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Plasma Cell MyelomaPhase 2NCT01547806

Collection of Transplant Stem Cells for Plasma Cell Myeloma

A Phase 2 study testing Filgrastim, Plerixafor and Apheresis in people with Plasma Cell Myeloma and Multiple Myeloma, run by National Cancer Institute (NCI) at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 to 75 years
Conditions the study is for
Plasma Cell Myeloma, Multiple Myeloma
What is being tested?
Filgrastim, Plerixafor and Apheresis
Could I receive a placebo?
No, everyone receives an active treatment
How many people
49 participants
Where
1 siteUnited States
Study dates
22 Feb 2012to 11 Jan 2018
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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.
Show all 11Show fewer
  • 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

Who cannot join

  • 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
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  • 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.

Trial locations and contacts

1 site in 1 country.

United States1 site
  • National Institutes of Health Clinical Center, 9000 Rockville Pike

    Bethesda, Maryland, 20892

Other trials in United States

About this study

Official title: Mobilization and Collection of Autologous Stem Cell for Transplantation (ASCT) for Plasma Cell Myeloma (PCM)

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.
Read more

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.
Read the 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

Being tested
Filgrastim, Plerixafor, Apheresis
Keywords
Autologous Hematopoietic Cell Transplantation, Plasma Cell Myeloma, Mobilization, Apheresis, Plerixafor
Medical subject headings
Multiple Myeloma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • FilgrastimDrug

    Also called Neupogen

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

  • PlerixaforDrug

    Also called Mozobil

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

  • ApheresisProcedure or surgery

    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.

Study groups in NCT01547806
Study groupRoleWhat they receive
Hematopoietic Progenitor Cells (HPC)ExperimentalSubjects will undergo mobilization and collection of HPC, Apheresis for subsequent use in various clinical protocols.
Receives: Drug: Filgrastim; Drug: Plerixafor; Procedure: Apheresis

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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.

    Measured: Day 1 of apheresis

  2. 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.

    Measured: Through Day 2 of collection

  3. 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.

    Measured: Through Day 2 of collection

  4. 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.

    Measured: Through Day 2 of collection

  5. Range of Cluster of Differentiation 34 (CD34) Cells Collected

    Progenitor cells by apheresis was determined by flow cytometry.

    Measured: Through Day 2 of collection

  6. 25th and 75th Percentile Values of Cluster of Differentiation 34 (CD34) Cells Collected

    Progenitor cells by apheresis was determined by flow cytometry.

    Measured: Through Day 2 of collection

  7. 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.

    Measured: 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 (6)
  1. 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.

    Measured: 27 months and 27 days

  2. 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

    Measured: One week of mobilization therapy

  3. 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.

    Measured: Through Day 2 of collection

  4. 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.

    Measured: Day one of collection

  5. Degree of Tumor Cell Contamination in the Final Product

    Flow cytometry to detect tumor contamination.

    Measured: Day 1 of apheresis

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

    Flow cytometry to detect tumor contamination.

    Measured: Day 1 of apheresis

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted8 Mar 2012 (estimated)
  2. Study start22 Feb 2012
  3. Primary completion17 Jun 2014
  4. Study completion11 Jan 2018

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
49 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01547806
Sponsor's protocol ID
120074
Other IDs
12-C-0074
Status last verified
Feb 2018
First submitted
6 Mar 2012
Last updated
7 Mar 2018
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • 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.

    PubMed 18287387
  • 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.

    PubMed 2301376
  • 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.

    PubMed 15693790
  • 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.

    PubMed 27735212

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Filgrastim, Plerixafor and Apheresis in people with Plasma Cell Myeloma and Multiple Myeloma, run by National Cancer Institute (NCI) at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 11 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 7 Mar 2018. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 31 Jul 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome 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 (time frame: Day 1 of apheresis). The study lists 7 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01547806, which also lists 4 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Mar 2018.

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