A Study of Granix to Disrupt the Bone Marrow Microenvironment in Patients With Multiple Myeloma Undergoing Autologous Transplantation

NCT02112045 | Terminated Phase 2 Results DMC FDA Drug

• 1 other identifier: 201405057
• Study Type: interventional
• Target: 90
• Locations: 1 country
• Sites: 1

Brief Summary

This randomized phase II trial compares how well adding XMO2 Filgrastim (Granix) to melphalan before a stem cell transplant works in treating patients with multiple myeloma. Chemotherapy drugs, such as melphalan, are given to prepare the bone marrow for the stem cell transplant. Giving colony-stimulating factors, such as XMO2 Filgrastim (Granix), may help multiple myeloma cells move from the patient's bone marrow to the blood where they may be more sensitive to treatment with melphalan. It is not yet known whether adding XMO2 Filgrastim (Granix) to melphalan before a stem cell transplant will work better than melphalan alone in treating multiple myeloma

Conditions

Multiple Myeloma

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Complete Response or Stringent Complete Response

  Complete response (CR) requires all of the following: * Disappearance of monoclonal protein by both protein electrophoresis and immunofixation studies from the blood and urine * \<5% plasma cells in the bone marrow * Disappearance of soft tissue plasmacytomas Stringent complete response (sCR) requires all of the following: * CR as defined above * Normal free light chain ratio * Absence of clonal cells in the bone marrow by immunohistochemistry or immunofluorescence

  Day +100

Secondary Outcomes (6)

• Number of Participants With Adverse Events

  Up through Day 30

• Number of Participants With Overall Response

  Up to 2 years

• Overall Survival as Measured by Number of Participants Alive at Last Follow-up

  Up to 2 years

• Progression-free Survival as Measured by Number of Participants Without Disease Progression at Last Follow-up

  Up to 2 years

• Number of Participants With Neutrophil Engraftment

  Up to Day 30

Study Arms (2)

Experimental: Granix and high dose melphalan (HDM)

EXPERIMENTAL

Granix on Day -7 through Day -2. HDM intravenously (IV) on Day -2. Autologous stem cell transplantation on Day 0

Drug: Granix; Drug: High dose melphalan (HDR); Procedure: Autologous Stem Cell Transplant (ASCT)

Control: High dose melphalan (HDM)

ACTIVE COMPARATOR

HDM intravenously (IV) on Day -2. Autologous stem cell transplantation on Day 0.

Drug: High dose melphalan (HDR); Procedure: Autologous Stem Cell Transplant (ASCT)

Interventions

Granix DRUG

Also known as: XM02 filgrastim

Experimental: Granix and high dose melphalan (HDM)

High dose melphalan (HDR) DRUG

Also known as: Alkeran® Tablets, Phenylalanine mustard

Control: High dose melphalan (HDM); Experimental: Granix and high dose melphalan (HDM)

Autologous Stem Cell Transplant (ASCT) PROCEDURE

Control: High dose melphalan (HDM); Experimental: Granix and high dose melphalan (HDM)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Symptomatic multiple myeloma requiring treatment
• Received at least two cycles of any regimen as initial systemic therapy for multiple myeloma and are within 2-12 months of the first dose of initial therapy
• At least 18 years of age
• Adequate autologous stem cell collection, defined as an unmanipulated, cryopreserved, peripheral blood stem cell collection containing at least 2 × 10\^6 CD34+ cells/kg based on patient body weight.
• Adequate organ function as measured by:
• Cardiac function: Left ventricular ejection fraction at rest ≥40%
• Hepatic function: Bilirubin ≤2 × ULN and aspartate amino transferase/alanine amino transferase (AST/ALT) ≤3 × ULN
• Renal function: Creatinine clearance ≥40 mL/minute (measured or calculated/estimated)
• Pulmonary function: Carbon monoxide diffusing capacity (DLCO; corrected for hemoglobin \[Hgb\]), forced expiratory volume in 1 second (FEV1), forced expiratory vital capacity (FVC) ≥50% of predicted value
• Oxygen saturation ≥92% on room air
• Eastern Cooperative Oncology Group (ECOG) performance status 0, 1, or 2.
• Able to understand and willing to sign an IRB-approved written informed consent document

You may not qualify if:

• Evidence of multiple myeloma disease progression (as defined by IMWG) any time prior to ASCT
• Prior stem cell transplant (autologous or allogeneic)
• Smoldering MM not requiring therapy
• Plasma cell leukemia
• Systemic amyloid light chain amyloidosis
• Active bacterial, viral, or fungal infection
• Seropositive for human immunodeficiency virus (HIV)
• Known, active hepatitis A, B, or C Infection
• Pregnant or breastfeeding.
• Receiving other concurrent anticancer therapy (including chemotherapy, radiation, hormonal treatment, or immunotherapy, but excluding corticosteroids) within 7 days prior to the ASCT or planning to receive any of these treatments prior to the last study visit on Day +100.
• Hypersensitive or intolerant to any component of the study drug(s) formulation
• Receiving growth factors (filgrastim, XM02-filgrastim, peg-filgrastim, plerixafor, etc) or undergoing apheresis \< 14 days prior to the start of treatment on protocol (Day -7).

Sponsors & Collaborators

• Washington University School of Medicine: lead

Study Sites (1)

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Related Links

• Alvin J. Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of Medicine

MeSH Terms

Conditions

Multiple Myeloma

Interventions

Filgrastim; Melphalan

Condition Hierarchy (Ancestors)

Neoplasms, Plasma Cell; Neoplasms by Histologic Type; Neoplasms; Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Paraproteinemias; Blood Protein Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemorrhagic Disorders; Lymphoproliferative Disorders; Immunoproliferative Disorders; Immune System Diseases

Intervention Hierarchy (Ancestors)

Granulocyte Colony-Stimulating Factor; Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phenylalanine; Amino Acids, Aromatic; Amino Acids, Cyclic; Amino Acids

Results Point of Contact

• Title: Meagan Jacoby, M.D., Ph.D.
• Organization: Washington University School of Medicine

mjacoby@wustl.edu

314-454-8304

Study Officials

• Meagan Jacoby, M.D., Ph.D.

  Washington University School of Medicine

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 4, 2014
• First Posted: April 11, 2014
• Study Start: January 20, 2015
• Primary Completion: December 13, 2017
• Study Completion: September 10, 2019
• Last Updated: November 26, 2019
• Results First Posted: January 7, 2019

Record last verified: 2019-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)