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

• Status: Completed
• Trial end date: 2017-09-27
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Assistance Publique - Hôpitaux de Paris
• Treatments: JM 3100; Plerixafor

Criteria

Inclusion Criteria:

• 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

Exclusion Criteria:

• 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)

• Pulmonary High blood pressure confirmed by catheterization

• Renal with calculated clearance with of creatinine < 30%

• Severe iron overload with abnormal cardiac T2* MRI < 10 ms

• Lung with level of oxygen saturation <90% (outside times of crisis) or DLCO < 60% in the absence of infection

• Current participation in another interventional clinical trial

• Polynuclear superior or egal at 10.000/mm3 to the base state without infection or inflammatory syndrome

• Patient under Medical Assistance State

• Patient under guardianship

• Hypersensitivity to plerixafor or any excipient contained in MOZOBIL®