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Collection of Transplant Stem Cells for Plasma Cell Myeloma

Primary Purpose

Plasma Cell Myeloma, Multiple Myeloma

Status

Completed

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

Filgrastim

Plerixafor

Apheresis

About this trial

This is an interventional treatment trial for Plasma Cell Myeloma focused on measuring Autologous Hematopoietic Cell Transplantation, Plasma Cell Myeloma, Mobilization, Apheresis, Plerixafor

Eligibility Criteria

18 Years - 75 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

National Institutes of Health Clinical Center, 9000 Rockville Pike

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Hematopoietic Progenitor Cells (HPC)

Arm Description

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

Outcomes

Primary Outcome Measures

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

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

Progenitor cells by apheresis was determined by flow cytometry.

Average Number of Cluster of Differentiation 34 (CD34) Cells Collected (Per kg Recipient Body Weight (BW))

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)

Progenitor cells by apheresis was determined by flow cytometry.

Range of Cluster of Differentiation 34 (CD34) Cells Collected

Progenitor cells by apheresis was determined by flow cytometry.

25th and 75th Percentile Values of Cluster of Differentiation 34 (CD34) Cells Collected

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)

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

Number of Participants With Serious and Non-Serious Adverse Events

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 Required Plerixafor + Granulocyte-colony Stimulating Factor (G-CSF) And Only G-CSF (no Plerixafor)

Percentage of patients that required Plerixafor injection in addition to G-CSF mobilization or none at all

Percentage of Patients That Achieved or Did Not Achieve 5 x 10^6 Cluster of Differentiation 34 (CD34) Cells/kg

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 Achieved ≥ 2 x 10^6 But Less Than 5 x 10^6 Cluster of Differentiation 34 (CD34) Cells/kg (Day One Collection)

Percentage of patents achieving collecting the minimum but not optimal CD34 cell number.

Degree of Tumor Cell Contamination in the Final Product

Flow cytometry to detect tumor contamination.

Impact of Plerixafor in the Degree of Tumor Cell Contamination in the Final Product

Flow cytometry to detect tumor contamination.

Full Information

NCT ID

NCT01547806

First Posted

March 6, 2012

Last Updated

February 6, 2018

Sponsor

National Cancer Institute (NCI)

1. Study Identification

Unique Protocol Identification Number

NCT01547806

Brief Title

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

2. Study Status

Record Verification Date

February 2018

Overall Recruitment Status

Completed

Study Start Date

February 22, 2012 (Actual)

Primary Completion Date

June 17, 2014 (Actual)

Study Completion Date

January 11, 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

National Cancer Institute (NCI)

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Plasma Cell Myeloma, Multiple Myeloma

Keywords

Autologous Hematopoietic Cell Transplantation, Plasma Cell Myeloma, Mobilization, Apheresis, Plerixafor

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

49 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Hematopoietic Progenitor Cells (HPC)

Arm Type

Experimental

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

Filgrastim

Other Intervention Name(s)

Neupogen

Intervention Description

Filgrastim will be administered as a single daily dose in a dose range of 10-16ug/kg/day subcutaneously for 5-7days

Intervention Type

Drug

Intervention Name(s)

Plerixafor

Other Intervention Name(s)

Mozobil

Intervention Description

Plerixafor will be given on day 4, 8-10 hours before the day 5 apheresis, dose calculated according to patient weight

Intervention Type

Procedure

Intervention Name(s)

Apheresis

Intervention Description

The minimum cluster of differentiation 34 (CD34)+ cell dose that must be collected in order to proceed with a single autologous transplantation is 2 x 106 CD34+ cells/kg.

Primary Outcome Measure Information:

Title

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

Description

Progenitor cells by apheresis was determined by flow cytometry. The stated goal was a minimum dose of 2x10EE^6/kg following apheresis.

Time Frame

Day 1 of apheresis

Title

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

Description

Progenitor cells by apheresis was determined by flow cytometry.

Time Frame

Through Day 2 of collection

Title

Average Number of Cluster of Differentiation 34 (CD34) Cells Collected (Per kg Recipient Body Weight (BW))

Description

Progenitor cells by apheresis was determined by flow cytometry.

Time Frame

Through Day 2 of collection

Title

Median and Standard Deviation of Cluster of Differentiation 34 (CD34) Cells Collected (Per Kg Recipient Body Weight) (BW)

Description

Progenitor cells by apheresis was determined by flow cytometry.

