Tiny study tests drug to harvest stem cells in sickle cell patients
NCT ID NCT02212535
First seen Jun 25, 2026 · Last updated Jun 26, 2026 · Updated 1 time
Summary
This early-phase study tested whether a single injection of plerixafor could safely and effectively mobilize stem cells in 3 adults with severe sickle cell disease who lacked a matched sibling donor. The goal was to see if enough stem cells could be collected for a possible future transplant. The study was very small and focused only on the collection step, not the transplant itself.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- plerixafor
- What this could lead to
- If successful, this could show that plerixafor safely mobilizes stem cells in sickle cell patients, potentially enabling future stem cell transplants for those without a matched donor.
- What could go wrong
- This is a very early, tiny study (only 3 patients) testing only the stem cell collection step, not the transplant itself. It may not lead to a treatment, and risks include complications from the procedure.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
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3 people
The number who actually took part.
- Started
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Jan 2016
- Finished
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Sep 2017
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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®
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Hôpital Necker - Enfants Malades
Paris, 75015, France