EUCTR2017-002704-27-NL进行中（未招募）1 期
A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study with Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - VISION DMD
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 120
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(s) or legal guardian(s) has (have) provided written informed consent and Health Insurance Portability and Accountability Act (HIPAA) authorization, where applicable, prior to any study-related procedures; participants will be asked to give written or verbal assent according to local requirements
- •2. Subject has a centrally confirmed (by TRiNDS central genetic counselor[s]) diagnosis of DMD as defined as:
- •Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin deficiency, and clinical picture consistent with typical DMD, OR
- •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
- •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 clinical picture consistent with typical DMD;
- •3. Subject is =4 years and <7 years of age at time of enrollment in the study;
- •4. Subject weighs >13.0 kg and =39.9 kg at the Screening Visit;
- •5. Subject is able to walk independently without assistive devices;
- •6. Subject is able to complete the Time to Stand Test (TTSTAND) without assistance in <10 seconds, as assessed at the Screening Visit;
- •7. 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];
- •8. 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 local laboratory at the Screening Visit;
- •9. Subject is able to swallow tablets, as confirmed by successful test aswallowing of placebo tablets during the Screening Period; and
- •10. Subject and parent(s)/guardian(s) 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: 120
- •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 a history of primary hyperaldosteronism;
- •6. Subject has evidence of symptomatic cardiomyopathy [Note: Asymptomatic cardiac abnormality on investigation would not be exclusionary];
- •7. 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 indication other than DMD 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 glucocorticoids prescribed for an indication other than DMD are permitted if last use is at least 4 weeks prior to first dose of study medication or are administered at stable dose beginning at least 4 weeks prior to first dose of study medication, and are anticipated to be used at the stable dose regimen for the duration of the study];
- •8. Subject has an allergy or hypersensitivity to the study medication or to any of its constituents;
- •9. Subject has used idebenone within 4 weeks prior to the first dose of study medication;
- •10. Subject has severe behavioural or cognitive problems that preclude participation in the study, in the opinion of the Investigator;
- •11. 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;
- •12. Subject is taking (or has taken within 4 weeks prior to the first dose of study medication) herbal remedies and supplements which can impact muscle strength and function (e.g. Co-enzyme Q10, Creatine, Proglandine etc);
- •13. Subject is taking (or has taken within 3 months prior to the first dose of study medication) any medication indicated for DMD, including Exondys51 and Translarna;
- •14. Subject is currently taking any other investigational drug or has taken any other investigational drug within 3 months prior to the first dose of study medication; or
- •15. Subject has previously been enrolled in the study.
- •Note: Any parameter/test may be repeated at the Investigator’s discretion during Screening to determine 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 injury, or if ineligible due to negative anti-varicella IgG antibody test result.
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