IRB Number:

07-004157

Trial Status:

Open for Enrollment

Phase: II

Why is this study being done?

Patients are being asked to take part in this research study because they have a cancer of the blood called multiple myeloma.
The purpose of this study is to see how safe and effective an investigational new drug [a drug that has not received approval by the U.S. Food and Drug Administration (FDA)] called carfilzomib is in patients with multiple myeloma who have received prior treatment for their disease. The research staff want to find out what effects, good and/or bad, it has on the patient and their cancer.
Carfilzomib is a type of drug called a proteasome inhibitor. A proteasome is a protein found within cells that has the important role of identifying and marking damaged proteins that are needed to be destroyed by the cell for survival. The inhibition of the proteasome allows for damaged protein to build up within cells. This build up of damaged protein causes the cell to die. In the laboratory, the way carfilzomib works is similar to another drug that has been approved by the FDA to treat Multiple Myeloma.

Who is Eligible to Participate in the Study?

-Age 18 or older
-Diagnosed with multiple myeloma
-Received at least one but no more than three prior therapies for multiple myeloma and have relapsed following the patient's last treatment

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

What is Involved With this Study?

If agreeing to be in the study, patients will be asked to participate in the following:

Within 14 days before beginning the study:
Patients will need to have the following exams, tests or procedures performed to find out if they can be in the study. These exams, tests or procedures are part of regular cancer care and may be done even if the patient does not join the study. If they have had some of them done recently, they may not need to be repeated.
- Complete Physical Examination (including neurologic assessment, measuring heart rate, blood pressure, temperature, height, weight, and body surface area)
- Neurologic questionnaire (to ask about pain or discomfort the patient may be feeling)
- 12-lead Electrocardiogram (to check heart function)
- Urinalysis (to be sure kidneys and other body functions are working well)
- Serum Chemistry, phosphorus and magnesium (to check general body functions)
- Coagulation Tests (to check blood clotting)
- Complete Blood Count (CBC) (to check blood count)
- Blood and / or Urine M-protein levels (to check the state of the patient's disease)
- Bone marrow biopsy and aspirate (to determine percent plasma cell involvement and chromosome arrangement)
- X-ray Skeletal survey (to detect any fractures or tumors of the bone)

During the study:
If the exams, tests and procedures show that the patient is eligible to be in the study, and they choose to take part, the patient will receive carfilzomib into one of their veins. Carfilzomib will be given over two minutes and is given twice a week for three weeks followed by twelve days of rest. This four week period of time is called a cycle. The cycle will be repeated for as long as the patient continues to do well on the medication up to 12 total cycles.

Patients will have the following procedures performed during each cycle:
On Day 1 the patient will get a physical examination, have vital signs taken and have a serum chemistry, phosphorus and magnesium and CBC blood test done. Carfilzomib will
be given to the patient by IV. During Cycle 1 only the patient will stay for observation for a minimum of one hour after receiving the drug.

On Day 2 the patient will have vital signs taken and have a serum chemistry, phosphorus and magnesium blood test done. Carfilzomib will be given to the patient by IV. During Cycle 1 only the patient will stay for observation for a minimum of one hour after receiving the drug.

On Day 8 the patient will get a physical examination, have vital signs taken and have a serum chemistry, phosphorus and magnesium and CBC blood test done. Carfilzomib will
be given to the patient by IV.

On Day 9 the patient will get a physical examination and have a serum chemistry, phosphorus and magnesium blood test done. Carfilzomib will be given to the patient by IV.

On Day 15 the patient will get a physical examination, have vital signs taken and have a serum chemistry, phosphorus and magnesium and CBC blood test done. Carfilzomib will
be given to the patient by IV. During Cycle 1 only the patient will also have their Blood and/or Urine M-protein levels checked to see the state of their disease.

On Day 16 the patient will have vital signs taken and Carfilzomib will be given to the patient by IV.

During Cycles 2, 4, 6, 9 and 12 only:
On Day 24 +/- 3 days the patient will be given a neurologic questionnaire and have a neurologic assessment. They will also have their blood and/or urine M-protein levels checked to see the state of their disease.

Within 30 days of stopping study treatment:
Patients will be asked to have a final examination with their study doctor at the end of their participation in the study. At this visit the patient will have the following tests and procedures:
Physical Examination (including measuring heart rate, blood pressure, temperature, height, weight, and body surface area)
- 12-lead Electrocardiogram (to check heart function)
- Serum Chemistry, phosphorus and magnesium (to check general body functions)
- Complete Blood Count (CBC) (to check blood count)
- Blood and / urine M-protein levels (to check the state of the patient's disease)
- Urinalysis (to be sure kidneys and other body functions are working well)
- Bone marrow biopsy and aspirate (to determine percent plasma cell involvement)
- X-ray Skeletal survey (to detect any fractures or tumors of the bone)
- Neurologic Questionaire (to ask about pain or discomfort the patient may be feeling)
- Neurologic Assessment (to determine the level of pain or discomfort the patient may be
feeling)

QUESTIONNAIRES: For this study the patient will be asked to complete a neurological questionnaire. It will ask the patient questions about their general physical, social and emotional well being. It will also ask about whether they are having certain types of symptoms that can occur with drugs that are similar to carfilzomib. It may take five to 10 minutes to complete the questionnaire.

How long will the Study run?

Patients will be in the study for up to 12 months or until their disease worsens. They will be asked to take carfilzomib for a minimum of two cycles (about two months). A cycle consists of two days a week for three weeks followed by 12 days of rest. The patient may continue treatment for up to twelve cycles or until their doctor determines that they should stop receiving the drug or they decide to stop participating in the study. After the patient is finished treatment with carfilzomib, the study doctor will ask the patient to visit the office for a follow-up exam.

Sponsor(s): Proteolix, Inc.

Study Activation/Registration Date: 10/16/2007

IRB Review and Approval Date: 09/20/2007

Study Type: Treatment

Projected Accrual: The plan is to enroll up to 100 patients to participate from multiple medical centers.

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

Principal Investigator: Alexander Keith Stewart, 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
• Scottsdale and Phoenix, AZ

Last updated: 11/17/2008