Safety of Blood Stem Cell Mobilization With Plerixafor in Patients With Sickle Cell Disease

NCT03664830 | Active Not Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: 17426
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

Brief 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

Conditions

Sickle Cell Disease

Keywords

plerixafor; sickle cell disease; mobilization; hematopoietic stem cell; gene therapy

Outcome Measures

Primary Outcomes (1)

• Toxicities

  Scored using the Common Terminology Criteria for Adverse Events (CTCAE) of the National Cancer Institute (NCI) version 4.03

  120 hours (5 days) from the last injection of plerixafor

Secondary Outcomes (1)

• Stem cell mobilization feasibility

  6 hours after the first injection of plerixafor.

Study Arms (1)

Plerixafor

EXPERIMENTAL

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

Drug: Plerixafor

Interventions

Plerixafor DRUG

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

Plerixafor

Eligibility Criteria

• Age: 18 Years - 40 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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)
• 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;

You may not qualify if:

• 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;
• 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;

Sponsors & Collaborators

• City of Hope Medical Center: lead

Study Sites (1)

City of Hope Medical Center

Duarte, California, 91010, United States

Location

MeSH Terms

Conditions

Anemia, Sickle Cell

Interventions

plerixafor

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Leo Wang, MD

  City of Hope Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Each SCD research participant will receive up to two subcutaneous injections of plerixafor (starting dose level: 240 µg/kg/dose)

Study Record Dates

• First Submitted: September 7, 2018
• First Posted: September 11, 2018
• Study Start: September 19, 2018
• Primary Completion (Estimated): August 3, 2026
• Study Completion (Estimated): August 3, 2026
• Last Updated: September 16, 2025

Record last verified: 2025-09

Locations

US (1)