Collection of Transplant Stem Cells for Plasma Cell Myeloma

Phase 2

Completed

Conditions: Plasma Cell Myeloma
Multiple Myeloma

Interventions: Drug: Filgrastim
Drug: Plerixafor
Procedure: Apheresis

Registration Number: NCT01547806

Lead Sponsor: National Cancer Institute (NCI)

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

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 49

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type: INTERVENTIONAL

Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Hematopoietic Progenitor Cells (HPC)	Apheresis	Subjects will undergo mobilization and collection of HPC, Apheresis for subsequent use in various clinical protocols.
Hematopoietic Progenitor Cells (HPC)	Filgrastim	Subjects will undergo mobilization and collection of HPC, Apheresis for subsequent use in various clinical protocols.
Hematopoietic Progenitor Cells (HPC)	Plerixafor	Subjects will undergo mobilization and collection of HPC, Apheresis for subsequent use in various clinical protocols.

Primary Outcome Measures

Name	Time	Method
Average Number of Cluster of Differentiation 34 (CD34) Cells Collected (Per kg Recipient Body Weight (BW))	Through Day 2 of collection	Progenitor cells by apheresis was determined by flow cytometry.
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	Day 1 of apheresis	Progenitor cells by apheresis was determined by flow cytometry. The stated goal was a minimum dose of 2x10EE\^6/kg following apheresis.
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	Through Day 2 of collection	Progenitor cells by apheresis was determined by flow cytometry.
Median and Standard Deviation of Cluster of Differentiation 34 (CD34) Cells Collected (Per Kg Recipient Body Weight) (BW)	Through Day 2 of collection	Progenitor cells by apheresis was determined by flow cytometry.
Range of Cluster of Differentiation 34 (CD34) Cells Collected	Through Day 2 of collection	Progenitor cells by apheresis was determined by flow cytometry.
25th and 75th Percentile Values of Cluster of Differentiation 34 (CD34) Cells Collected	Through Day 2 of collection	Progenitor cells by apheresis was determined by flow cytometry.
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)	Indefinitely until a referring physician requests the product for standard clinical care or until product(s) is no longer needed and disposed of	The cryopreserved stem cells are stored under Good Manufacturing Practice (GMP) conditions in the National Institutes of Health (NIH) Department of Transfusion Medicine until a referring physician requests the products for standard clinical care.

Secondary Outcome Measures

Name	Time	Method
Percentage of Patients That Achieved or Did Not Achieve 5 x 10^6 Cluster of Differentiation 34 (CD34) Cells/kg	Through Day 2 of collection	Here is the percentage of patients that achieved or did not achieve 5 x 10\^6 CD34 cells/kg in a single apheresis.
Percentage of Patients That Required Plerixafor + Granulocyte-colony Stimulating Factor (G-CSF) And Only G-CSF (no Plerixafor)	One week of mobilization therapy	Percentage of patients that required Plerixafor injection in addition to G-CSF mobilization or none at all
Number of Participants With Serious and Non-Serious Adverse Events	27 months and 27 days	Here is the number of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned.
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)	Day one of collection	Percentage of patents achieving collecting the minimum but not optimal CD34 cell number.
Degree of Tumor Cell Contamination in the Final Product	Day 1 of apheresis	Flow cytometry to detect tumor contamination.
Impact of Plerixafor in the Degree of Tumor Cell Contamination in the Final Product	Day 1 of apheresis	Flow cytometry to detect tumor contamination.