STEM CELL MOBILIZATION WITH HIGH-DOSE PLERIXAFOR IN PATIENTS WITH SICKLE CELL DISEASE (MobiSCD)
HEMATOPOIETIC STEM CELL MOBILIZATION USING HIGH-DOSE PLERIXAFOR IN PATIENTS WITH SICKLE CELL DISEASE: A CONTROLLED PHASE I/II TRIAL.
The objective of this study is to demonstrate whether high-dose plerixafor can effectively mobilize hematopoietic stem cells in patients with sickle cell disease. It will also learn about the safety of this drug in higher doses in these patients. The main questions it aims to answer are:
Does high-dose plerixafor mobilize enough hematopoietic stem cells? What medical problems do participants have when taking high-dose plerixafor?
Participants will:
Undergo transfusion Take high-dose plerixafor Be submitted to stem cell collection by apheresis Visit the clinic 10 days after the procedure Be contacted by the research team 30 days after the procedure.
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
Study Type
Enrollment (Estimated)
Phase
- Phase 2
- Phase 1
Contacts and Locations
Study Contact
- Name: Karina Tozatto Maio, MD, PhD
- Phone Number: +551121511233
- Email: karina.maio@einstein.br
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
- Adult
Accepts Healthy Volunteers
Description
Inclusion Criteria:
Patients with sickle cell disease 18 to 25 years old At least one allogeneic HSCT indication followin the Brazilian Bone Marrow Transplant Society (SIMÕES et al., [s.d.]) ECOG/ Karnofsky/Lansky scores > 80 Hemoglobin > 7 g/dL, WBC counts > 3000/mm3, neutrophil counts > 1500/mm3, platelet counts > 150000/mm3 No evidence of severe hepatic disfunction, defined as aspartate aminotransferase and alanine aminotransferase < 5 times ULN or bilirubin < 2,5 times ULN No evidence of renal disfunction, defined as creatinine <1,5 mg/dL ou GFR> 60 mL/min LVEF > 40% and no signals of pulmonary hypertension Negative serologies for HIV, HBV, HCV, syphilis, Chagas disease or HTLV Being able to undergo partial exchange transfusion to lower HbS <30% within one week before CD34+ mobilization and collection No pregnancy or breastfeeding; acceptance to use two contraceptive methods during the study.
Exclusion Criteria:
Emergency room admission or hospitalization in the past 14 days prior to first dose of study drug Major surgery in the past 30 days prior to first dose of study drug Active and painful splenomegaly or splenomegaly (size greater than upper limit of normal on examination).
Participant who, by medical history, requires rare donor registry RBC units for transfusion, or is unable to receive routine transfusion. Eligible study participants must have undergone prior work-up for the presence of red cell alloantibodies and confirmation of available compatible blood product support Known allergy to or contraindication for motixafortide administration, or medications routinely administered during apheresis Participant who has had a prior autologous or allogeneic transplantation, inclusive of gene therapy Active viral, bacterial, fungal, or parasitic infection. History of cancer, excluding squamous carcinoma of the skin and cervical carcinoma in situ.
Participant who has received experimental therapy within 4 weeks prior to providing informed consent Poorly controlled diabetes mellitus, as assessed by the Investigator Concomitant treatment with alternative investigational agent unable to be held for 30 days Unwillingness to use a highly effective method of contraception for 1 month after motixafortide Pregnancy Inability or unwillingness of research participant or legal guardian/ representative to give written informed consent.
Inability or unwillingness of research participant to hold hydroxyurea for 30 days prior to first dose of study drug
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
|---|---|
|
Experimental: Arm A: high-dose plerixafor
480 mcg/kg plerixafor
|
Plerixafor will be administered as a single dose of 480 mcg/kg.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
|---|---|---|
|
CD34⁺ cell count
Time Frame: 0 days
|
Proportion of patients who achieve a CD34⁺ cell count > 5 × 10⁶/kg body weight after HSC collection using plerixafor at a dose of 480 µg/kg
|
0 days
|
Collaborators and Investigators
Study record dates
Study Major Dates
Study Start (Estimated)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- MobiSCD Study
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
product manufactured in and exported from the U.S.
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