Clinical trial NCT01547806: Collection of Transplant Stem Cells for Plasma Cell Myeloma
Official title: Mobilization and Collection of Autologous Stem Cell for Transplantation (ASCT) for Plasma Cell Myeloma (PCM)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Plasma Cell Myeloma
Multiple Myeloma
Keywords: Autologous Hematopoietic Cell Transplantation
Plasma Cell Myeloma
Mobilization
Apheresis
Plerixafor
Lead sponsor: National Institutes of Health Clinical Center (CC)
Sponsor class: NIH
First posted: 2012-03-08
Enrollment: 49
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Background:

- One beneficial treatment for plasma cell myeloma is high-dose chemotherapy followed by stem cell transplant. Researchers want to collect stem cells from the blood for later transplant.

Objectives:

- To collect stem cells for transplant as part of treatment for plasma cell myeloma.

Eligibility:

- Individuals at least 18 years of age who will have chemotherapy and stem cell transplant for plasma cell myeloma.

Design:

* Participants will be screened with a physical exam and medical history. Blood and urine samples will be collected.
* Participants will have filgrastim injections for 5 days before collection. This will move stem cells from the bone marrow to the blood.
* Participants will have apheresis to collect the stem cells.
* Participants who need additional apheresis procedures to collect stem cells will have filgrastim and a dose of plerixafor to improve the collection yield.

Detailed description: Background:

High-dose chemotherapy followed by autologous hematopoietic cell transplant (AHCT) remains a critical part of the Plasma Cell Myeloma (PCM) treatment in subjects eligible for the procedure. The timing of the procedure however, has become more controversial recently. This protocol will allow collection of Hematopoietic Progenitor Cells by Apheresis (HPC, Apheresis) in potential candidates for various PCM protocols at the Clinical Center.

The mobilizing agent plerixafor (Mozobil, Genzyme) has been recently approved by the Food and Drug Administration (FDA) for mobilization in PCM. However, the best and most cost effective strategy for its use remains to be defined.

Objectives:

Evaluate the overall validity of an HPC mobilization strategy (with granulocyte-colony stimulating factor (G-CSF) alone or in combination with plerixafor) using a formula calculating the likelihood of collecting greater than or equal to 5 time 10\^6 cluster of differentiation 34 (CD34) plus cells/kg in a single mobilization cycle.

Collect mobilized Hematopoietic Progenitor Cells by Apheresis (HPC, Apheresis) prior to AHCT for PCM

Eligibility:

Subjects with a possible indication for AHCT for the treatment of newly diagnosed PCM.

Subjects with recurrent or persistent evaluable disease who have not undergone AHCT for the treatment of the PCM.

Design:

Subjects will undergo mobilization and collection of HPC, Apheresis for subsequent use in various clinical protocols.

Mobilization will be provided by a 5-daily administration of filgrastim according to standard procedure.

The need for an additional mobilizing agent (plerixafor) to be given on day 4 of mobilization will be evaluated in real time in each patient, based on the peripheral blood CD34 count on the morning of day 4 of filgrastim administration.

Study accrual over a 3-year period: 70 subjects

Eligibility criteria: * INCLUSION CRITERIA:

Multiple Myeloma Criteria:

Subjects with an indication for autologous hematopoietic cell transplant (AHCT) for the treatment of PCM as determined by the principal investigator (PI) or lead associate investigator (LAI).

* Subjects following induction treatment for plasma cell myeloma (PCM)
* Subjects with recurrent or persistent evaluable disease who have not undergone AHCT for the treatment of the PCM.

Other Eligibility Criteria:

Age greater than or equal to 18 years and less than or equal to 75 years. In subjects between 65 and 75 years of age, physiologic age and co-morbidity will be thoroughly evaluated before enrolling.

Karnofsky performance status of 70% or greater (Eastern Cooperative Oncology Group ((ECOG) 0 or 1)

Ejection fraction (EF) by multigated acquisition scan (MUGA) or 2-D echocardiogram within institution normal limits. In case of low ejection fraction (EF), the subject may remain eligible after a stress echocardiogram is performed if the EF is more than 35% and if the increase in EF with stress is estimated at 10% or more.

