EUCTR2018-001145-14-FR进行中（未招募）1 期
A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfan and Fludarabine in Subjects =17 Years of Age With Cerebral Adrenoleukodystrophy (CALD)
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 20
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
入选标准
- •1. Informed consent is obtained from a competent custodial parent or guardian with legal capacity to execute a local IRB/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. Males 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 VLCFA values, and
- •b. Active central nervous system (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. Neurologic Function Score (NFS) =1.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 20
- •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. Prior receipt of an allogeneic transplant or gene therapy.
- •2. Use of statins, Lorenzo’s Oil, or dietary regimens used to lower VLCFA levels.
- •3. Receipt of an investigational study drug or procedure within 3 months before Screening that might confound study outcomes. Use of investigational study drugs is prohibited throughout the course of the study.
- •4. Any conditions that make it impossible to perform MRI studies (including allergies to anesthetics or contrast agents).
- •5. Hematological compromise as evidenced by:
- •a. Peripheral blood ANC count <1500 cells/mm3,
- •b. Platelet count <100,000 cells/mm3, or
- •c. Hemoglobin <10 g/dL.
- •d. Uncorrected bleeding disorder.
- •6. Hepatic compromise as evidenced by:
- •a. Aspartate transaminase (AST) value >2.5 × ULN,
- •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.
- •7. Baseline estimated glomerular filtration rate <70 mL/min/1.73 m2.
- •8. Cardiac compromise as evidenced by left ventricular ejection fraction <40%.
- •9. Immediate family member with a known or suspected Familial Cancer Syndrome.
- •10. Clinically significant uncontrolled, active bacterial, viral, fungal, parasitic, or prion associated infection.
- •11. Positive for HIV, hepatitis B or C virus, or human T lymphotrophic virus 1 (HTLV-1).
- •12. Absence of adequate contraception for fertile subjects.
- •13. Any contraindications to the use of G-CSF or plerixafor during the mobilization of hematopoietic stem cells, and any contraindications to the use of busulfan or fludarabine, including known hypersensitivity to the active substances or to any of the excipients in their formulations.
研究者
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