Escalation of Plerixafor for Mobilization of CD34+ Hematopoietic Progenitor Cells and Evaluation of Globin Gene Transfer in Patients With Sickle Cell Disease

March 28, 2025 updated by: Memorial Sloan Kettering Cancer Center

Safety and Efficacy Trial of Escalation of Plerixafor for Mobilization of CD34+ Hematopoietic Progenitor Cells and Evaluation of Globin Gene Transfer in Patients With Sickle Cell Disease

The purpose of this research study is to test the safety and efficacy of a drug called Plerixafor. Plerixafor is approved by the US FDA for use in increasing blood stem cell counts before collection in cancer patients. It is not yet approved for patients with sickle cell disease. The investigators want to find out if Plerixafor can be used to increase cell counts in patients with sickle cell disease.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

25

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • New York
      • New York, New York, United States, 10065
        • Weill Cornell Medical College
      • New York, New York, United States, 10065
        • Memorial Sloan Kettering Cancer Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 65 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Patients must have confirmed and measurable Sickle Cell Disease, defined by SS or Sβ thalassemia confirmed by hemoglobin fractionation.
  • ≥ 18 to 65 years of age
  • Patient must have a ECOG performance status ≤2 or Karnofsky score > 70%

  • Patients must have acceptable organ and marrow function as defined below:

    • WBC ≥ 3,000/μL
    • ANC ≥ 1,500/μL
    • platelets ≥150,000//μL
    • Hemoglobin ≥ 6 gm/dL
    • Calculated creatinine clearance ≥ 60ml/min * *Using the Cockcroft-gault equation [140 - Age(yrs)] [Weight(kg)] x 0.85 if Female 72 [Serum Creatinine (mg/dL]
  • Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately.
  • Each patient must be willing to participate as a research subject and must sign an informed consent form.

Exclusion Criteria:

  • Patients who are:

    • Receiving or received treatment with an investigational agent within 4 weeks prior to entering the study OR
    • have not recovered from adverse events due to agents administered more than 4 weeks earlier as determined by the treating physician.
  • Patients with ALT(SGPT) > 2.5 X upper limit of normal
  • Patients with a creatinine clearance of < 60 ml/min

  • Patients who have uncontrolled illness including, but not limited to:

    • Ongoing or active infection
    • Emergency room admission or hospitalization in the past 14 days
    • Major surgery in the past 30 days
    • Medical/psychiatric illness/social situations that would limit compliance with study requirements as determined by the treating physician.
  • Female patients who are pregnant or breast-feeding
  • Patients with active hepatitis B, hepatitis C, or HIV infection
  • Patients with poor cardiac function as defined by an ejection fraction < 40% are excluded due to potential poor tolerance of the fluid shifts with leukapheresis (only for patients enrolled on second phase of protocol for Leukapheresis).

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Plerixafor
Patients will receive a single dose of subcutaneous plerixafor with peripheral blood studies at approximately 0-2 hours before, approximately 6-12 hours after, and approximately 20-48 hours after plerixafor administration, with leukapheresis in the last 3 patients on the protocol. Collected HPCs will be transferred to the MSKCC CTCEF to determine if the HPCs are amenable to transduction with a lentiviral vector encoding the normal ß- globin gene.
Drug: Plerixafor

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
safety
Time Frame: up to 30 days
Safety is assessed using a dose limiting toxicity (DLT) endpoint. Definition of a DLT is the occurrence of any of the below events that meets the following criteria: The occurrence of a vasoocclusive crisis requiring hospitalization, acute chest syndrome, CNS acute event, or any other disease related ischemic-based adverse event (AE) should be considered as a DLT, if occurring in the 48 hours DLT observation period.
up to 30 days
efficacy
Time Frame: ≥ 30/ul at either 6-12 hours or 24-48 hours post plerixafor.
Efficacy is defined as 100% of evaluable patients reaching a PB CD34 concentration ≥ 30/uL.
≥ 30/ul at either 6-12 hours or 24-48 hours post plerixafor.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Memorial Sloan Kettering Cancer Center

Collaborators

Sanofi
Duke University
Weill Medical College of Cornell University
New York Blood Center

Investigators

  • Principal Investigator: Roni Tamari, MD, Memorial Sloan Kettering Cancer Center

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 17, 2014

Primary Completion (Actual)

March 27, 2025

Study Completion (Actual)

March 27, 2025

Study Registration Dates

First Submitted

July 15, 2014

First Submitted That Met QC Criteria

July 15, 2014

First Posted (Estimated)

July 17, 2014

Study Record Updates

Last Update Posted (Actual)

March 30, 2025

Last Update Submitted That Met QC Criteria

March 28, 2025

Last Verified

March 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 13-229

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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