Clinical trial NCT02112045: A Study of Granix to Disrupt the Bone Marrow Microenvironment in Patients With Multiple Myeloma Undergoing Autologous Transplantation
Official title: A Study of Granix to Disrupt the Bone Marrow Microenvironment in Patients With Multiple Myeloma Undergoing Autologous Stem Cell Transplantation
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Multiple Myeloma
Lead sponsor: Washington University School of Medicine
Sponsor class: OTHER
First posted: 2014-04-11
Enrollment: 90
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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


Eligibility criteria: Inclusion Criteria:

* 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

Exclusion Criteria:

* 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).

Primary outcomes:
measure: Number of Participants With Complete Response or Stringent Complete Response
timeFrame: Day +100
Secondary outcomes:
measure: Number of Participants With Adverse Events
timeFrame: Up through Day 30
measure: Number of Participants With Overall Response
timeFrame: Up to 2 years
measure: Overall Survival as Measured by Number of Participants Alive at Last Follow-up
timeFrame: Up to 2 years
measure: Progression-free Survival as Measured by Number of Participants Without Disease Progression at Last Follow-up
timeFrame: Up to 2 years
measure: Number of Participants With Neutrophil Engraftment
timeFrame: Up to Day 30
measure: Number of Participants With Platelet Engraftment
timeFrame: Up to Day 100
Other outcomes:
Locations:
city: Saint Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
Study officials:
affiliation: Washington University School of Medicine
name: Meagan Jacoby, M.D., Ph.D.
role: PRINCIPAL_INVESTIGATOR
References: