EUCTR2017-001684-21-GB进行中（未招募）1 期
Phase I/II, Open-Label, Dose Escalation Study of the Safety, Pharmacokinetics, and Pharmacodynamics of NV1205 in Pediatric Male Subjects with Childhood Cerebral Adrenoleukodystrophy (CCALD)
eurov Acquisition LLC0 个研究点目标入组 25 人开始时间: 2017年10月26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
- 25
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. Males =4 and <18 years of age
- •2. CCALD diagnosis confirmed by genetic testing
- •a. Genetic testing for CCALD diagnosis is not performed as part of the study screening assessments, and historic record is expected to be available to verify this requirement. However, if prior genetic verification of CCALD diagnosis was not done, or is not available, genetic testing must be performed to verify this eligibility requirement prior to initiating drug
- •3. Loes score of >0 and =15
- •4. Not eligible for HSCT, does not have access to HSCT, or opts not to receive HSCT, and is not expected to receive HSCT
- •5. VLCFA levels above normal limit in plasma or blood spot test
- •a. For subjects not on VLCFA lowering agents such as Lorenzo’s oil, a test within 3 months of enrollment is acceptable.
- •b. Patients on VLCFA lowering agents such as Lorenzo’s oil must stop the medication and have high VLCFA levels before enrollment
- •6. Signed informed consent by subject’s parent(s)/legally acceptable representative(s). In addition, signed children’s assent form according to local requirements.
- •7. Use of adequate contraception for subjects of reproductive potential during the study and for 93 days after the last dose of the study drug
- •a. Adequate contraception include vasectomy, true abstinence when this is in line with the preferred and usual lifestyle of the subject or consistent use of condom by subjects, or the use of effective contraceptive methods such as hormonal contraception, intrauterine device or hormone-releasing system, or bilateral tubal ligations by subject’s partners. Periodic abstinence (calendar, symptothermal, post-ovulation methods), withdrawal (coitus interruptus), spermicides only, and lactational amenorrhoea method are not acceptable methods of contraception.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 6
- •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. Electrocardiographic (ECG) evidence of a previous myocardial infarction (MI) or ECG changes that likely represent cardiac ischemia, an unstable cardiac condition within the last 3 months (e.g., MI, revascularization, congestive heart failure, acute coronary syndrome), or ventricular tachycardia, unexplained syncope or syncope due to a cardiac etiology, long QT syndrome, or a family history of sudden cardiac death
- •2. History of cerebrovascular accident
- •3. QTcF >450 msec; significant ST-T wave changes that make interpretation of the QT interval challenging. PR >240 msec; resting heart rate <50 or >100 beats per minute
- •4. History of cancer within the past 5 years
- •5. Expected to initiate a major dietary change known to affect levels of VLCFA
- •6. Use of prohibited medications, including QTc prolonging drugs, oncologic chemotherapy for cancer treatment, metformin, statins, or Lorenzo's Oil within 14 days of first dose or at least 5.5 known half-lives of the relevant medication
- •7. Use of any investigational drug or procedures within 30 days of first dose of NV1205
- •8. Evidence of cataract development
- •9. Other significant medical conditions judged by the Investigator to preclude entry
- •10. HSCT recipient or receiving conditioning treatment for HSCT
研究者
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