Plerixafor for Sickle Cell Disease

Recruiting in Palo Alto (17 mi)

+1 other location

Overseen byRoni Tamari, MD

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1

Waitlist Available

Sponsor: Memorial Sloan Kettering Cancer Center

No Placebo Group

Approved in 2 Jurisdictions

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.

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.

See 3 more

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.

See 4 more

Treatment Details

Interventions

Plerixafor (Hematopoietic Stem Cell Mobilizer)

Trial OverviewThe 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

1Treatment groups

Experimental Treatment

Group I: PlerixaforExperimental Treatment1 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)