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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

25 participants

Study Classification

INTERVENTIONAL

Study Start Date

2014-09-17

Study Completion Date

2025-03-27

Brief Summary

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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.

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Detailed Description

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Conditions

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Sickle Cell Disease

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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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.

Group Type EXPERIMENTAL

Plerixafor

Intervention Type DRUG

Interventions

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Plerixafor

Intervention Type DRUG

Eligibility Criteria

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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).

Minimum Eligible Age

18 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No