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

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ClinicalTrials.gov Identifier: NCT02212535
Recruitment Status : Completed
First Posted : August 8, 2014
Last Update Posted : December 21, 2017
Sponsor:
Information provided by (Responsible Party):
Assistance Publique - Hôpitaux de Paris

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.

Condition or disease Intervention/treatment Phase
Major Sickle Cell Syndrome of Type SS or Sβ Thalassemia Drug: Plerixafor Phase 1 Phase 2

  Show Detailed Description

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 3 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Open Study of Phase I / II Evaluating Tolerance and Efficacy of Mobilization and Collection of Peripheral Hematopoietic Stem Cells Device After a Single Injection of 0.24mg/kg of Plerixafor in Sickle Cell Patients
Actual Study Start Date : January 15, 2016
Actual Primary Completion Date : April 4, 2017
Actual Study Completion Date : September 27, 2017

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Thalassemia
Drug Information available for: Plerixafor

Arm Intervention/treatment
Experimental: Plerixafor
Adult patients affected by major sickle cell syndrome (SS or Sβ thalassemia)
Drug: Plerixafor
0.24 mg / kg / day, by subcutaneous injection, 11h before the beginning of cytapheresis




Primary Outcome Measures :
  1. Complication of disease [ Time Frame: Day 0 until Month 6 post treatment ]
    Clinical examination


Secondary Outcome Measures :
  1. Efficacy of HSC mobilization [ Time Frame: Day 1 ]
    Assessed by the Rate of circulating CD34+

  2. Evaluation of HSC collection [ Time Frame: Day 1 ]
    Apheresis



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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®

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02212535


Locations
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France
Hôpital Necker - Enfants Malades
Paris, France, 75015
Sponsors and Collaborators
Assistance Publique - Hôpitaux de Paris
Investigators
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Study Director: Jean - Antoine RIBEIL, MD, PhD Hôpital Necker - Enfants Malades, Public Hospitals of Paris

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier: NCT02212535     History of Changes
Other Study ID Numbers: HAO13017
2014-001650-42 ( EudraCT Number )
First Posted: August 8, 2014    Key Record Dates
Last Update Posted: December 21, 2017
Last Verified: December 2017
Keywords provided by Assistance Publique - Hôpitaux de Paris:
Sickle cell disease
Open monocenter study
Phase I/II
hematopoietic stem cells
Plerixafor
Additional relevant MeSH terms:
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Thalassemia
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Plerixafor octahydrochloride
Anti-HIV Agents
Anti-Retroviral Agents
Antiviral Agents
Anti-Infective Agents