Sickle Cell DiseasePhase 1PISMONCT03664830
Safety of Blood Stem Cell Mobilization With Plerixafor in Patients With Sickle Cell Disease
A Phase 1 study testing Plerixafor in people with Sickle Cell Disease, run by City of Hope Medical Center at 1 site in United States.
Trial summary
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 Read more about this study
- Who is it for?
- Ages 18 to 40 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Plerixafor
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteUnited States
- Study dates
- 19 Sep 2018to 6 Jul 2027
- Phase
- Phase 1
- 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 40 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Weight between 50 and 120 kg;
- Karnofsky performance status (KPS) ≥70%;
- Confirmed diagnosis of sickle cell disease with βS/βS or βS/β0 or βS/β+ genotype;
- Must have had one or more of the following events in the 2 year period preceding enrollment:
- History of ≥2 severe vaso-occlusive pain crises (VOC) (or at least two episodes in the year preceding the setting up of regular transfusion protocol). A severe VOC is defined as an episode of pain lasting more than 2 hours severe enough to require care at a medical facility.
- History of ≥1 episodes of acute chest syndrome despite the institution of supportive care measures (i.e. asthma therapy and/or hydroxyurea)
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- Clinically significant neurological event (stroke) or any neurological deficit lasting 24 hours. A stroke is defined as a sudden neurological change lasting more than 24 hours that is accompanied by cerebral magnetic resonance imaging (MRI) changes.
- Prior treatment with regular RBC transfusion therapy, defined as receiving ≥8 transfusions per year for >1 year to prevent vaso-occlusive clinical complications (i.e. pain, stroke, and acute chest syndrome)
- Osteonecrosis of two or more joints;
- Anti-erythrocyte alloimmunization (>2 antibodies);
- Presence of sickle cell cardiomyopathy documented by Doppler echocardiography;
- Presence of any significant cerebral abnormality such as stenosis or occlusions on magnetic resonance imaging (MRA)
- Meet current eligibility requirements for donation for mobilization at the COH DAC;
- Adequate renal function: defined as a creatinine estimated FDR (eGFR) of ≥60 ml/min;
- Adequate liver function: defined by a serum conjugated (direct) bilirubin <2.5x upper limit of normal (ULN) for age; AST and ALT <5x ULN for age as per laboratory;
- Adequate cardiac function: defined as left ventricular ejection fraction >50%;
- Adequate hematologic parameters: WBC ≥2.5 x 10^9/L; platelet count ≥120 x10^9/L; hemoglobin >8 g/dL;
- Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry.
Who cannot join
- Diagnosed with alpha thalassemia (two or more gene deletions or any α-globin structural variants);
- Seropositivity for HIV-1/2 (Human Immunodeficiency Virus) or HTLV-1/2(Human T-Lymphotropic Virus);
- Evidence of uncontrolled bacterial, viral, or fungal infections (currently taking medication and progression of clinical symptoms) within one month prior to treatment. Participants with fever should await resolution of symptoms before starting the treatment;
- Any clinically significant active infection which, in the opinion of the investigator, would require significant medical intervention;
- Abnormal pulmonary function tests (adults with mild or moderate obstruction or restriction or diffusion defects are eligible, per Investigator discretion).
- History of pulmonary hypertension, proven by cardiac catheterization;
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- History of malignancy or immunodeficiency disorder, (i.e., subjects with prior malignancy must be disease-free for 5 years), except curatively-treated basal cell carcinoma or cutaneous squamous cell carcinoma;
- Participation in any study with an investigational agent or medical device within 90 days of screening;
- Major surgery in the past 30 days;
- Prior receipt of any gene transfer product;
- Bone marrow harvest in the past year;
- Known myelodysplasia of the bone marrow or abnormal bone marrow cytogenesis;
- Known hypersensitivity to plerixafor or any excipient contained in Mozobil;
- G-CSF or plerixafor medication within 4 weeks of treatment;
- Pregnant or nursing women;
- Any condition or chronic physical, neurological, or mental illness, which in the opinion of the investigator, makes participation ill advised.
Trial locations and contacts
1 site in 1 country.
United States1 site
City of Hope Medical Center
Duarte, California, 91010
About this study
Official title: A Pilot Study To Evaluate The Safety And Feasibility of Hematopoietic Progenitor Cell Mobilization With Plerixafor as Part of a Gene Therapy Strategy in Sickle Cell Disease
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
Read the detailed description
Allogeneic hematopoietic cell transplantation (HCT) is the only curative option for sickle cell disease (SCD) patients but its use is significantly limited by the lack of compatible donors. Gene therapy using autologous hematopoietic stem cell (HSC) transplantation represents an alternative approach but requires the collection of significant numbers of cells. Severe adverse events have been observed in SCD patients after mobilization using the standard agent granulocyte colony-stimulating factor (G-CSF), and bone marrow harvesting is also associated with side effects. The use of a single administration of plerixafor has been suggested as an alternative mobilization strategy in SCD but may not mobilize sufficient number of HSC. In this study, our primary objective is to assess if up to two injections of plerixafor (starting dose level: 240 µg/kg/dose) are safe in SCD patients and can recruit enough blood stem cells needed for future gene therapy.
- Conditions
- Sickle Cell Disease
- Being tested
- Plerixafor
- Keywords
- plerixafor, sickle cell disease, mobilization, hematopoietic stem cell, gene therapy
- 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.
