NCT01280955

A Phase I/II Study: Enhancing Donor Hematopoietic Cell Engraftment With Plerixafor in Myeloablative Allogeneic Hematopoietic Cell Transplantation

Completed Phase 1/Phase 2 Results posted Last updated 24 March 2017

What this trial tests

Phase 1/Phase 2 trial testing Plerixafor in Failure of Bone Marrow Graft in 41 participants. Completed in 1 May 2015.

Timeline

1 December 2011

Primary endpoint
1 September 2014

1 May 2015

Quick facts

Lead sponsor	Mitchell Horwitz, MD
Phase	Phase 1/Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	41
Start date	1 December 2011
Primary completion	1 September 2014
Estimated completion	1 May 2015
Sites	2 locations across United States

Drugs / interventions tested

• Plerixafor (plerixafor) — full drug profile →

Conditions studied

• Failure of Bone Marrow Graft — all drugs for Failure of Bone Marrow Graft →

Sponsor

Mitchell Horwitz, MD — full company profile →

Who can join

Adults 18 to 65, any sex, with Failure of Bone Marrow Graft. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Time to Neutrophil Recovery
  Time frame: 100 Days post Transplant
  leukocytes \> 500/ul on 2 consecutive days
• Time to Platelet Recovery
  Time frame: 100 Days Post Transplant
  platelet \> 20,000/ul on 2 consecutive days
• Plerixafor-associated Adverse Events
  Time frame: 100 Days Post Transplant

Sponsor's own description

This phase I/II clinical trial will test the safety and the efficacy of post transplant administration of plerixafor in enhancing hematological recovery in humans. Patients who are appropriate candidates for myeloablative allogeneic stem cell transplantation from an HLA-matched sibling, matched unrelated donor or umbilical cord blood are eligible for enrollment. The investigators plan to enroll a total of 50 patients for this study (30 patients with HLA-matched sibling or matched unrelated donor transplant, and 20 patients with umbilical cord blood transplant). During phase I study, a small number of patients (3-6 patients from each group) will be enrolled to determine the safety of post transplant administration of plerixafor. Patients will receive plerixafor given at 240 µg/kg subcutaneously every other day beginning at day +2 after transplant until day +21 or engraftment. Limiting toxicities are defined as primary or secondary graft failure, plerixafor-related severe premature ventricular arrhythmia or death. If safety criteria are met from the investigators phase I study, the investigators will proceed with phase II study to determine the efficacy of post transplant administration of plerixafor in enhancing haematological recovery. The experimental aspect of this study is the use of plerixafor and all other aspects of care will be in line with the standard of care. Both Phase I and Phase II patients will be combined for efficacy analysis, and data collected from this study will be compared with the investigators historical control. The results from this study will set the stage and provide the justification for a larger phase 3 trial.

Publications & conference data

3 peer-reviewed publications reference this trial (live from Europe PMC):

1. Define cancer-associated fibroblasts (CAFs) in the tumor microenvironment: new opportunities in cancer immunotherapy and advances in clinical trials.
   Zhang H, Yue X, Chen Z, Liu C, et al · · 2023 · cited 317× · PMID 37784082 · DOI 10.1186/s12943-023-01860-5
2. Plerixafor (a CXCR4 antagonist) following myeloablative allogeneic hematopoietic stem cell transplantation enhances hematopoietic recovery.
   Green MM, Chao N, Chhabra S, Corbet K, et al · · 2016 · cited 21× · PMID 27535663 · DOI 10.1186/s13045-016-0301-2
3. Proteomic analysis of murine bone marrow niche microenvironment identifies thioredoxin as a novel agent for radioprotection and for enhancing donor cell reconstitution.
   An N, Janech MG, Bland AM, Lazarchick J, et al · · 2013 · cited 8× · PMID 23994289 · DOI 10.1016/j.exphem.2013.08.004