EUCTR2018-001145-14-NL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
- 35
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 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: 35
- •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, and either
- •b. Platelet count <100,000 cells/mm3, or
- •c. Hemoglobin <10 g/dL.
- •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. Any clinically significant cardiovascular, hematological or pulmonary disease, or other disease or condition that would be contraindicated for any of the
- •other study procedures.
- •13. Absence of adequate contraception for fertile subjects.
- •14. 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.
- •15. Known hypersensitivity to protamine sulfate.
研究者
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