EUCTR2014-003572-23-FR进行中（未招募）1 期
Allogeneic Stem Cell Transplantation of CordIn™, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients with Hemoglobinopathies
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 15
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1.Patients must be 2–25 years of age and at least 10kg
- •2.Patient is a candidate for allogeneic SCT for treatment of SCD or thalassemia:
- •a.Patient must have clinically severe SCD(eg. SS, SC or SBeta0 Thal) with at least one of the following clinical complications:
- •Recurrent painful events(at least 3 in the 2 years prior to enrollment) that cannot be explained by other causes. Pain may occur in typical sites associated with vaso-occlusive painful events and cannot be explained by causes other than sickle cell disease. Pain lasts at least 4 hours and requires parenteral narcotic treatment,equianalgesic dose of oral narcotics or parenteral nonsteroidal anti-inflamrnatory drugs. These painful events may be treated in any setting,but events managed at home will be considered only if there is documentation of the event in a clinical record that may be reviewed by an investigator. These events must occur despite adequate supportive care measures.
- •Acute chest syndrome with at least two episodes with the development of a new infiltrate on chest radiograph and/or having a perfusion defect demonstrable on a lung radioisotope scan within the past two years that required hospitalization, oxygen therapy, and RBC transfusion. These episodes must occur despite adequate supportive care measures.
- •Any combination of painful events and acute chest syndrome episodes that total three events within the two years before transplantation.
- •Any clinically significant neurologic event (stroke or hemorrhage) or any neurologic defect lasting more than 24 hours.
- •Patients on chronic PRBC transfusion therapy, defined as receiving 8 or more transfusions per year for > 1 year to prevent vaso-occlusive clinical complications (e.g. pain, stroke, and acute chest syndrome)
- •Abnormal cerebral MRI and abnormal cerebral MRA.
- •Abnormal Transcranial Doppler (TCD), as defined by a TCD velocity that exceeds 200 cm/sec by the non-imaging technique (or TCD measurement of >185 cm/sec by the imaging technique) measured at a minimum of two separate occasions one month or more apart
- •b.Patient must have thalassemia major requiring regular RBC transfusions
- •3.Patients must have a partially HLA-matched CBU. The unit must be HLA-matched at 4-6/6 HLA class I (HLA-A & HLA-B, low resolution) and II (HLA-DRB1, high resolution) loci with the subject. For patients weighing = 25 kg, the CBU must have a pre-cryopreserved, total CD34+ cell dose of =10x10^6 as well as a pre-cryopreserved total nucleated cell dose of =1.8x10^9. For patients weighing < 25 kg, the CBU must have a pre-cryopreserved, total CD34+ cell dose of =7x10^6 as well as a pre-cryopreserved total nucleated cell dose of =1.6x10^9.
- •4.The CBU will have undergone volume reduction (both plasma and red blood cell depletion) prior to cryopreservation.
- •5.Patients must have autologous stem cells harvested from bone marrow as a backup in case of graft failure.
- •6.Patients’ Performance score =70% by Lansky or Karnofsky performance status scale
- •7.Patient has sufficient physiologic reserves including:
- •Cardiac: Left ventricular ejection fraction (LVEF) > 50% by echocardiogram radionuclide scan or cardiac MRI; or LV shortening fraction > 26%.
- •Pulmonary: Pulse oximetry with a baseline O2 saturation of = 85% is required for all patients, DLCO > 60% of predicted for age (corrected for hemoglobin) for patients in whom pulmonary function testing can be performed.
- •Renal: Serum creatinine = 1.5 x upper limit of normal for age and GFR >
排除标准
- •1.Evidence of uncontrolled bacterial, viral or fungal infections or severe concomitant diseases, which in the judgment of the Principal Investigator, indicate that the patient could not tolerate transplantation
- •2.Evidence of HIV infection or HIV positive serology
- •3.Evidence of active Hepatitis B, Hepatitis C or EBV as determined by serology or PCR
- •4.Pregnancy, as indicated by a positive serum human chorionic gonadotrophin (HCG) test, or lactation
- •5.Patients with 10/10 or 9/10 HLA-matched related family donor or unrelated donor able to donate
- •6.Severe alloimmunization with inability to guarantee a supply of adequate PRBC donors
- •7.Evidence of anti-HLA antibodies to the selected CordIn™ CBU (MFI>1000)
- •8.Prior allogeneic hematopoietic stem cell transplant within last 12 months .
- •9.Allergy to bovine, Gentamicin, or to any product which may interfere with the treatment
- •10.Psychologically incapable of undergoing bone marrow transplant (BMT) with associated strict isolation or documented history of medical non-compliance and/or psychiatric illness and/or social situations that would limit compliance with study requirements
- •11.Enrolled in another clinical trial or received an investigational treatment during the last 30 days, unless approved by Sponsor
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