Status:
UNKNOWN
Comparison of Allogeneic Matched Related Haematopoietic Stem Cell Transplantation After a Reduced Intensity Conditioning Regimen With Standard of Care in Adolescents and Adults With Severe Sickle Cell Disease
Lead Sponsor:
Assistance Publique - Hôpitaux de Paris
Conditions:
Sickle Cell Disease
Eligibility:
All Genders
15-45 years
Phase:
PHASE3
Brief Summary
Although the survival of children with sickle cell disease (SCD) has dramatically improved over the last decades in the US and Europe, mortality remains high in adults. Moreover, many children and mos...
Eligibility Criteria
Inclusion
- Inclusion criteria
- SCD patients (SS/Sβ0)
- Aged :15 to 45 years
- With at least one non-SCD sibling \> 18 years from the same parental couple
- Who presented at least one of the following criteria:
- 3 VOC requiring hospitalization over one year within the past 2 years and at least a past history of an ACS
- At least 1 ACS within the past 2 years requiring transfusions
- History of ischemic stroke or cerebral/cervical arterial stenosis \> 50%
- Pulmonary hypertension defined by mean pulmonary artery pressure ≥ 25 mmHg at rest, determined by right heart catherization
- Requiring treatment with Hydroxyurea or chronic transfusion, or already treated by Hydroxyurea or transfusion program (TP) at inclusion.
- Patients already receiving chronic transfusions for VOC or ACS not responding to hydroxyurea, will be eligible, provided at least 3 VOC requiring hospitalization/year within the 2 years before initiation of chronic transfusions, and at least past history of an ACS.
- Contraception during all the study period by sirolimus for women of child bearing potential
- Signed informed consent
- Amenable to HLA typing, HSCT if an HLA-identical sibling is available.
- Patients affiliated to the French health care insurance
- Exclusion Criteria
- Performance status: ECOG scale\>1
- Pulmonary function: FEV1 et CVF \< 50% of the theorical value
- Post capillary and severe pre-capillary pulmonary hypertension with measured mean pulmonary artery pressure at rest \>35 mmHg
- Cardiac ejection fraction \< 45%
- Estimated glomerular fraction rate (GFR) \<50ml/mn /1.73m2
- Conjugate bilirubin \>50 µmole/L, cirrhosis, ALT\>4N
- Uncontrolled infection
- Known hypersensitivity of alemtuzumab
- Known hypersensitivity to murine proteins and to the following excepients: disodium edetate, polysorbate 80, potassium chloride, potassium phosphate monobasic, sodium chloride, dibasic sodium phosphate, water for injections
- Positivity for HIV
- Pregnancy or breast-feeding women
- Alloimmunization or Delayed Hemolytic Transfusion Reaction precluding red cell transfusions
Exclusion
Key Trial Info
Start Date :
October 15 2019
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
October 15 2024
Estimated Enrollment :
78 Patients enrolled
Trial Details
Trial ID
NCT04046705
Start Date
October 15 2019
End Date
October 15 2024
Last Update
September 23 2019
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