Escalation of Plerixafor for Mobilization of CD34+ Hematopoietic Progenitor Cells and Evaluation of Globin Gene Transfer in Patients With Sickle Cell Disease
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ClinicalTrials.gov Identifier: NCT02193191 |
Recruitment Status :
Recruiting
First Posted : July 17, 2014
Last Update Posted : April 10, 2020
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Condition or disease | Intervention/treatment | Phase |
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Sickle Cell Disease | Drug: Plerixafor | Phase 1 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 39 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | 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 |
Actual Study Start Date : | September 2014 |
Estimated Primary Completion Date : | July 2021 |
Estimated Study Completion Date : | July 2021 |

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.
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Drug: Plerixafor |
- 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.
- 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.

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Ages Eligible for Study: | 18 Years to 65 Years (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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%
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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:
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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
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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).

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02193191
Contact: Farid Boulad, MD | 212- 639-6684 | ||
Contact: Susan Prockop, MD | 212-639-6715 |
United States, New York | |
Memorial Sloan Kettering Cancer Center | Recruiting |
New York, New York, United States, 10065 | |
Contact: Farid Boulad, MD 212-639-6684 | |
Contact: Susan Prockop, MD 212-639-6715 | |
Principal Investigator: Farid Boulad, MD | |
Weill Cornell Medical College | Active, not recruiting |
New York, New York, United States, 10065 |
Principal Investigator: | Farid Boulad, MD | Memorial Sloan Kettering Cancer Center |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: | Memorial Sloan Kettering Cancer Center |
ClinicalTrials.gov Identifier: | NCT02193191 |
Other Study ID Numbers: |
13-229 |
First Posted: | July 17, 2014 Key Record Dates |
Last Update Posted: | April 10, 2020 |
Last Verified: | April 2020 |
Plerixafor 13-229 |
Anemia, Sickle Cell Hematologic Diseases Genetic Diseases, Inborn Anemia, Hemolytic, Congenital Anemia, Hemolytic Anemia |
Hemoglobinopathies Plerixafor octahydrochloride Anti-HIV Agents Anti-Retroviral Agents Antiviral Agents Anti-Infective Agents |