Time Frame

Through Day 2 of collection

Title

Range of Cluster of Differentiation 34 (CD34) Cells Collected

Description

Progenitor cells by apheresis was determined by flow cytometry.

Time Frame

Through Day 2 of collection

Title

25th and 75th Percentile Values of Cluster of Differentiation 34 (CD34) Cells Collected

Description

Progenitor cells by apheresis was determined by flow cytometry.

Time Frame

Through Day 2 of collection

Title

Description

Time Frame

Indefinitely until a referring physician requests the product for standard clinical care or until product(s) is no longer needed and disposed of

Secondary Outcome Measure Information:

Title

Number of Participants With Serious and Non-Serious Adverse Events

Description

Time Frame

27 months and 27 days

Title

Percentage of Patients That Required Plerixafor + Granulocyte-colony Stimulating Factor (G-CSF) And Only G-CSF (no Plerixafor)

Description

Percentage of patients that required Plerixafor injection in addition to G-CSF mobilization or none at all

Time Frame

One week of mobilization therapy

Title

Percentage of Patients That Achieved or Did Not Achieve 5 x 10^6 Cluster of Differentiation 34 (CD34) Cells/kg

Description

Here is the percentage of patients that achieved or did not achieve 5 x 10^6 CD34 cells/kg in a single apheresis.

Time Frame

Through Day 2 of collection

Title

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)

Description

Percentage of patents achieving collecting the minimum but not optimal CD34 cell number.

Time Frame

Day one of collection

Title

Degree of Tumor Cell Contamination in the Final Product

Description

Flow cytometry to detect tumor contamination.

Time Frame

Day 1 of apheresis

Title

Impact of Plerixafor in the Degree of Tumor Cell Contamination in the Final Product

Description

Flow cytometry to detect tumor contamination.

Time Frame

Day 1 of apheresis

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

75 Years

Accepts Healthy Volunteers

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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Jennifer Kanakry, M.D.

Organizational Affiliation

National Cancer Institute (NCI)

Official's Role

Principal Investigator

Facility Information:

Facility Name

National Institutes of Health Clinical Center, 9000 Rockville Pike

City

Bethesda

State/Province

Maryland

ZIP/Postal Code

20892

Country

United States

12. IPD Sharing Statement

Plan to Share IPD

Citations:

PubMed Identifier

18287387

Citation

Jemal A, Siegel R, Ward E, Hao Y, Xu J, Murray T, Thun MJ. Cancer statistics, 2008. CA Cancer J Clin. 2008 Mar-Apr;58(2):71-96. doi: 10.3322/CA.2007.0010. Epub 2008 Feb 20.

Results Reference

background

PubMed Identifier

2301376

Citation

Alexanian R, Barlogie B, Tucker S. VAD-based regimens as primary treatment for multiple myeloma. Am J Hematol. 1990 Feb;33(2):86-9. doi: 10.1002/ajh.2830330203.

Results Reference

background

PubMed Identifier

15693790

Citation

Tosi P, Zamagni E, Cellini C, Plasmati R, Cangini D, Tacchetti P, Perrone G, Pastorelli F, Tura S, Baccarani M, Cavo M. Neurological toxicity of long-term (>1 yr) thalidomide therapy in patients with multiple myeloma. Eur J Haematol. 2005 Mar;74(3):212-6. doi: 10.1111/j.1600-0609.2004.00382.x.

Results Reference

background

PubMed Identifier

27735212

Citation

Ogunniyi A, Rodriguez M, Devlin S, Adel N, Landau H, Chung DJ, Lendvai N, Lesokhin A, Koehne G, Mailankody S, Korde N, Reich L, Landgren O, Giralt S, Hassoun H. Upfront use of plerixafor and granulocyte-colony stimulating factor (GCSF) for stem cell mobilization in patients with multiple myeloma: efficacy and analysis of risk factors associated with poor stem cell collection efficiency. Leuk Lymphoma. 2017 May;58(5):1123-1129. doi: 10.1080/10428194.2016.1239261. Epub 2016 Oct 13.

Results Reference

background

Links:

URL

https://clinicalstudies.info.nih.gov/cgi/detail.cgi?B_2012-C-0074.html

Description

NIH Clinical Center Detailed Web Page

Learn more about this trial

Collection of Transplant Stem Cells for Plasma Cell Myeloma

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