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Randomized Phase II Trial of Two Stem Cell Doses To Reduce Transplant Induced Symptom Burden in High Risk Patients With Multiple Myeloma or Amyloidosis


Phase 2
60 Years
N/A
Open (Enrolling)
Both
Multiple Myeloma, Primary Amyloidosis

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Trial Information

Randomized Phase II Trial of Two Stem Cell Doses To Reduce Transplant Induced Symptom Burden in High Risk Patients With Multiple Myeloma or Amyloidosis


Stem cells are found in the bone marrow and bloodstream, and they rebuild blood, bone
marrow, and the immune system. This study uses autologous stem cells, which are stem cells
collected from your own blood. Melphalan is designed to damage the DNA (the genetic
material) of cells, which may cause cancer cells to die. Granulocyte-colony stimulating
factor (G-CSF) is designed to help the bone marrow to make more white blood cells. G-CSF is
used in this study to help move your stem cells out of the bone marrow and into the
bloodstream. If your doctor decides it is needed, you will also receive chemotherapy to
help your blood stem cells move out of your bone marrow and into your blood. This allows
the stems cells to be collected. Researchers also want to learn how the dose of infused
stem cells affects the levels of certain cytokines in your blood. Cytokines are proteins
that can cause symptoms such as fatigue and fever.

Treatment Plan:

If you are found to be eligible to take part in this study, you will need to have a central
venous catheter (CVC) placed. A CVC is a flexible sterile tube that will be placed into a
large vein that runs under your collarbone. Having the CVC allows blood to be drawn and
medications to be given more easily and with less discomfort. You will be asked to sign a
separate consent form to have the CVC placed, and the procedure will be more fully explained
to you at that time.

If your doctor decides it is needed, you will receive chemotherapy to help your blood stem
cells move out of your bone marrow and into your blood. You will be asked to sign a
separate consent form for this chemotherapy.

Starting on the day of the stem cell infusion, you will receive G-CSF by injection under
your skin. You will have the G-CSF injected once or twice a day for about 10 days. The
injections may be given at home, if possible. You will be taught how to inject the G-CSF,
or someone who agrees to be responsible for giving you the injections may also be taught.

Once the number of stem cells in your bloodstream is high enough, the stem cells will be
collected over the course of 2-5 days. You will be asked to sign a separate consent form
for the stem cell collection, and the process will be described in more detail at that time.
Not counting the blood that is returned to you, about 32 tablespoons of blood will be drawn
for the stem cell collection.

You will keep receiving the daily G-CSF injections on the days that your stem cells are
collected. A procedure called apheresis will be used to collect the stem cells. During the
apheresis, blood will be collected either through your CVC or from a vein in one arm. The
blood will be processed through a machine to remove the stem cells, and then the rest of the
blood will be returned to you through your CVC or through a vein in the other arm. The
apheresis procedure will last several hours each time. You will be asked to sign a separate
consent form for the apheresis procedure. You will have enough stem cells collected so that
you will be able to have either 1 transplant with high doses of stem cells or 2 transplants
with standard doses of stem cells. Your stem cells will be frozen (cryopreserved) until
being given back to you.

About 2 weeks after your stem cells have been collected, you will have chemotherapy with
melphalan. The melphalan will be given over 30 minutes through your CVC. Since this
treatment destroys the normal bone marrow in addition to the myeloma cells, your blood stem
cells must be given back to you. This is because blood stem cells are able to refill the
bone marrow. If your doctor thinks it is needed, you may be admitted to the hospital the
day before you receive melphalan to receive fluids by vein to hydrate you.

Two (2) days after receiving the melphalan, your previously collected stem cells will be
unfrozen and given back to you through your CVC, similar to a blood transfusion. This is
called an autologous stem cell transplant. You will be randomly assigned (as in the toss of
a coin) to receive either a standard amount of stem cells or an experimental amount of stem
cells. You have a 50/50 chance of being assigned to either group. After you receive your
stem cells, you will be given G-CSF as an injection under the skin. You will keep having
G-CSF injections until your white blood cell count returns to normal.

After your stem cells have been given back to you, it will take about 2 weeks for enough
blood cells to be made. During this time, it is possible that you might not be making any
blood cells. If this happens, you may need to have several red blood cell and platelet
transfusions. You will receive antibiotics if infections occur, and you may need to be
given feedings through the CVC.

The entire stem cell transplantation process will be done on an outpatient basis, if
possible. Otherwise, you may need to stay in the hospital for up to 3-4 weeks.

