EUCTR2018-004009-22-FR进行中（未招募）1 期
A Randomized, Double-blind, Placebo-controlled, Efficacy and Safety Study of WVE-210201 in Ambulatory Patients with Duchenne Muscular Dystrophy
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 150
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. Patient and/or parent or legal guardian must have the ability and be willing to provide written informed consent prior to any study-related procedures.
- •2. Diagnosis of DMD based on clinical phenotype with increased serum creatine kinase.
- •3. Documented mutation in the Dystrophin gene associated with DMD that is amenable to exon 51 skipping.
- •4. Ambulatory male, able to walk independently for at least 10 meters in 20 seconds or less at the time of Screening visit
- •5. Age of =5 and =12 years at time of randomization
- •6. Willing and able to comply with scheduled visits, drug administration plan, laboratory tests, study restrictions, and all study procedures, including undergoing the biopsy procedures.
- •7. Stable pulmonary and cardiac function, as measured by:
- •a. Reproducible percent predicted forced vital capacity (FVC) =50%
- •b. Left ventricular ejection fraction (LVEF) >55% in patients <10 years of age and >45% in patients =10 years of age, as measured (and documented) by echocardiogram.
- •8. Currently on a stable corticosteroid therapy regimen, defined as: initiation of systemic corticosteroid therapy occurred =6 months prior to Screening, and no changes in dose =3 months prior to Screening visit.
- •9. Adequate deltoid muscle at baseline to perform open muscle biopsies
- •10. Sexually mature males must be willing to use contraception for the duration of the study, if the patient is sexually active.
- •11. Patient and caregivers must agree not to post any study-related information on social media.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 150
- •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. Clinically significant medical finding on the physical examination other than DMD that, in the judgment of the Investigator, will make the patient unsuitable for participation in, and/or completion of the study procedures.
- •2. Other prior or ongoing medical conditions including:
- •a. Acute illness within 4 weeks of the initial Screening visit;
- •b. Abnormal physical findings, other than those associated with musculoskeletal findings attributable to DMD
- •3. Laboratory abnormality, that, in the Investigator's opinion, could adversely affect the safety of the patient, make it unlikely that the course of treatment or follow-up would be completed, or impair the assessment of study results. These include, but are not limited to:
- •a. Renal insufficiency;
- •b. Impaired hepatic function glutamate dehydrogenase (GLDH) = 2.5x upper limit of normal (ULN) and bilirubin = 2x ULN (or International Normalized Ratio [INR] = 1.5x ULN);
- •c. Activated partial thromboplastin time (aPTT) values above ULN;
- •d. Platelet count < lower limit of normal (LLN).
- •4. Documented positive hepatitis B surface antigen or hepatitis C antibody test.
- •5. Known to be positive for human immunodeficiency virus (HIV).
- •6. Severe mental retardation and/or behavioral problems that, in the opinion of the Investigator, could prohibit participation in this study.
- •7. Cardiac insufficiency:
- •a. Severe cardiomyopathy that, in the opinion of the Investigator, prohibits participation in this study; however, cardiomyopathy that is managed by ACE inhibitors or beta blockers is acceptable provided the patient meets the LVEF inclusion criterion
- •b. Any other evidence of clinically significant structural or functional heart abnormality
- •c. A cardiac troponin I value > 0.2 ng/mL
- •8. Need for daytime mechanical or non-invasive ventilation OR anticipated need for daytime mechanical or non-invasive ventilation within the next year, in the opinion of the Investigator. Nighttime non-invasive ventilation is permitted.
- •9. Changes in nutritional or herbal supplements or concomitant medications within 1 month prior to Screening visit or plans to modify (dose or regimen) during the study.
- •10. Currently on anticoagulants or drugs that are known to significantly increase the risk of bleeding, such as NSAIDs and heparin.
- •11. Received prior treatment with drisapersen or with an investigational peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO).
- •12. Received prior treatment with WVE-210201.
- •13. Received prior treatment with gene therapy for DMD
- •14. Received treatment with ataluren or eteplirsen within the 14 weeks prior to the planned Baseline biopsy collection.
- •15. Received any investigational drug within 3 months or 5 half-lives, whichever is longer prior to the planned baseline biopsy collection.
- •16. Known hypersensitivity to any oligonucleotide, as demonstrated by a systemic allergic reaction such as changes in pulse, blood pressure, breathing function, etc.
- •17. Parent or legal guardian is directly or indirectly involved in the conduct and administration of this study as an Investigator, sub-investigator, study coordinator, or other study staff member, or the patient is a first-degree family member, significant other, or relative residing with one of the above persons involved directly or indirectly in the study.
研究者
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