EUCTR2016-004262-26-GB进行中（未招募）1 期
VBP15-002 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 MuscularDystrophy (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. Participant’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. Participant 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;
- •Participant is = 4 years and < 7 years of age at time of enrollment in the study;
- •4. Participant is able to complete the Time to Stand Test (TTSTAND) without assistance, as assessed at the Screening
- •or 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.Participant 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. Participant and caregiver 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: 48
- •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. Participant has current or history of major renal or hepatic impairment, diabetes mellitus or immunosuppression;
- •2. Participant has current or history of chronic systemic fungal or viral infections;
- •3. Participant has had an acute illness within 4 weeks prior to the first dose of study medication;
- •4. Participant 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. Participant has evidence of symptomatic cardiomyopathy [Note: Asymptomatic cardiac abnormality on investigation would not be exclusionary];
- •6. Participant 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. Participant has used idebenone within 4 weeks prior to the first dose of study medication;
- •8. Participant has an allergy or hypersensitivity to the study medication or to any of its constituents;
- •9. Participant has severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the
- •Investigator;
- •10.Participant 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.Participant 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.Participant 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.
研究者
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