Hemoglobin (Hgb) greater than or equal to 8 g/dl (transfusion acceptable)

No history of abnormal bleeding tendency.

Patients must be able to give informed consent

EXCLUSION CRITERIA:

Prior allogeneic stem cell transplantation

Hypertension not adequately controlled by 3 or less medications.

Clinically significant cardiac pathology: myocardial infarction within 6 months prior to enrollment, Class III or IV heart failure according to New York Heart Association (NYHA), uncontrolled angina, severe uncontrolled ventricular arrhythmias, or electrocardiographic evidence of acute ischemia or active conduction system abnormalities. Specifically, any history of cardio-vascular pathology or symptoms, not clearly fitting this exclusion criterion will prompt an evaluation by a Clinical Center Cardiologist and eligibility will be considered on a case-by-case basis. Should the cardiologist deem the patients findings on work-up to be not clinically significant pathology, the patient will have met this exclusion criterion.

Patients with a history of coronary artery bypass grafting or angioplasty will receive a cardiology evaluation and be considered on a case-by-case basis.

Active hepatitis B or C infection

Human immunodeficiency virus (HIV) seropositive, with positive confirmatory nucleic acid test

Patients known or found to be pregnant.

Patients of childbearing age who are unwilling to practice contraception.

Patients may be excluded at the discretion of the principal investigator (PI)/lead associate investigator (LAI) if it is deemed that allowing participation would represent an unacceptable medical or psychiatric risk.

Primary outcomes:
measure: Percentage of Patients Achieving at Least 2 x 10^6 Cluster of Differentiation 34 (CD34) Cells Per Kg Recipient Body Weight on Day 1 of Apheresis
timeFrame: Day 1 of apheresis
measure: Percentage of Patients Requiring 2 Days to Achieve at Least 2 x 10^6 Cluster of Differentiation 34 (CD34) Cells Per Kg Recipient Body Weight
timeFrame: Through Day 2 of collection
measure: Average Number of Cluster of Differentiation 34 (CD34) Cells Collected (Per kg Recipient Body Weight (BW))
timeFrame: Through Day 2 of collection
measure: Median and Standard Deviation of Cluster of Differentiation 34 (CD34) Cells Collected (Per Kg Recipient Body Weight) (BW)
timeFrame: Through Day 2 of collection
measure: Range of Cluster of Differentiation 34 (CD34) Cells Collected
timeFrame: Through Day 2 of collection
measure: 25th and 75th Percentile Values of Cluster of Differentiation 34 (CD34) Cells Collected
timeFrame: Through Day 2 of collection
measure: Number of Hematopoietic Progenitor Cell (HPC) Apheresis Products Collected and Cryopreserved for Subsequent Use in Autologous Hematopoietic Cell Transplantation (AHCT) in Subjects With Plasma Cell Myeloma (PCM)
timeFrame: Indefinitely until a referring physician requests the product for standard clinical care or until product(s) is no longer needed and disposed of
Secondary outcomes:
measure: Number of Participants With Serious and Non-Serious Adverse Events
timeFrame: 27 months and 27 days
measure: Percentage of Patients That Required Plerixafor + Granulocyte-colony Stimulating Factor (G-CSF) And Only G-CSF (no Plerixafor)
timeFrame: One week of mobilization therapy
measure: Percentage of Patients That Achieved or Did Not Achieve 5 x 10^6 Cluster of Differentiation 34 (CD34) Cells/kg
timeFrame: Through Day 2 of collection
measure: Percentage of Patients That Achieved ≥ 2 x 10^6 But Less Than 5 x 10^6 Cluster of Differentiation 34 (CD34) Cells/kg (Day One Collection)
timeFrame: Day one of collection
measure: Degree of Tumor Cell Contamination in the Final Product
timeFrame: Day 1 of apheresis
measure: Impact of Plerixafor in the Degree of Tumor Cell Contamination in the Final Product
timeFrame: Day 1 of apheresis
Other outcomes:
Locations:
city: Bethesda
country: United States
facility: National Institutes of Health Clinical Center, 9000 Rockville Pike
state: Maryland
Study officials:
affiliation: National Cancer Institute (NCI)
name: Jennifer Kanakry, M.D.
role: PRINCIPAL_INVESTIGATOR
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