Possible Treatment for AL Amyloidosis Falters in Phase 3 Trial

According to a story from home.cableone.net, the biopharmaceutical company Takeda Pharmaceutical Company Ltd. recently announced the latest updates to their phase 3 clinical trial which is testing the company’s drug ixazomib (marketed at NINLARO TM) as a treatment for AL amyloidosis. Unfortunately, the news from the trial is not good; the treatment, which was being used in combination with dexamethasone, was unable to display significant advantages compared to current treatments.

About Amyloidosis

Amyloidosis is a group of diseases which are characterized by the build up amyloid fibrils in body tissue. Amyloid fibrils are a type of abnormal protein. As there are several different types of amyloidosis, the cause can vary. Some forms are acquired while others are linked to genetic factors. The most common form is amyloid light-chain (AL) amyloidosis. Symptoms of the disease include swelling, heart failure, irregular heartbeat, shortness of breath, fatigue, weight loss, easy bruising and bleeding, stroke, lung problems, enlarged liver, and changes in skin color. Treatment approaches for amyloidosis includes chemotherapy and stem cell transplant; other options vary depending on the type of amyloidosis. Prognosis varies depending on the type; AL amyloidosis has a median survival of one or two years without treatment. To learn more about amyloidosis, click here.

About The Phase 3 Clinical Trial

As the drug combination has failed to reach its first primary endpoint, officials at Takeda have made the decision to discontinue the clinical trial. The trial was one of the largest clinical studies ever conducted that was focused on AL amyloidosis. The trial compared ixazomib plus dexamethasone against a physician’s choice of chemotherapy in patients with AL amyloidosis that was refractory or relapsed and was systemic in nature. Endpoints that were evaluated in this trial included overall mortality, two year vital organ function/deterioration, and hematologic response.

Ixazomib is currently approved as a treatment for the blood cancer multiple myeloma. While the drug failed to make an impact for AL amyloidosis, the therapy is continuing to be tested in a number of clinical trials for various indications in multiple myeloma. 


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