Assessment of Tolerance of Mobilizing Peripheral Hematopoietic Stem Cells by Plerixafor in Sickle Cell Patients

NCT02212535 | Completed Phase 1

• 2 other identifiers: HAO130172014-001650-42
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to assess the tolerance and efficacy of mobilizing hematopoietic stem cells after a single injection of plerixafor (0.24mg/kg) in 3 adult patients (or 5, if results of the first 3 patients are not reproducible) affected by sickle cell disease.

Conditions

Major Sickle Cell Syndrome of Type SS or Sβ Thalassemia

Keywords

Sickle cell disease; Open monocenter study; Phase I/II; hematopoietic stem cells; Plerixafor

Outcome Measures

Primary Outcomes (1)

• Complication of disease

  Clinical examination

  Day 0 until Month 6 post treatment

Secondary Outcomes (2)

• Efficacy of HSC mobilization

  Day 1

• Evaluation of HSC collection

  Day 1

Study Arms (1)

Plerixafor

EXPERIMENTAL

Adult patients affected by major sickle cell syndrome (SS or Sβ thalassemia)

Drug: Plerixafor

Interventions

Plerixafor DRUG

0.24 mg / kg / day, by subcutaneous injection, 11h before the beginning of cytapheresis

Plerixafor

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age ≥ 18 years old
• Affiliated or beneficiary of a health insurance regimen
• For women of childbearing age, not pregnant and use effective contraception during the entire participation in research.
• Affected by a major sickle cell SS or Sβ thalassemia whose diagnosis must have been confirmed by a study of hemoglobin.
• Have the potential indication of allogenic bone marrow and don't have identical-HLA siblings.
• Have a general condition corresponding to a functional index of Lansky ≥ 80%
• Have been treated and followed for at least the previous two years in a specialized center where they got a full assessment of their disease
• In addition to the general eligibility criteria, sickle cell patients must have one or more of the following risk factors despite hydroxyurea treatment with for at least 4 months, except in cases of bad tolerance to hydroxyurea:
• Severe recurrent vaso-occlusive episodes of duration \> 48 hours or having required hospitalization for more than 24 hours (defined by at least two episodes during the previous year or in the year preceding the setting up of regular transfusion protocol)
• And /or recurrent Acute Chest Syndrome (at least 2 episodes) - defined by the presence of a new pulmonary infiltration involving at least one complete pulmonary segment (but excluding atelectasis) with chest pain and/or fever (\> 38 5), and / or tachypnea, and / or wheezing or cough without infectious syndrome
• Osteonecrosis of 2 or more joints.
• Anti-erythrocyte alloimmunization (\>2 antibodies).
• Presence of sickle cell cardiomyopathy documented by Doppler echocardiography.
• Informed and signed consent

You may not qualify if:

• Patient who to his knowledge and that of the investigator, is unable to follow the visits required by the protocol
• Any form of disorder that, according to the investigator, may compromise the ability of the patient to give an informed written consent and / or to conform to all required procedures of the study.
• Positive serology for HIV-1/2, HTLV-1/2, syphilis, HCV and / or HBsAg
• Bacterial, viral, fungal or parasitic active infection with clinical signs requiring hospitalization for more than 24 hours
• Recurring Malaria
• Personal history of cancer, myeloproliferative hematopathy or immune deficiency
• Cerebral vasculopathy highlighted by transcranial Doppler ultrasound or pathological MRI
• Heart failure and / or heart rhythm disorder and / or myocardial infarction
• History of allogeneic graft of hematopoietic stem cells
• Diagnosis of a psychiatric disorder that could compromise his/her ability to participate in the study
• Current Pregnancy or breastfeeding
• For women of childbearing potential no use effective contraception throughout the whole treatment duration
• Major dysfunction of :
• Liver : transaminases superior or egal at 3 times more than normal
• Heart with alteration of the left ventricular ejection fraction (LVEF)

Sponsors & Collaborators

• Assistance Publique - Hôpitaux de Paris: lead
• URC-CIC Paris Descartes Necker Cochin: collaborator

Study Sites (1)

Hôpital Necker - Enfants Malades

Paris, 75015, France

Location

Related Publications (1)

• Lagresle-Peyrou C, Lefrere F, Magrin E, Ribeil JA, Romano O, Weber L, Magnani A, Sadek H, Plantier C, Gabrion A, Ternaux B, Felix T, Couzin C, Stanislas A, Treluyer JM, Lamhaut L, Joseph L, Delville M, Miccio A, Andre-Schmutz I, Cavazzana M. Plerixafor enables safe, rapid, efficient mobilization of hematopoietic stem cells in sickle cell disease patients after exchange transfusion. Haematologica. 2018 May;103(5):778-786. doi: 10.3324/haematol.2017.184788. Epub 2018 Feb 22.

  PMID: 29472357 BACKGROUND

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

• Jean - Antoine RIBEIL, MD, PhD

  Hôpital Necker - Enfants Malades, Public Hospitals of Paris

  STUDY DIRECTOR

Study Design

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

Study Record Dates

• First Submitted: August 6, 2014
• First Posted: August 8, 2014
• Study Start: January 15, 2016
• Primary Completion: April 4, 2017
• Study Completion: September 27, 2017
• Last Updated: September 5, 2025

Record last verified: 2025-08

Locations

FR (1)