EUCTR2016-004262-26-SE进行中（未招募）1 期
A Phase IIa, Open-Label, Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD)
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 48
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. Subject’s parent or legal guardian has provided written informed consent/Health Insurance Portability and Accountability Act (HIPAA) authorization prior to any study-related procedures;
- •2. Subject has a confirmed (by Central Genetic Counselor) diagnosis of DMD as defined as:
- •a. Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin deficiency, and clinical picture consistent with typical DMD, OR
- •b. Identifiable mutation within the DMD gene (deletion/duplication of one or more exons) where reading frame can be predicted as ‘out-of-frame’, and clinical picture consistent with typical DMD, OR
- •c. Complete dystrophin gene sequencing showing an alteration (point mutation, duplication, other) that is expected to preclude production of the dystrophin protein (i.e. nonsense mutation, deletion/duplication leading to a downstream stop codon), with a typical clinical picture of DMD;
- •3. Subject is = 4 years and < 7 years of age at time of enrollment in the study;
- •4. Subject is able to complete the Time to Stand Test (TTSTAND) without assistance, as assessed at the Screening and Baseline Visits;
- •5. Clinical laboratory test results are within the normal range at the Screening Visit, or if abnormal, are not clinically significant, in the opinion of the Investigator. (Note: Serum gamma glutamyl transferase [GGT], creatinine, and total bilirubin all must be = upper limit of the normal range at the Screening Visit);
- •6. Subject has evidence of chicken pox immunity as determined by presence of IgG antibodies to varicella, as documented by a positive test result from the testing laboratory at the Screening Visit; and
- •7. Subject and parent/guardian are willing and able to comply with scheduled visits, study drug administration plan, and study procedures.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 5
- •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. Subject has current or history of major renal or hepatic impairment, diabetes mellitus or immunosuppression;
- •2. Subject has current or history of chronic systemic fungal or viral infections;
- •3. Subject has had an acute illness within 4 weeks prior to the first dose of study medication;
- •4. Subject has used mineralocorticoid receptor agents, such as spironolactone, eplerenone, canrenone (canrenoate potassium), prorenone (prorenoate potassium), mexrenone (mexrenoate potassium) within 4 weeks prior to the first dose of study medication;
- •5. Subject has evidence of symptomatic cardiomyopathy. [Note: Asymptomatic cardiac abnormality on investigation would not be exclusionary];
- •6. Subject is currently being treated or has received previous treatment with oral glucocorticoids or other immunosuppressive agents. [Notes: Past transient use of oral glucocorticoids or other oral immunosuppressive agents for no longer than 3 months cumulative, with last use at least 3 months prior to first dose of study medication, will be considered for eligibility on a case-by-case basis. Inhaled and/or topical corticosteroids prescribed for an indication other than DMD are permitted but must be administered at stable dose for at least 3 months prior to study drug administration];
- •7. Subject has used idebenone within 4 weeks prior to the first dose of study medication;
- •8. Subject has an allergy or hypersensitivity to the study medication or to any of its constituents;
- •9. Subject has severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the Investigator;
- •10. Subject has previous or ongoing medical condition, medical history, physical findings or laboratory abnormalities that could affect safety, make it unlikely that treatment and follow-up will be correctly completed or impair the assessment of study results, in the opinion of the Investigator;
- •11. Subject is taking any other investigational drug currently or has taken any other investigational drug within 3 months prior to the start of study treatment; or
- •12. Subject has previously been enrolled in the study.
- •Note: Any parameter/test may be repeated at the Investigator’s discretion during Screening and/or Day 1 to determine sustainability and reproducibility. In addition, subjects may be rescreened if ineligible due to a transient condition which would prevent the subject from participating, such as an upper respiratory tract infection, or if ineligible due to negative anti-varicella IgG antibody test result.
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