Plerixafor for Sickle Cell Disease
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a drug called Plerixafor to determine if it can safely and effectively boost blood cell counts in people with sickle cell disease. While Plerixafor is already used in cancer patients to increase stem cells, it is not yet approved for sickle cell disease. The trial involves administering a single dose of Plerixafor and monitoring its effects on blood cells. Individuals with sickle cell disease confirmed by specific blood tests (SS or Sβ thalassemia) who feel generally well enough to participate might be suitable candidates. As a Phase 1 trial, this research aims to understand how Plerixafor works in people with sickle cell disease, offering a chance to be among the first to receive this treatment.
Will I have to stop taking my current medications?
The trial information does not specify if you need to stop taking your current medications. However, if you are taking an investigational drug, you must stop at least 4 weeks before joining the study.
Is there any evidence suggesting that Plerixafor is likely to be safe for humans?
Research has shown that plerixafor is generally safe for people with sickle cell disease. In various studies, patients tolerated plerixafor well, and researchers reported no major safety issues. One study found that plerixafor could safely aid in collecting stem cells from patients, potentially leading to new treatments for sickle cell disease.
The FDA has already approved plerixafor to increase blood stem cells in cancer patients, indicating its safety for humans.
Overall, early research suggests plerixafor is safe for use in sickle cell disease, but further studies are needed to confirm these findings.12345Why do researchers think this study treatment might be promising for sickle cell disease?
Researchers are excited about Plerixafor for sickle cell disease because it offers a unique approach to treatment. Unlike current therapies that often focus on managing symptoms or boosting fetal hemoglobin levels, Plerixafor works by mobilizing hematopoietic progenitor cells (HPCs) from the bone marrow into the bloodstream. This is significant because these HPCs can potentially be modified with a lentiviral vector to carry the normal ß-globin gene, which could address the genetic root of sickle cell disease. The possibility of directly correcting the genetic defect responsible for sickle cell disease is what makes this treatment particularly promising.
What evidence suggests that Plerixafor might be an effective treatment for sickle cell disease?
Research has shown that Plerixafor, the treatment under study in this trial, can increase stem cell numbers in people with sickle cell disease (SCD). In one study, 87% of participants with SCD successfully reached the target for stem cell increase, which is crucial for potential treatments. Another study found that 53% of SCD patients treated with Plerixafor achieved the desired stem cell levels in their blood. These stem cells integrate well into the body and function properly. These findings suggest that Plerixafor could improve stem cell availability in people with SCD.23678
Who Is on the Research Team?
Roni Tamari, MD
Principal Investigator
Memorial Sloan Kettering Cancer Center
Are You a Good Fit for This Trial?
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
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive a single dose of subcutaneous plerixafor with peripheral blood studies conducted at specified intervals
Follow-up
Participants are monitored for safety and effectiveness after treatment, including assessment of dose limiting toxicity
What Are the Treatments Tested in This Trial?
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.
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
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:
- Autologous stem cell transplantation for patients with lymphoma and multiple myeloma
- 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)
Find a Clinic Near You
Who Is Running the Clinical Trial?
Memorial Sloan Kettering Cancer Center
Lead Sponsor
New York Blood Center
Collaborator
Weill Medical College of Cornell University
Collaborator
Sanofi
Industry Sponsor
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
Citations
Safety and efficacy of plerixafor dose escalation for the ...
Eight of 15 patients (53%) with SCD treated with plerixafor reached the peripheral blood CD34 cell target count of at least 30 CD34+ cells/μL, including three ...
2.
ashpublications.org
ashpublications.org/bloodadvances/article/5/9/2403/475877/Disease-severity-impacts-plerixafor-mobilized-stemDisease severity impacts plerixafor-mobilized stem cell ...
Recent studies suggest that plerixafor mobilization and apheresis in patients with sickle cell disease (SCD) is safe and can allow ...
Disease severity impacts plerixafor-mobilized stem cell ...
Plerixafor mobilization of HSCs is effective for most subjects with SCD. Eighty-seven percent of participants (n = 20) successfully met the ...
Safe and efficient peripheral blood stem cell collection in ...
Our data suggest plerixafor mobilized HSC in SCD are enriched for long-term engrafting HSC, which is not true of HSC from SCD bone marrow (BM), ...
Study Details | NCT02989701 | Pilot and Feasibility Trial of ...
Plerixafor has been compared to G-CSF in a sickle cell mouse model, and results showed effective mobilization of HSC subsets, without neutrophil or endothelial ...
Plerixafor enables safe, rapid, efficient mobilization of ...
Plerixafor can be safely used to mobilize hematopoietic stem cells in sickle cell patients; this finding opens up new avenues for treatment approaches.
Safe and efficient peripheral blood stem cell collection in ...
Our data suggest plerixafor mobilized HSC in SCD are enriched for long-term engrafting HSC, which is not true of HSC from SCD bone marrow (BM), supporting a ...
Safety of Blood Stem Cell Mobilization With Plerixafor in ...
Safety of Blood Stem Cell Mobilization With Plerixafor in Patients With Sickle Cell Disease (PISMO). ClinicalTrials.gov ID NCT03664830.
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