EUCTR2021-003471-34-DE进行中（未招募）1 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
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 200
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
- All
入选标准
- •1.mtDNA-PMM subjects: Completed treatment in the STRIDE study or participated in Study REN001-101, and in the opinion of the Investigator and Sponsor had been compliant with the study requirements.
- •nDNA-PMM subjects: Subjects aged 18 years or older with known
- •nuclear (nDNA) pathogenic 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 condition. For subjects under 25 years old only: confirmation of bone growth plate closure by wrist radiograph.
- •2.Have PMM which continues to be primarily characterised by exercise intolerance or active muscle pain.
- •3.Willing and able to swallow the REN001 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. If subjects are transferred to a commercial supply of REN001 they must be advised to adhere to the contraceptive requirements.
- •Are the trial subjects under 18? no
- •Number of subjects for this age range: 0
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 180
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 20
排除标准
- •1.Anticipated to need a peroxisome proliferator-activated receptor (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) (See Table 1).
- •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, psychiatric or laboratory condition 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 study.
- •7.Pregnant or nursing females.
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