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

Sickle Cell Disease Clinical Trial

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT02193191
• Other study ID #: 13-229
• Status: Active, not recruiting
• Phase: Phase 1
• Start date: September 2014
• Est. completion date: July 2025

Study information

• Verified date: November 2023
• Source: Memorial Sloan Kettering Cancer Center
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 25
• Est. completion date: July 2025
• Est. primary completion date: July 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 65 Years

Eligibility

Inclusion Criteria:

• Patients must have confirmed and measurable Sickle Cell Disease, defined by SS or Sß 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).

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Sickle Cell Disease

Intervention

Drug:
• Plerixafor

Locations

Country	Name	City	State
United States	Memorial Sloan Kettering Cancer Center	New York	New York
United States	Weill Cornell Medical College	New York	New York

Sponsors (5)

Lead Sponsor	Collaborator
Memorial Sloan Kettering Cancer Center	Duke University, New York Blood Center, Sanofi, Weill Medical College of Cornell University

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	safety	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
Primary	efficacy	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.

External Links

•   Memorial Sloan Kettering Cancer Center