EUCTR2011-001953-10-FR进行中（未招募）1 期
A phase 2/3 study of the efficacy and safety of hematopoietic stem cells transduced with Lenti D lentiviral vector for the treatment of childhood cerebral adrenoleukodystrophy (CCALD)
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
- Male
入选标准
- •1.Informed consent is obtained from a competent custodial parent or guardian with legal capacity to execute a local Institutional Review Board (IRB)/Independent Ethics Committee (IEC) approved consent. (Informed assent will be sought from capable subjects, in accordance with the directive of the IRB/IEC and with local requirements.)
- •2.Boys aged 17 years and younger, at the time of parental/guardian consent and, where appropriate, subject assent.
- •3.Active cerebral ALD as defined by:
- •a.Elevated plasma VLCFA levels, and
- •b.Active CNS disease established by central radiographic review of brain MRI demonstrating
- •i.Loes score between 0.5 and 9 (inclusive) on the 34-point scale, and
- •ii.Gadolinium enhancement on MRI of demyelinating lesions.
- •4.NFS = 1 .
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 15
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1.Receipt of an allogeneic transplant or gene therapy.
- •2.Availability of a willing 10/10 HLA-matched sibling donor (excluding female heterozygote).
- •3.Use of statins, Lorenzo’s Oil, or dietary regimens used to lower VLCFA levels. Note: subjects must discontinue use of these medications at time of consent.
- •4.Receipt of an investigational study drug or procedure within 3 months before Screening that might counfound study outcomes. Use of investigational study drugs is prohibited throughout the course of the study .
- •5.Any conditions that make it impossible to perform MRI studies (including allergies to anesthetics or contrast agents).
- •6.Hematological compromise as evidenced by:
- •a.Peripheral blood absolute neutrophil count (ANC) < 1500 cells/mm3,
- •b.Platelet count < 100,000 cells/mm3, or
- •c.Hemoglobin < 10 g/dL.
- •d.Uncorrected bleeding disorder.
- •7.Hepatic compromise as evidenced by:
- •a.Aspartate transaminase (AST) value > 2.5× the upper limit of normal (ULN) or
- •b.Alanine transaminase (ALT) value > 2.5×ULN
- •c.Total bilirubin value > 3.0 mg/dL, except if there is a diagnosis of Gilbert’s Syndrome and the subject is otherwise stable
- •8.Renal compromise as evidenced by:
- •a.Abnormal renal function (creatinine clearance < 50 mL/min)
- •9.Cardiac compromise as evidenced by:
- •a.Left ventricular ejection fraction < 40%
- •10.Immediate family member with a known or suspected Familial Cancer Syndrome (including but not limited to hereditary breast and ovarian cancer syndrome, hereditary non-polyposis colorectal syndrome, and familial adenomatous polyposis).
- •11.Clinically significant active bacterial, viral, fungal, or parasitic or prion assiociated infections.
- •12.Positive for presence of human immunodeficiency virus type 1 or 2 (HIV 1, HIV 2), hepatitis B, hepatitis C, or human T lymphotrophic virus 1 (HTLV 1). (Note that subjects who have been vaccinated against hepatitis B [hepatitis B surface antibody-positive] who are negative for other markers of prior hepatitis B infection [eg, negative for hepatitis B core antibody] are eligible. Also note that subjects who are positive for anti-hepatitis C antibody are eligible as long as they have a negative hepatitis C viral load by quantitative polymerase chain reaction [qPCR].)
- •13.Any clinically significant cardiovascular or pulmonary, or other disease or condition that would be contraindicated for any of the other study procedures.
研究者
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