AMD3100 for Sensitizing in Allogeneic Blood or Marrow Transplant for Chemotherapy Resistant Pediatric Acute Leukemia

NCT01655875 | Terminated Phase 1 DMC

• 2 other identifiers: IRB00053638CHOA AMD3100 Pilot
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

Brief Summary

This study is for patients 2-21 years old who have acute leukemia that has not responded well to chemotherapy and will have a bone marrow transplant. This is a pilot (phase 1) study of AMD3100(also called Plerixafor, Mozobil). AMD3100 is given in combination with a standard pre-transplant conditioning regimen (total body irradiation, etoposide and cyclophosphamide). The conditioning regimen is the treatment that is given just before the transplant. This treatment kills leukemia cells as well as healthy bone marrow and immune cells. Researchers want to learn more about how AMD3100 affects acute leukemia cells. Blood and bone marrow samples from study participants will be collected to find out if AMD3100 is making patients' cells more sensitive to the conditioning regimen and to find out how it does this. The first six patients receive three daily doses (240 mcg/kg via IV). If it appears that three doses do not significantly increase the side effects of transplant conditioning, the investigators will give a second group of six patients five daily doses.

Conditions

Pediatric Acute Myeloblastic Leukemia, Relapsed; Pediatric Acute Lymphoblastic Leukemia, Relapsed

Keywords

Allogeneic Blood and Marrow Transplantation

Outcome Measures

Primary Outcomes (1)

• AMD3100 safety

  Incidence of grade 3 and 4 regimen-related toxicity assessed per Bearman scale at day 42. Patients in dose level 1 get 3 days of AMD3100. If 2 cases of grade 4 toxicity or 3 cases of grade 3-4 toxicity occur, dose level 1 and study will be closed. Otherwise, after 6 patients have been assessed at day 42, the dose will be escalated to 5 days of AMD3100 (dose level 2). If 2 cases of grade 4 toxicity or 3 cases of grade 3-4 toxicity occur, the study will be closed. Otherwise, after 6 patients have been enrolled at this level the study will be closed to enrollment.

  day 42 after bone marrow transplant

Secondary Outcomes (1)

• AMD3100 correlative biology analyses

  2 years

Study Arms (1)

bone marrow transplant

EXPERIMENTAL

Drug: AMD3100

Interventions

AMD3100 DRUG

AMD3100 will first be administered at 240 mcg/kg (the FDA approved dose for mobilization of autologous PBSC) IV once daily prior to conditioning for 3 days (days -6, -5 and -4) in the first group of participants. If toxicity criteria are met, the dosing will be escalated in the second group of participants to 240 mcg/kg IV once daily prior to conditioning for 5 days (days -8, -7, -6, -5 and -4).

Also known as: Mozobil, Plerixafor

bone marrow transplant

Eligibility Criteria

• Age: 2 Years - 22 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Must have chemotherapy-resistant acute leukemia (primary refractory or relapsed and refractory AML, ALL, undifferentiated, bi-lineage or mixed lineage leukemia)
• Participant must have a well HLA matched related, mismatched related or unrelated marrow donor with whom the patient is allele matched at at least 7 of 8 HLA loci or a single unrelated cord blood unit matched at at least 4 of 6 HLA loci with minimal dose of 4x10(7)NC/Kg

You may not qualify if:

• Prior allogeneic or autologous hematopoietic stem cell transplantation
• Prior exposure to AMD3100
• Active central nervous system leukemia
• Uncontrolled viral, bacterial, fungal, protozoal infection
• HIV infection
• Does not meet standard organ function for transplant

Sponsors & Collaborators

• Emory University: lead

Study Sites (1)

Children's Healthcare of Atlanta

Atlanta, Georgia, 30322, United States

Location

MeSH Terms

Conditions

Recurrence; Precursor Cell Lymphoblastic Leukemia-Lymphoma

Interventions

plerixafor

Condition Hierarchy (Ancestors)

Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Leukemia, Lymphoid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases

Study Officials

• Kuang-Yueh Chiang, MD, PhD

  Emory University

  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: July 27, 2012
• First Posted: August 2, 2012
• Study Start: June 1, 2012
• Primary Completion: April 1, 2014
• Study Completion: April 1, 2014
• Last Updated: April 7, 2014

Record last verified: 2014-04

Locations

US (1)