After the stem cell transplant is completed, you will need to return to the clinic for
follow-up visits at least once a week for a month, or more often if the doctor feels it is
necessary. The follow-up visits will include the same tests and procedures as is standard
of care. Research blood (about 2-4 teaspoons each time) will also be drawn on Day -2, Day
0, Day +3, Day +5, and Day +7; twice a week during weeks 2 and 3; and once during week 4 for
the first month after the transplant, and then once a month at Months 3, 6, and 12 for
immune system tests.

Questionnaires and Physical Tests:

At several times during the study, you will be asked to complete a questionnaire about your
symptoms and quality of life. Sometimes the questionnaire will be done over the phone with
an automated phone system. Rating your symptoms using the telephone system should take less
than 5 minutes for each call. The rest of the time questionnaires will be performed on
paper and through phone interviews. You will also be asked to perform physical tests,
including walking for 6 minutes and getting in and out of a chair.

The research nurse will teach you how to use the telephone system for measuring symptoms and
your quality of life. You will perform the physical function tests and complete the symptom
questionnaire. You will also have tests of the sensitivity of the nerves in your arms and
legs. Blood (about 4-5 tablespoons) will be drawn for routine tests. Completing the
questionnaires and tasks at this first visit should take about an hour.

During your hospital stay or clinic visits for the stem cell transplant, you will be asked
on Day -2 (2 days before the transplant), Day 0, Day +3, Day +5, and Day +7 after the
transplant to fill out a paper questionnaire that measures symptoms. You will also be asked
on the same days to complete brief tasks that measure physical function.

During Weeks 2 and 3 you will be asked to fill out the paper questionnaire twice a week and
complete the tasks of physical function once a week. During Week 4, you will be asked to
fill out the questionnaire once and complete the tasks of physical function once. During
Week 4, the sensory testing of the nerves in your arms and legs will also be repeated. On
the same day as the physical tests, the research nurse will ask you to practice using the
telephone system for measuring symptoms.

After you leave the hospital or clinic, the telephone system will call you at home. The
calls will occur 1 time a week from Week 5 through Week 12 after the transplant, then 1 time
a month during Months 3, 6, 9, and 12 (+/- 1 month). The telephone system will ask you to
rate how strong and tolerable the symptoms are, and how much the symptoms interfere with
your daily life.

During clinic visits that occur about 30, 90, 180, and 360 days after the transplant, you
will be asked to complete the symptom survey and to complete brief tasks that measure
physical function. At the visit around 90 days after the transplant, you will also have the
last test of the nerves in your arms and legs.

You will be on study for about 1 year.

This is an investigational study. Melphalan with autologous stem cell transplantation is
FDA-approved for the treatment of myeloma and amyloidosis. The medications and treatment
for this study are all commercially available. Up to 100 patients will take part in this
study. All will be enrolled at MD Anderson.


Inclusion Criteria:



1. Patients with multiple myeloma over the age of 60 in any of the following disease
categories: a) Primary refractory disease b) Consolidation of a first partial or
complete remission. OR

2. Patients with primary amyloidosis.

3. Zubrod PS of <2 or Karnofsky >/= 70.

4. Left ventricular ejection fraction >/= 40%. No uncontrolled arrhythmias or
symptomatic cardiac disease.

5. FEV1, FVC and DLCO >/= 40%. No symptomatic pulmonary disease.

6. Serum bilirubin evidence of chronic active hepatitis or cirrhosis. No effusion or ascites >1L prior
to drainage.

7. HIV-negative.

8. Patient is not pregnant.

9. Patient or guardian able to sign informed consent.

10. Have greater than or equal to 10 x 10 e 6 CD34+ cells per kg of autologous stem cells
cryopreserved for stem cell transplantation, procured with 5 or fewer apheresis
collections.

Exclusion Criteria:

1) Patients unable to perform MDASI assessments due to language or cultural barriers.

Type of Study:

Interventional

Study Design:

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Mean Symptom Severity Score

Outcome Time Frame:

At one week after infusion of high dose chemotherapy

Safety Issue:

No

Principal Investigator

Nina Shah, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

UT MD Anderson Cancer Center

Authority:

United States: Institutional Review Board

Study ID:

2005-0601

NCT ID:

NCT00651937

Start Date:

March 2008

Completion Date:

Related Keywords:

  • Multiple Myeloma
  • Primary Amyloidosis
  • Multiple Myeloma
  • Primary Amyloidosis
  • Cytokine Modulation
  • Melphalan
  • Alkeran
  • Transplant Induced Symptoms
  • Stem Cell Infusion
  • Autologous stem cell transplant
  • ASCT
  • Granulocyte-colony stimulating factor
  • G-CSF
  • Apheresis
  • Amyloidosis
  • Multiple Myeloma
  • Neoplasms, Plasma Cell

Name

Location

UT MD Anderson Cancer CenterHouston, Texas  77030