Sickle Cell DiseasePhase 1/2MobiSCDNCT07341022
STEM CELL MOBILIZATION WITH HIGH-DOSE PLERIXAFOR IN PATIENTS WITH SICKLE CELL DISEASE
A Phase 1/2 study testing Plerixafor Dose Escalation in people with Sickle Cell Disease, run by Hospital Israelita Albert Einstein.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 25 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Plerixafor Dose Escalation
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- No sites listed yet
- Study dates
- 1 Jun 2026to Jun 2028
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 25 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: 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.
Read the detailed description
Sickle cell disease (SCD), the most common monogenic disorder in the world, is currently considered a global public health problem, which underscores the need to develop curative therapies for this condition. Gene therapy has been emerging as a safe and effective curative therapeutic strategy, with the advantage of using autologous hematopoietic progenitor cells (HPCs) that are collected from the patient, edited in the laboratory, and reinfused-a process known as ex vivo gene therapy. However, mobilizing and collecting HPCs in this population is challenging, both due to disease-related factors such as bone marrow inflammation, necrosis, and ineffective erythropoiesis, and because the quantity of HPCs required for gene therapy protocols is very high. Since the use of granulocyte colony-stimulating factor (G-CSF) is contraindicated in SCD, the CXCR4 inhibitor plerixafor has been validated in this population as a safe drug, although with variable effectiveness. In healthy volunteers, plerixafor has been tested at twice the usual dose, with a significant increase in mobilization efficacy. However, in the context of sickle cell disease, and even in other diseases, this approach has never been tested. Additionally, although plerixafor is already approved for commercial use for other conditions in Brazil, it has never been tested in patients with SCD in our country, whose genetic background is more ethnically admixed compared to populations in high-income countries. This project aims to assess the efficacy and safety of plerixafor in a population of Brazilian patients with SCD. To do so, 12 patients will be enrolled using an adaptive design model that will include 3 patients at a time. The primary endpoint will be the proportion of patients who achieve a CD34+ cell count after HPC collection > 5×10⁶/kg of body weight following a single dose of plerixafor at 480 mcg/kg. Secondary endpoints will evaluate safety, efficacy, preservation of cell potency, and a preliminary cost-effectiveness analysis. This study not only introduces an unprecedented approach but also validates findings from other studies in a genetically diverse population. Moreover, it brings to Brazil the expertise necessary for HPC mobilization in SCD, an essential step for the success of future clinical gene therapy protocols to be implemented based on ongoing preclinical studies in the country, with potential for incorporation into the Brazilian Unified Health System (SUS).
- Conditions
- Sickle Cell Disease
- Being tested
- Plerixafor Dose Escalation
- Keywords
- sickle cell disease, plerixafor, stem cell mobilization
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Plerixafor Dose EscalationDrug
Plerixafor will be administered as a single dose of 480 mcg/kg.
| Study group | Role | What they receive |
|---|---|---|
| Arm A: high-dose plerixafor | Experimental | 480 mcg/kg plerixafor Receives: Drug: Plerixafor Dose Escalation |
What is being measured
The primary outcome is the main question the study is designed to answer.
CD34⁺ cell count
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
Measured: 0 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jan 2026
- Study start1 Jun 2026 (estimated)
- Primary completionDec 2027 (estimated)
- Study completionJun 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hospital Israelita Albert Einstein
- Sponsor type
- University, hospital or other
- Collaborators
- Conselho Nacional de Desenvolvimento Científico e Tecnológico
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07341022
- Sponsor's protocol ID
- MobiSCD Study
- Acronym
- MobiSCD
- Status last verified
- Dec 2025
- First submitted
- 5 Jan 2026
- Last updated
- 14 Jan 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- HEMATOPOIETIC STEM CELL MOBILIZATION USING HIGH-DOSE PLERIXAFOR IN PATIENTS WITH SICKLE CELL DISEASE: A CONTROLLED PHASE I/II TRIAL.
- ClinicalTrials.gov ID
- NCT07341022
- Acronym / sponsor ID
- MobiSCD / MobiSCD Study
- Conditions
- Sickle Cell Disease
- Keywords
- sickle cell disease, plerixafor, stem cell mobilization
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Hospital Israelita Albert EinsteinUniversity, hospital or other
- Collaborators
- Conselho Nacional de Desenvolvimento Científico e Tecnológico
- Study start
- 1 Jun 2026 (estimated)
- Primary completion
- Dec 2027 (estimated)
- Study completion
- Jun 2028 (estimated)
- First posted
- 14 Jan 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Sickle cell disease (SCD), the most common monogenic disorder in the world, is currently considered a global public health problem, which underscores the need to develop curative therapies for this condition. Gene therapy has been emerging as a safe and effective curative therapeutic strategy, with the advantage of using autologous hematopoietic progenitor cells (HPCs) that are collected from the patient, edited in the laboratory, and reinfused-a process known as ex vivo gene therapy. However, mobilizing and collecting HPCs in this population is challenging, both due to disease-related factors such as bone marrow inflammation, necrosis, and ineffective erythropoiesis, and because the quantity of HPCs required for gene therapy protocols is very high. Since the use of granulocyte colony-stimulating factor (G-CSF) is contraindicated in SCD, the CXCR4 inhibitor plerixafor has been validated in this population as a safe drug, although with variable effectiveness. In healthy volunteers, plerixafor has been tested at twice the usual dose, with a significant increase in mobilization efficacy. However, in the context of sickle cell disease, and even in other diseases, this approach has never been tested. Additionally, although plerixafor is already approved for commercial use for other conditions in Brazil, it has never been tested in patients with SCD in our country, whose genetic background is more ethnically admixed compared to populations in high-income countries. This project aims to assess the efficacy and safety of plerixafor in a population of Brazilian patients with SCD. To do so, 12 patients will be enrolled using an adaptive design model that will include 3 patients at a time. The primary endpoint will be the proportion of patients who achieve a CD34+ cell count after HPC collection > 5×10⁶/kg of body weight following a single dose of plerixafor at 480 mcg/kg. Secondary endpoints will evaluate safety, efficacy, preservation of cell potency, and a preliminary cost-effectiveness analysis. This study not only introduces an unprecedented approach but also validates findings from other studies in a genetically diverse population. Moreover, it brings to Brazil the expertise necessary for HPC mobilization in SCD, an essential step for the success of future clinical gene therapy protocols to be implemented based on ongoing preclinical studies in the country, with potential for incorporation into the Brazilian Unified Health System (SUS).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Arm A: high-dose plerixafor | Experimental | Drug: Plerixafor Dose Escalation480 mcg/kg plerixafor |
Who may qualify
- Ages
- 18 to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (1 listed)
- 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.
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Plerixafor Dose EscalationPlerixafor will be administered as a single dose of 480 mcg/kg. | Drug | Arm A: high-dose plerixafor |
Primary outcome
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Jan 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Plerixafor Dose Escalation in people with Sickle Cell Disease, run by Hospital Israelita Albert Einstein.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 14 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Karina Tozatto Maio, MD, PhD (+551121511233, karina.maio@einstein.br), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
CD34⁺ cell count (time frame: 0 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Jan 2026.