remains experimental for now.  It has not yet been approved by the FDA.

If you have metastatic colorectal cancer that has progressed on your first treatment, you might want to consider a second-line trial of regorafenib in combination with FOLFIRI chemotherapy.  This Phase II trial is specifically for patients who have KRAS or BRAF tumor mutations. The trial is not for refractory patients, however, who have already been treated with FOLFIRI.

The trial is active and recruiting new patients in a number of sites in the United States.  It is being led nationally by Richard Goldberg, M.D., who is a member of the Fight Colorectal Cancer Medical Advisory Board.

For more information about this clinical trial, contact the Fight Colorectal Cancer Clinical Trials Matching Service online or call them at 866-278-0392.

Disclosure: Fight Colorectal Cancer has accepted funding for projects and educational programs from Bayer Healthcare in the form of unrestricted educational grants. Fight Colorectal Cancer has ultimate authority over website content.