Plerixafor for Sickle Cell Disease

Recruiting at 1 trial location

Overseen By Roni Tamari, MD

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Memorial Sloan Kettering Cancer Center

Disqualifiers: Active infection, Major surgery, Hepatitis, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 3

Duration: 1 week

25 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Research Team

Roni Tamari, MD

Principal Investigator

Memorial Sloan Kettering Cancer Center

Eligibility Criteria

This trial is for adults aged 18-65 with confirmed Sickle Cell Disease (SS or Sβ thalassemia). Participants must have a certain level of physical fitness, adequate organ and marrow function, and not be pregnant or breastfeeding. They should not have any major health issues that could affect study participation and must agree to use contraception.

Inclusion Criteria

My blood and kidney functions are within the required ranges.

I have confirmed Sickle Cell Disease, either SS or Sβ thalassemia.

I agree to use birth control during the study.

Exclusion Criteria

My heart's pumping ability is normal.

My kidney function is reduced with a creatinine clearance below 60 ml/min.

I am not pregnant or breastfeeding.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single dose of subcutaneous plerixafor with peripheral blood studies conducted at specified intervals

1 week

3 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, including assessment of dose limiting toxicity

4 weeks

Treatment Details

Interventions

• Plerixafor

Trial Overview The trial is testing the safety and effectiveness of Plerixafor in patients with sickle cell disease. Plerixafor is already FDA-approved for cancer patients to increase blood stem cell counts before collection but hasn't been approved yet for those with sickle cell disease.

Participant Groups

Group I: Plerixafor Experimental Treatment 1 Intervention

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.

Plerixafor is already approved in European Union, United States for the following indications:

Approved in European Union as Mozobil for:

• Autologous stem cell transplantation for patients with lymphoma and multiple myeloma

Approved in United States as Mozobil for:

• Use in combination with granulocyte-colony stimulating factor (G-CSF) to mobilize hematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in patients with non-Hodgkin's lymphoma (NHL) and multiple myeloma (MM)

Who Is Running the Clinical Trial?

Memorial Sloan Kettering Cancer Center

Lead Sponsor

Trials

1,998

Recruited

602,000+

New York Blood Center

Collaborator

Trials

25

Recruited

28,400+

Weill Medical College of Cornell University

Collaborator

Trials

1,103

Recruited

1,157,000+

Sanofi

Industry Sponsor

Trials

2,246

Recruited

4,085,000+

Paul Hudson

Sanofi

Chief Executive Officer since 2019

Degree in Economics from Manchester Metropolitan University

Christopher Corsico

Sanofi

Chief Medical Officer

MD from Cornell University, MPH in Chronic Disease Epidemiology from Yale University

Duke University

Collaborator

Trials

2,495

Recruited

5,912,000+