Amyloidosis

Clinical Trials

Below is a list of Amyloidosis clinical trials from the clinical trials database at Mayo Clinic.

This list includes only trials about which Mayo researchers choose to publish information. Mayo Clinic may be conducting other trials which are not in this database. Mayo's clinical trials include experimental treatments, often unavailable elsewhere, which frequently lead to improved patient care for people worldwide. Patients should ask their doctor at Mayo about clinical trials appropriate for their situation.

H23750, The Effect of Diflunisal (Dolobid) (IND 68092) On Familial Amyloidosis: A randomized, double blind, placebo controlled, international multi-center trial of Diflunisal on neurologic disease progression in 200 familial amyloidal patients
Patients are being asked to participate in this research study because they have been diagnosed with Familial Amyloid Polyneuropathy (FAP) and have symptoms of nerve and/or heart damage. FAP is an inherited disease that attacks the nerves and heart, generally causing death 7-15 years after diagnosis. At present, the only treatment for FAP is liver transplantation.

Medicines are needed to treat this disease. A common anti-inflammatory drug called diflunisal (Dolobid) that is similar to Motrin has been shown to prevent amyloid formation in a test tube and in the blood of normal human volunteers. The research staff would like to find out if diflunisal stops amyloid production in patients with FAP. All patients participating in the study will be encouraged to seek liver transplantation. Patients pursuing liver transplantation will be welcomed into the study provided the operation is unlikely to be performed within 12 months of entering the study based on the status of the liver transplant waiting list. Participation in this study will not affect the patient's current status on a liver transplant waiting list, nor future listing for transplantation.

This research study is designed to establish whether diflunisal, an anti-inflammatory drug that has been used for over 20 years in patients with arthritis, can stop nerve and heart damage resulting from amyloid produced by patients with FAP. Researchers know that diflunisal prevents formation of amyloid in the test tube. This study will determine if diflunisal can block amyloid production in FAP patients.
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MC0685, "A Phase II Study of Lenalidomide (Revlimid), Cyclophosphamide (Cytoxan) and Dexamethasone (Decadron) in Patients with Primary Systemic Amyloidosis"
This study is being done to find out what good and bad effects the lenalidomide (Revlimid), dexamethasone and cyclophosphamide drugs have on a patient and their amyloidosis. Lenalidomide is approved by the U.S. Food and Drug Administration (FDA) for the treatment of specific types of myelodysplastic syndrome (MDS) and in combination with dexamethasone for patients with multiple myeloma (MM) who have received at least one prior therapy. MDS and MM are cancers of the blood. It is currently being tested in a variety of cancer conditions. In this case it is considered experimental.
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