A Phase 3 Clinical Study for People With Relapsed or Refractory AL Amyloidosis

Deciding whether to join a clinical study is a big decision. You will want to gather as much information as possible before you make your decision. Below are answers to some key questions designed to give you a better understanding of the study and its goals. You can also visit ClinicalTrials.gov for more information.

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What is the official title of the Tourmaline AL1 study?

The official title of this study is:

A Phase 3, Randomized, Controlled, Open-Label, Multicenter, Safety and Efficacy Study of Dexamethasone Plus MLN9708 or Physician's Choice of Treatment Administered to Patients With Relapsed or Refractory Systemic Light Chain (AL) Amyloidosis

It is also referred to as Protocol C16011.

What are the goals of the study?

  • To find out if AL amyloidosis patients treated with MLN9708 plus dexamethasone respond better than patients treated with other chemotherapies that physicians use
  • To determine if MLN9708 plus dexamethasone can change the chance that amyloidosis will cause the heart and/or kidneys to begin to work poorly, leading to hospitalization or death

What are the possible treatment groups that I could be assigned to in this study?

There are 2 treatment groups in this study. If you are eligible and you choose to participate, you will be selected at random to receive either:

MLN9708 + Dexamethasone OR Your physician will select one of the following treatments: Dexamethasone, Dexamethasone + melphalan, Dexamethasone + cyclophosphamide, Dexamethasone + thalidomide, Dexamethasone + lenalidomide

What will researchers be measuring in this study?

Every study has a primary endpoint, or the main result that is measured at the end of the study.

In this study, there are 2 primary endpoints that researchers will be looking at:

  • Overall response rate (ORR) including complete response, very good partial response, and partial response
  • Rate of hospitalization for heart failure or progression to end-stage renal disease or death at 2 years

In addition to the primary endpoints, there are additional endpoints. These are results that are measured at the end of a study, but do not determine the overall success of the study. Some additional endpoints include:

  • Overall survival (OS)
  • Progression-free survival (PFS)
  • Number of patients with cardiac and/or kidney response
  • Safety

How many patients will participate in this study?

Approximately 250 patients will participate in this study worldwide.

How long will the study last?

The study is estimated to be completed in August 2018.

What should I know about MLN9708?

MLN9708 is an investigational targeted anti-cancer drug given orally. MLN9708 is being studied in multiple myeloma, various hematologic malignancies, and solid tumors.

To learn more about this clinical study, visit ClinicalTrials.gov
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