PlerixaforDrug
Up to two subcutaneous injections of plerixafor (starting dose level: 240 µg/kg/dose)
| Study group | Role | What they receive |
|---|---|---|
| Plerixafor | Experimental | Up to two subcutaneous injections of plerixafor (starting dose level: 240 µg/kg/dose) Receives: Drug: Plerixafor |
What is being measured
The primary outcome is the main question the study is designed to answer.
Toxicities
Scored using the Common Terminology Criteria for Adverse Events (CTCAE) of the National Cancer Institute (NCI) version 4.03
Measured: 120 hours (5 days) from the last injection of plerixafor
Secondary outcomes (1)
Stem cell mobilization feasibility
Assessed by measuring the number of mobilized CD34+ cells/µL of peripheral blood
Measured: 6 hours after the first injection of plerixafor.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Sep 2018
- Study start19 Sep 2018
- Primary completion6 Jul 2027 (estimated)
- Study completion6 Jul 2027 (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.
- More trials run by City of Hope Medical CenterThe sponsor's other registered studies.
- 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Each SCD research participant will receive up to two subcutaneous injections of plerixafor (starting dose level: 240 µg/kg/dose)
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- City of Hope Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Leo Wang, MD, Principal investigator, City of Hope Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03664830
- Sponsor's protocol ID
- 17426
- Acronym
- PISMO
- Status last verified
- Aug 2026
- First submitted
- 7 Sep 2018
- Last updated
- 2 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study To Evaluate The Safety And Feasibility of Hematopoietic Progenitor Cell Mobilization With Plerixafor as Part of a Gene Therapy Strategy in Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT03664830
- Acronym / sponsor ID
- PISMO / 17426
- Conditions
- Sickle Cell Disease
- Keywords
- plerixafor, sickle cell disease, mobilization, hematopoietic stem cell, gene therapy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 5 participants (actual)
- Lead sponsor
- City of Hope Medical CenterUniversity, hospital or other
- Study start
- 19 Sep 2018 (actual)
- Primary completion
- 6 Jul 2027 (estimated)
- Study completion
- 6 Jul 2027 (estimated)
- First posted
- 11 Sep 2018 (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 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
Detailed description
Allogeneic hematopoietic cell transplantation (HCT) is the only curative option for sickle cell disease (SCD) patients but its use is significantly limited by the lack of compatible donors. Gene therapy using autologous hematopoietic stem cell (HSC) transplantation represents an alternative approach but requires the collection of significant numbers of cells. Severe adverse events have been observed in SCD patients after mobilization using the standard agent granulocyte colony-stimulating factor (G-CSF), and bone marrow harvesting is also associated with side effects. The use of a single administration of plerixafor has been suggested as an alternative mobilization strategy in SCD but may not mobilize sufficient number of HSC. In this study, our primary objective is to assess if up to two injections of plerixafor (starting dose level: 240 µg/kg/dose) are safe in SCD patients and can recruit enough blood stem cells needed for future gene therapy.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Each SCD research participant will receive up to two subcutaneous injections of plerixafor (starting dose level: 240 µg/kg/dose)
| Arm | Type | Receives |
|---|---|---|
| Plerixafor | Experimental | Drug: PlerixaforUp to two subcutaneous injections of plerixafor (starting dose level: 240 µg/kg/dose) |
Who may qualify
- Ages
- 18 to 40 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (18 listed)
- Weight between 50 and 120 kg;
- Karnofsky performance status (KPS) ≥70%;
- Confirmed diagnosis of sickle cell disease with βS/βS or βS/β0 or βS/β+ genotype;
- Must have had one or more of the following events in the 2 year period preceding enrollment:
- Meet current eligibility requirements for donation for mobilization at the COH DAC;
Key exclusion criteria (16 listed)
- Diagnosed with alpha thalassemia (two or more gene deletions or any α-globin structural variants);
- Seropositivity for HIV-1/2 (Human Immunodeficiency Virus) or HTLV-1/2(Human T-Lymphotropic Virus);
- Evidence of uncontrolled bacterial, viral, or fungal infections (currently taking medication and progression of clinical symptoms) within one month prior to treatment. Participants with fever should await resolution of symptoms before starting the treatment;
- Any clinically significant active infection which, in the opinion of the investigator, would require significant medical intervention;
- Abnormal pulmonary function tests (adults with mild or moderate obstruction or restriction or diffusion defects are eligible, per Investigator discretion).
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 |
|---|---|---|
| PlerixaforUp to two subcutaneous injections of plerixafor (starting dose level: 240 µg/kg/dose) | Drug | Plerixafor |
Primary outcome
Toxicities
Time frame: 120 hours (5 days) from the last injection of plerixafor
Scored using the Common Terminology Criteria for Adverse Events (CTCAE) of the National Cancer Institute (NCI) version 4.03
1 secondary outcome
Stem cell mobilization feasibility
Time frame: 6 hours after the first injection of plerixafor.
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 2 Sep 2026.
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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. This study is not recruiting at the moment.
Leo Wang, MD principal investigator
City of Hope Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Plerixafor in people with Sickle Cell Disease, run by City of Hope Medical Center at 1 site in United States.
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 18 inclusion and 16 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: under way, not looking for participants, as of the sponsor's last update on 2 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
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 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.
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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Toxicities (time frame: 120 hours (5 days) from the last injection of plerixafor). 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 2 Sep 2026.