NCT01319864

Brief Summary

In this Phase I study, we will test the safety of the drug plerixafor (MOBOZIL) at different dose levels, used together with other anti-cancer drugs-cytarabine and etoposide. We want to find out what effects, good and /or bad, this combination of drugs has on leukemia. Plerixafor is a drug that blocks a receptor on the leukemia cell, which prevents it from staying in the bone marrow where it can be resistant to chemotherapy. Plerixafor is FDA approved for mobilizing stem cells from the bone marrow in preparation for an autologous stem cell transplant. Cytarabine and etoposide have been used as part of standard chemotherapy for ALL and AML. However, the use of plerixafor with cytarabine and etoposide in pediatric patients with relapsed or refractory ALL, AML and MDS is considered experimental.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
20

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Mar 2011

Longer than P75 for phase_1

Geographic Reach
2 countries

9 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

March 1, 2011

Completed
10 days until next milestone

First Submitted

Initial submission to the registry

March 11, 2011

Completed
11 days until next milestone

First Posted

Study publicly available on registry

March 22, 2011

Completed
2.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 28, 2013

Completed
3 years until next milestone

Study Completion

Last participant's last visit for all outcomes

June 28, 2016

Completed
Last Updated

September 7, 2018

Status Verified

September 1, 2018

Enrollment Period

2.3 years

First QC Date

March 11, 2011

Last Update Submit

September 5, 2018

Conditions

Keywords

leukemia

Outcome Measures

Primary Outcomes (1)

  • Maximum Tolerated Dose (MTD) of Plerixafor given in combination with chemotherapy

    To determine the safety and tolerability of plerixafor in combination with reinduction chemotherapy in pediatric and young adult patients with relapsed/refractory acute leukemia (AML/MDS and ALL)

    6 months post final enrollment

Secondary Outcomes (7)

  • Response Rate

    6 months post completion of treatment for final enrollment

  • Measure Peak Plasma Concentration (Cmax) of Plerixafor using serial peripheral blood sampling

    12 months following last sample collection

  • Leukemic blast mobilization

    12 months after final sample collection

  • CXCR4 expression on leukemic blasts

    12 months after last patient completes therapy

  • Measure Area Under the Concentration-Time Curve (AUC) of Plerixafor using serial peripheral blood sampling

    12 months following last sample collection

  • +2 more secondary outcomes

Study Arms (1)

Plerixafor, Dose Escalation

EXPERIMENTAL

Dose escalation of plerixafor administered intravenously in combination with IV cytarabine and IV etoposide in pediatric patients wtih relapsed/refractory AML/ALL.

Drug: Plerixafor Dose Escalation

Interventions

Plerixafor dose escalation Dose Level -1 = 3 mg/m2/dose Dose Level 1 = 6 mg/m2/dose Dose Level 2 = 9 mg/m2/dose Dose Level 3 = 12 mg/m2/dose Dose Level 4 = 15 mg/m2/dose Doses administered 4 hours prior to chemotherapy, then at the same approximate time of day on subsequent days, through the end of that cycle of chemotherapy.

Also known as: AMD3100, MOBOZIL
Plerixafor, Dose Escalation

Eligibility Criteria

Age3 Years - 29 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • \>= 3 years of age and \<30 years old at study entry
  • diagnosis of relapsed/refractory AML, ALL, secondary AML/MDS, or acute leukemia of ambiguous lineage and meet the following criteria:
  • AML/MDS or leukemia with ambiguous lineage must have \>5% blast in bone marrow
  • ALL must have an M3 marrow
  • ALL and AML must not have CNS disease
  • patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy or radiotherapy prior to entering study
  • Karnofsky score \>50% for patients \>16 years of age and Lansky \>50% for patients \<= 16 years of age
  • adequate renal and hepatic function as defined in protocol
  • adequate cardiac function as defined in protocol

You may not qualify if:

  • ALL and AML patients with CNS disease
  • Absolute blast count greater than 50,000/mcl
  • Systemic fungal, bacterial, viral or other infection without improvement despite appropriate antibiotics or other treatment
  • Significant concurrent disease, illness, psychiatric disorder or social issue that would compromise patient safety or compliance
  • Patients who have second cancer, not including secondary AML
  • Patients who are pregnant

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (9)

Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

Location

The Children's Hospital of Denver

Denver, Colorado, 80045, United States

Location

Children's Healthcare of Atlanta/Emory University

Atlanta, Georgia, 30322, United States

Location

Johns Hopkins Medical Center

Baltimore, Maryland, 21231, United States

Location

The Children's Mercy Hospital and Clinics

Kansas City, Missouri, 64108, United States

Location

Memorial Sloan-Kettering Cancer Center

New York, New York, 10021, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Penn State Hershey Children's Hospital

Hershey, Pennsylvania, 17033, United States

Location

Alberta Children's Hospital

Calgary, Alberta, T3B 6A8, Canada

Location

Related Links

MeSH Terms

Conditions

Leukemia, Myeloid, AcuteRecurrenceLeukemia, Biphenotypic, AcuteLeukemia

Interventions

plerixafor

Condition Hierarchy (Ancestors)

Leukemia, MyeloidNeoplasms by Histologic TypeNeoplasmsHematologic DiseasesHemic and Lymphatic DiseasesDisease AttributesPathologic ProcessesPathological Conditions, Signs and SymptomsLeukemia, LymphoidLymphoproliferative DisordersLymphatic DiseasesImmunoproliferative DisordersImmune System Diseases

Study Officials

  • Todd Cooper, DO

    Emory University/Children's Healthcare of Atlanta

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Associate Professor

Study Record Dates

First Submitted

March 11, 2011

First Posted

March 22, 2011

Study Start

March 1, 2011

Primary Completion

June 28, 2013

Study Completion

June 28, 2016

Last Updated

September 7, 2018

Record last verified: 2018-09

Locations