Clinical trial NCT01753453: An Exploratory Safety Study to Investigate the Extent of Tumor Cell Mobilization (TCM) After Use of G-CSF Alone or G-CSF Plus Plerixafor in Multiple Myeloma (MM) Patients Who May be Poor Mobilizers of Stem Cells
Official title: A Pilot, Exploratory, Randomized, Phase 2 Safety Study Evaluating Tumor Cell (Plasma Cell) Mobilization and Apheresis Product Contamination in Plerixafor Plus Non-pegylated G-CSF Mobilized Patients and in Non-pegylated G-CSF Alone Mobilized Patients
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Multiple Myeloma
Keywords: Hematopoietic stem cell transplantation
Tumour cell mobilization
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
Collaborators: name: Genzyme, a Sanofi Company
First posted: 2012-12-20
Enrollment: 23
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: The primary objective of this study is to evaluate tumor cell mobilization (TCM) with non-pegylated G-CSF alone compared with non-pegylated G-CSF plus plerixafor in patients with multiple myeloma (MM) who are potentially poor mobilizers of hematopoietic stem cells (HSC).

Second objectives are to evaluate survival and disease status of G-CSF alone compared with GCSF plus plerixafor, and the efficacy and safety of G-CSF plus plerixafor when used to mobilize stem cells for autologous transplantation.


Eligibility criteria: Inclusion Criteria:

* Patients with a diagnosis of MM in partial response or complete response, who are undergo an autologous hematopoietic stem cell transplantation and could be considered potentially poor mobilizers.

Exclusion Criteria:

* Does not have an Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 1.
* Has a history of any acute or chronic leukemia (including myelodysplastic syndrome).
* Had prior allogeneic or autologous transplantation.
* Less than 3 to 6 weeks since last anti-cancer therapy.
* Chemotherapy for mobilization is not allowed.
* Has bone marrow involvement \>10% assessed based on the most recent bone marrow aspirate or biopsy performed prior to first dose of G-CSF.
* Was treated with G-CSF or other cytokine within 14 days prior to the first dose of G-CSF for mobilization.
* Has previously received plerixafor.
* Is known to be HIV positive.
* Has active hepatitis B or hepatitis C.
* Has an acute infection within 24 hours prior to dosing or antibiotic therapy within 7 days prior to the first dose of G-CSF.
* Has hypercalcaemia as evidenced by \>1 mg/dL above upper limit of normal (ULN).
* Previously received investigational therapy within 4 weeks of screening in this protocol or currently enrolled in another investigational protocol during the mobilization phase.
* Has central nervous system involvement including brain metastases or leptomeningeal disease.
* Has an electrocardiogram (ECG) or study result indicative of cardiac ischemia or a history of clinically significant rhythm disturbance(arrhythmias), or other conduction abnormality.
* Has co-morbid condition(s), which may render the patient at high risk from treatment complications or impairs his/her ability to comply with the study treatment and protocol.
* Has a white blood cell (WBC) count \<2.5 x 10\^9/L.
* Has an absolute neutrophil count (ANC) \<1.5 x 10\^9/L.
* Has a platelet count \<100 x 10\^9/L.
* Has an estimated creatine clearance ≤50 mL/min.
* Has aspartate aminotransferase/serum glutamic oxaloacetic transaminase (AST/SGOT), alanine aminotransferase/serum glutamic pyruvic transaminase (ALT/SGPT), and total bilirubin ≥2.5 x ULN.
* Does not have adequate cardiac, and pulmonary function sufficient to undergo apheresis and transplantation.
* Pregnant or breastfeeding women.
* Does not agree to use a highly effective method of contraception while on study treatment and for at least 3 months following study treatment.

Primary outcomes:
measure: The presence of myeloma tumor cells as measured by the percentage of myeloma tumor cells/CD34+ cells
timeFrame: Day 1 to Day 8 of the apheresis/treatment period
measure: The presence of myeloma tumor cells as measured by the percentage of myeloma tumor cells/plerixafor cumulative dose/kg body weights
timeFrame: Day 5 to Day 8 of the apheresis/treatment period
measure: The presence of myeloma tumor cells as measured by the percentage of myeloma tumor cells/G-CSF cumulative dose/kg body weight
timeFrame: Day 5 to Day 8 of the apheresis/treatment period
measure: The change in tumor cell mobilization(TCM) in the peripheral blood
timeFrame: Day 4 pre-G-CSF to Day 5 pre-G-CSF
measure: The number of myeloma tumor cells per patient at each apheresis
timeFrame: Day 1 to Day 8 of the apheresis/treatment period
measure: The number of patients who mobilize at least 4.5x10^5 myeloma tumor cells/kg body weight as measured in each apheresis product
timeFrame: Day 5 to Day 8 of the apheresis/treatment period
Secondary outcomes:
measure: CD34+ stem cell yield in the apheresis product
timeFrame: Day 1 to Day 8 of the apheresis/treatment period
measure: The number of patients that proceed to transplantation
timeFrame: Up to 2 months after final apheresis
measure: Overall survival
timeFrame: Day 100 post transplant and up to 2 years post first-G-CSF dose
Other outcomes:
Locations:
city: Brugge
country: Belgium
facility: Investigational Site Number 056002
city: Tallinn
country: Estonia
facility: Investigational Site Number 233001
city: Vilnius
country: Lithuania
facility: Investigational Site Number 440001
city: Stockholm
country: Sweden
facility: Investigational Site Number 752001
city: Umeå
country: Sweden
facility: Investigational Site Number 752002
Study officials:
affiliation: Sanofi
name: Clinical Sciences & Operations
role: STUDY_DIRECTOR
References: