NL-OMON54719已完成3 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) - ALD-104
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 6
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 2 至 17（—）
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. 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 very long chain fatty acids (VLCFA) values, and
- •b. Active central nervous system (CNS) disease established by central
- •radiographic review of brain magnetic resonance imaging (MRI) demonstrating
- •i. Loes score between 0.5 and 9 (inclusive) on the 34-point scale, and
- •ii. Gadolinium enhancement (GdE) on MRI of demyelinating lesions.,
- •4. Neurologic Function Score (NFS) <=1.
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
- •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 absolute neutrophil count (ANC) count <1500 cells/mm3, and
- •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 × upper limit of normal (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 human immunodeficiency virus type 1 or 2 (HIV-1, HIV-2);
- •hepatitis B virus (HBV); hepatitis C virus (HCV); human T lymphotrophic virus 1
- •(HTLV-1). (Note that subjects who have been vaccinated against HBV [positive
- •for HBV surface antibodies] who are negative for other markers of prior HBV
- •infection [e.g., negative for HBVhepatitis B core antibody {HBVc Ab]}] are
- •eligible. Subjects with past exposure to HBV [HBcAb [HBc antibodies {Ab}
- •positive and/or hepatitis B e-antigen antibody {HBeAb}-positive] are also
- •eligible for the study provided they have a negative test for HBV
- •deoxyribonucleic acid [DNA.]. Also note that subjects who are positive for
- •anti-hepatitis C Ab are eligible as long as they have a negative hepatitis C
- •viral load).
- •12. Any clinically significant cardiovascular, haematological 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. Male subjects and
- •their female partners are required to use two different effective methods of
- •contraception from Screening through at least 6 months after eli-cel infusion.
- •If subjects are truly sexually abstinent (where true sexual abstinence is
- •defined as being in line with the preferred and usual lifestyle of the
- •subject), no second method is required.
- •14. Any contraindications to the use of G-CSF or plerixafor during the
- •mobilization of hematopoietic stem cells (HSCs), 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 sulphate
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