IRB Number:

1691-05

Trial Status:

Open for Enrollment

Phase: III

Why is this study being done?

This study is being done to determine whether the response rate is different for patients who receive melphalan in one dose followed by stem cell vs. the same total dose of melphalan given over ten doses with six weeks between each dose.

Who is Eligible to Participate in the Study?

-Age 18 or older
-Diagnosed with Primary Systemic Amyloidosis (AL)

*More specific, detailed eligibility and/ or exclusion criteria are associated with this trial.

What is Involved With this Study?

This is a phase III study where patients will receive one of two treatments. One treatment group (Group A) will get melphalan given on day one and dexamethasone on days 1-4
and 22-25. This is considered 1 cycle of treatment and will be repeated every six weeks for a total of ten cycles. The other treatment group (Group B) will have peripheral blood
stem cells collected and stored before they receive the study drug (melphalan). These are called autologous stem cells (special blood cells obtained from the patient which are able to regenerate the blood tissue). Patients will get melphalan over a two-day period (day -2 and day -1) of time followed by stem cells (day 0) in order to assist recovery of their blood counts. Most patients are hospitalized for 3-4 weeks for treatment of side effects from this treatment. Patients will need to return to the Mayo Clinic on a regular basis for assessment of any benefit or side effects from treatment.

Which treatment the patient will receive can be assigned randomly, as in the flip of a coin. There is currently not enough information to say that either of these treatments is better than the other, which is why the researchers are doing this study. If the patient's treatment assignment is made through randomization, the patient would not have a say in which treatment they would receive but would be assigned one randomly. If the patient has a strong preference for one treatment over another being studied in this trial and thus refuses to be randomized, the patient will still be able to participate in this trial and choose the treatment they prefer.

Prior to and during the collection of the patient's stem cells, they will receive filgrastim (G-CSF) once each day by injection under the skin to increase the production of stem cells in the blood. The exact number of injections may vary depending on how fast the patient's stem cells increase in the blood. Typically the patient will need five days of injections before the collection and the collection may take 1-5 days to complete.

To obtain the stem cells, a needle will be inserted into a vein in each arm and/or a central venous catheter will be used to perform this procedure (apheresis). The patient's primary doctor will make arrangements for the placement of the central venous catheter or needle prior to their scheduled apheresis treatment. Blood will be taken from one of the patient's veins and will be run through a special device which will separate the blood into specific
components. Red blood cells and all other components that are not being collected will be returned to the patient through the vein in their other arm or a different line in the central venous catheter.

Each stem cell collection (apheresis) procedure will take from one to five hours per day. The number of days needed to collect the needed number of stem cells will depend on
how many cells are collected during each apheresis procedure.

Before entering the study, patients will see their doctor for tests to make sure they can safely participate. During this first visit, patients will have a medical history and tests to determine they are able to take part in this study. If they take part, the patient will be asked questions about their general health, a full medical history, as well as medications they are currently taking.

Once enrolling in the study, patients will have additional blood and urine testing as described below. Some of these tests are done to ensure the patient's safety while others are done to see the effects of the drug.

Below are the tests and procedures that will be done at each visit during this study:

Screening: (within 30 days of start of the study unless otherwise specified)
- Medical history
- Complete physical exam, including vital signs (blood pressure, heart rate, respiratory rate and body temperature) and weight
- Lab tests (24-hour urine collections, complete blood count, blood chemistry test, and urinalysis) to check the status of the patient's amyloidosis
- Bone Marrow Aspirate
- In addition, the lab tests will include testing for human immunodeficiency virus (HIV) and hepatitis
- Complete skeletal survey
- Chest x-ray
- Electrocardiogram (EKG)
- Echocardiogram (within six weeks of start of the study)
- Fat aspirate
- Blood pregnancy test if patient is a woman, still have their uterus (womb) and have had a menstrual period in the last two years
- Additional samples from the patient's blood tests (about 4 Tablespoons), urine tests, and bone marrow aspirate (about 4 Tablespoons) will be collected and used for research tests

Pre-transplant evaluation and Discharge from Transplant center (Group B only):
- Physical exam

Cycle 3 (Group A) or Day +100 after transplant (Group B):
- Physical exam
- Lab tests (blood and urine)
- Bone marrow aspirate
- Electrocardiogram (EKG)
- Echocardiogram

Cycle 7 (Group A) or nine months after transplant (Group B):
- Physical exam
- Lab tests (blood and urine)
- Fat aspirate
- Chest x-ray
- Electrocardiogram (EKG)
- Echocardiogram

End of treatment (Group A) or 12 months after transplant (Group B):
- Physical exam
- Lab tests (blood and urine)
- Fat aspirate
- Chest x-ray
- Electrocardiogram (EKG)
- Echocardiogram

Every six months after end of treatment (Group A) or after 12 month post transplant visit
(Group B):
- Physical exam
- Lab tests (blood and urine)
- Fat aspirate
- Chest x-ray
- Electrocardiogram (EKG)
- Echocardiogram

Patients will be asked to complete a questionnaire five times during this study (at each visit listed above). It will ask the patient questions about their health and will take about 15 minutes to complete. The patient will be asked to complete one other questionnaire, but this will be done at the beginning of the study.

How long will the Study run?

Patients will be in the study for 10 years from the time they are registered.

Sponsor(s): Mayo Clinic

Study Activation/Registration Date: 09/09/2005

IRB Review and Approval Date: 10/19/2005

Study Type: Treatment

Projected Accrual: The plan is to have up to 152 people take part in this study at Mayo Clinic.

Costs of Study:There may be standard patient care costs related to participating in a cancer research study.

Principal Investigator: Morie A. Gertz, MD

Who can I Contact for Additional Information on this Trial?

Complete Request Form for Cancer Clinical Trial Information at Mayo Clinic or
For more information about this study or other clinical trial options, please contact our Mayo Clinic Cancer Center Clinical Trials Referral Office.

• Patients living within the United States, call 507-538-7623, or e-mail cancerclinicaltrials@mayo.edu


• Patients living outside the United States may call our International Office at 507-284-8884, or e-mail intl.mcr@mayo.edu.

What is/are the Locations of this Clinical Trial?

• Rochester, MN

Last updated: 06/09/2008