NL-OMON53482尚未招募2 期
AN OPEN-LABEL, MULTI-CENTRE STUDY TO EVALUATE THE LONG-TERM SAFETY AND TOLERABILITY OF REN001 IN SUBJECTS WITH PRIMARY MITOCHONDRIAL MYOPATHY (PMM) - Open label study of REN001 in PMM subjects
适应症
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 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
入排标准
- 年龄范围
- 18 至 99（—）
入选标准
- •1. mtDNA-PMM subjects:Completed treatment in the STRIDE study or was
- •participating in Study REN001-101, and in the opinion of the Investigator and
- •the Sponsor have been compliant with the study requirements.
- •nDNA-PMM subjects: Subjects aged 18 years or older with known nuclear (nDNA)
- •variants with a major muscle phenotype consisting of objective myopathy with
- •poor exercise tolerance. Proof of pathogenicity must be provided. Must be able
- •to walk at least 100m in the screening 12MWT and the limitations in walk test
- •must be primarily due to the energy deficit and not due to ataxia or any other
- •For subjects under 25 years old only: confirmation of bone growth plate closure
- •by wrist radiograph.
- •2. Have PMM which continues to be primarily characterized by exercise
- •intolerance or active muscle pain.
- •3. Willing and able to swallow gelatin capsules.
- •4. Concomitant medications (including supplements) intended for the treatment
- •of PMM or other co-morbidities likely to remain stable throughout participation
- •in the study where clinically possible.
- •5. Signed and dated informed consent document indicating that the subject has
- •been informed of all pertinent aspects of the study.
- •6. Females should be either of non-child-bearing potential or must agree to use
- •highly effective methods of contraception from baseline through to
- •approximately 30 days after last dose of study drug. Males with partners who
- •are WOCBP must also use contraception from baseline through to 14 weeks after
- •last dose of study drug.
排除标准
- •1. Anticipated to need a PPAR agonist other than REN001 during the study.
- •2. Anticipated to need drugs during the study with a narrow therapeutic index
- •and Breast Cancer Resistant Protein (BCRP) mediated absorption, distribution,
- •metabolism and excretion (ADME).
- •3. Intent to donate blood, or blood components during the study or within one
- •month after completion of the study.
- •4. Current drug dependency. Use of opiates/cannabis for medical reasons is
- •acceptable with prescription evidence or at the Investigator*s discretion.
- •5. Current alcohol dependency.
- •6. Any medical or psychiatric condition or laboratory abnormality that may
- •increase the risk associated with study participation or interfere with the
- •interpretation of study results and, in the judgment of the Investigator and
- •Medical Monitor, would make the subject inappropriate for entry into this
- •7. Pregnant or nursing females.
研究者
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