Plerixafor for Sickle Cell Disease
(PISMO Trial)

Recruiting in Palo Alto (17 mi)

Overseen byLeo Wang, MD

Age: 18 - 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1

Waitlist Available

Sponsor: City of Hope Medical Center

No Placebo Group

Approved in 2 Jurisdictions

Trial Summary

What is the purpose of this trial?The objective of this study is to investigate if up to two injections of plerixafor represent a safe and effective strategy to mobilize adequate numbers of CD34+ hematopoietic stem progenitor cells (HSPC) for autologous hematopoietic cell transplantation (HCT) in sickle cell disease (SCD) patients

Eligibility Criteria

This trial is for individuals with sickle cell disease who weigh between 50-120 kg and have experienced severe pain crises or other complications like stroke, acute chest syndrome, or osteonecrosis. They should have good organ function and no history of certain conditions like alpha thalassemia, HIV/HTLV, uncontrolled infections, malignancy (except some skin cancers), recent major surgery, or prior gene therapy.

Inclusion Criteria

I have sickle cell disease with a specific genetic makeup.

Weight between 50 and 120 kg

I am able to care for myself but may not be able to do active work.

+17 more

Exclusion Criteria

Known hypersensitivity to plerixafor or any excipient contained in Mozobil

I have never received gene therapy.

Abnormal pulmonary function tests (adults with mild or moderate obstruction or restriction or diffusion defects are eligible, per Investigator discretion)

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Participant Groups

The study tests whether two injections of Plerixafor are safe and effective in mobilizing enough CD34+ stem cells for autologous transplantation in patients with sickle cell disease. It aims to improve treatment by potentially enabling successful transplants.

1Treatment groups

Experimental Treatment

Group I: PlerixaforExperimental Treatment1 Intervention

Up to two subcutaneous injections of plerixafor (starting dose level: 240 µg/kg/dose)

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)