EUCTR2019-000654-59-ES进行中（未招募）1 期
An exploratory, open-label, multicenter study in male pediatric patients with cerebral X-linked Adrenoleukodystrophie (cALD) to assess the effect of MIN-102 treatment on the progression of cerebral lesions.
Minoryx Therapeutics S.L.0 个研究点目标入组 10 人开始时间: 2019年4月30日最近更新:
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 10
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. Written informed consent by parent/legal guardian, or authorized legal representative to participate in the study
- •2. Males aged =2 and =12 years with a diagnosis of X-linked ALD based on genetic testing
- •3. White matter involvement as determined by cerebral MRI lesions without Gd enhancement at baseline (Population 1), or with Gd enhancement at baseline (Population 2).
- •4. Major Functional Disabilities (MFD) score of 0, as determined by key measures in the Neurological Function Scale (NFS)
- •5. Baseline Loes score >0 and =10
- •6. Baseline GIS =2
- •7. No signs or symptoms of adrenal insufficiency and morning cortisol and aldosterone levels within normal laboratory ranges for age, or appropriate steroid replacement if adrenal insufficiency is present. A history of adrenal insufficiency is not exclusionary if the foregoing is currently met.
- •8. Glycated hemoglobin (HbA1c) within normal range
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 10
- •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. Other chronic neurological disease
- •2. Known intolerance to pioglitazone or other thiazolidinediones
- •3. Use of pioglitazone or other thiazolidinediones within the past 6 months prior to screening
- •4. Use of biotin at a daily dose of >50 mg per day within the past 3 months prior to screening
- •5. Current participation in another interventional clinical study or participation in such a study within 6 months prior to screening
- •6. Previous HSCT
- •7. Current use of immunosuppressant medication, excluding corticosteroids
- •8. Requirement for a prohibited concomitant medication
- •9. Previous or current history of bladder polyps, bladder cell hyperplasia, or cancer (other than successfully treated basal cell carcinoma)
- •10. Previous or current history of congestive heart failure
- •11. Clinically significant anemia with hemoglobin <10 g/dL
- •12. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) level >2 times the ULN or total bilirubin >1.5 times the ULN (unless due to Gilbert's syndrome)
- •13. Moderate or severe hepatic impairment (groups B and C according to Child-Pugh classification)
- •14. Chronic kidney disease stage 3 or higher (according to chronic kidney disease staging by The Renal Association)
- •15. Pulmonary disease or cardiac disease of sufficient severity to limit participation in the study and/or completion of study procedures
- •16. Reduced left-ventricular ejection fraction or other clinically significant cardiac abnormalities on echocardiogram that, in the investigator's opinion, could predispose the subject to volume overload or its attendant consequences
- •17. Contraindication to MRI procedure, such as presence of paramagnetic materials (aneurysm clips, pacemaker, intraocular metal, cochlear implant) in the body
- •18. Conditions that could modify the absorption of the study drug
- •19. Inability or unwillingness of parent/legal guardian or subject to comply with the study procedures
- •20. Other medical, neurologic, psychiatric, or social condition that, in the opinion of the investigator, is likely to unfavorably alter risk-benefit of study participation, confound interpretation of safety or efficacy results, or interfere with the satisfactory completion of study requirements
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