Clinical trial NCT04552743: MGTA-145 + Plerixafor in the Mobilization of Hematopoietic Stem Cells for Autologous Transplantation in Multiple Myeloma
Official title: Phase II Study of MGTA-145 in Combination With Plerixafor in the Mobilization of Hematopoietic Stem Cells for Autologous Transplantation in Patients With Multiple Myeloma
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Multiple Myeloma
Lead sponsor: Stanford University
Sponsor class: OTHER
First posted: 2020-09-17
Enrollment: 25
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This study evaluates a new drug MGTA-145 in combination with plerixafor (Mozobil) to mobilize stem cells into the peripheral blood for collection by apheresis. The stem cells will be used for autologous stem cell transplant for treatment of multiple myeloma.

Detailed description: PRIMARY OBJECTIVE

1. To assess the efficacy of MGTA-145 in combination with plerixafor in mobilizing adequate number of hematopoietic stem cells (\> 2 x 10e6 CD34+ cells/kg) in patients with multiple myeloma (MM) in preparation for autologous stem cell transplantation (ASCT).

SECONDARY OBJECTIVES

1. To assess the efficacy of MGTA-145 and plerixafor in mobilizing different Hematopoietic stem cells (HSCs) target goals in patients with MM in preparation for ASCT.
2. To assess the safety and tolerability of MGTA-145 and plerixafor for mobilizing HSCs in patients with MM.
3. To assess the engraftment rate and time to engraftment following ASCT after HSC mobilization with MGTA-145 and plerixafor in patients with MM undergoing upfront ASCT.
4. To assess rate of ongoing engraftment at Day 30 and 100 after stem cell infusion in patients with MM who are mobilized with MGTA-145 and plerixafor undergoing upfront ASCT.
5. To assess transplant and disease-related outcomes after mobilization of HSCs with MGTA-145 and plerixafor in patients with MM undergoing upfront ASCT.

Eligibility criteria: Inclusion Criteria:

* Diagnosis of multiple myeloma (MM) per the International Myeloma Working Group (IMWG) criteria
* Eligible for autologous stem cell transplantation (ASCT) per institutional guidelines
* Within 1 year of start of therapy for multiple myeloma
* Cardiac and pulmonary status sufficient to undergo apheresis and transplantation per institutional transplant guidelines
* Calculated creatinine clearance \> 30 mL/min, according to the Modification of Diet in Renal Disease (MDRD) formula.
* Absolute neutrophil count (ANC) \> 1500 x 10e6/L
* Platelet count \> 100,000 x 10e6/L
* Ability to understand and the willingness to sign a written informed consent document.
* Agreement to use an approved form of contraception for male patients or female patients of childbearing potential.

Exclusion Criteria:

* History of prior stem cell transplant for multiple myeloma or other indications
* Planned tandem stem cell transplant
* Prior history of failure to collect HSCs.
* Total bilirubin \> 1.5x upper limit of normal (ULN) in the absence of a documented history of Gilbert's syndrome
* Aspartate aminotransferase (AST)/alanine aminotransferase (ALT) \> 3x ULN
* Known allergy to MGTA-145 or plerixafor.
* Lifetime exposure to lenalidomide or another immunomodulatory drug greater than 6 cumulative months of treatment, ie, \> 6 cycles of 28 days or \> 8cycles of 21 days
* Pregnant or lactating

Primary outcomes:
measure: Number of Participants For Whom 2.0 x 10e6 CD34+ HSC Cells/kg Could be Collected in 1 or 2 Apheresis Harvests
timeFrame: 2 days
Secondary outcomes:
measure: Other Measures of Hematopoietic Stem Cell (HSC) Yield in the Apheresis Product
timeFrame: 2 days
measure: Time To Neutrophil Engraftment
timeFrame: 15 days
measure: Maintenance of Neutrophil Engraftment [Absolute Neutrophil Count (ANC) ≥ 0.5 x 10e9/L]
timeFrame: 100 days
measure: Time To Platelet Engraftment ≥ 20 x 10e9/L
timeFrame: 33 days
measure: Time To Platelet Engraftment ≥ 50 x 10e9/L
timeFrame: 44 days
measure: Infusion-related Toxicities
timeFrame: 7 days after mobilization procedure
measure: Progression-free Survival (PFS)
timeFrame: 100 days after infusion procedure
measure: Transplant-related Mortality
timeFrame: 100 days after infusion procedure
measure: Non-relapse-related Mortality
timeFrame: 100 days after infusion procedure
measure: Overall Survival (OS)
timeFrame: 100 days after infusion procedure
Other outcomes:
Locations:
city: Stanford
country: United States
facility: Stanford University
state: California
Study officials:
affiliation: Stanford Medicine at Stanford University
name: Surbhi Sidana, MD
role: PRINCIPAL_INVESTIGATOR
References: