NL-OMON51665已完成2 期
A DOUBLE-BLIND, PLACEBO-CONTROLLED, STUDY TO EVALUATE THE EFFICACY AND SAFETY OF 24 WEEKS TREATMENT WITH REN001 IN PATIENTS WITH PRIMARY MITOCHONDRIAL MYOPATHY (PMM) - Phase 2b Safety and Efficacy Study of REN001 in Mitochondrial Myopathy
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 21
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. Subjects aged 18 years or older with PMM
- •2. A confirmed PMM diagnosis due to known pathogenic gene mutation or deletion
- •of the mitochondrial genome.
- •3. Documented PMM primarily characterized by exercise intolerance or active
- •muscle pain.
- •4. Subjects must be ambulatory and able to perform the 12MWT independently
- •(walking aids are allowed).
- •5. Distance walked of <=1000 meters at Screening in the 12MWT (must be obtained
- •at least 4 weeks before randomization).
- •6. Have no changes to any therapeutic exercise regimen within 30 days prior to
- •Day 1 and be willing to remain on the same therapeutic exercise regimen for the
- •duration of the study.
- •7. Be willing and able to swallow gelatin capsules.
- •8. Females should be either of non-child-bearing potential or must agree to use
- •highly effective methods of contraception from Screening through to 30 days
- •after last dose in the study. Males with partners who are WOCBP must also use
- •contraception.
- •9. Concomitant medications (including supplements) must be stable for at least
- •1 month prior to enrolment and throughout participation in the study.
- •10. For subjects under 25 years of age, confirmation of bone growth plate
- •closure by wrist radiograph
- •11. Evidence of a personally signed and dated informed consent document
- •indicating that the subject has been informed of all pertinent aspects of the
排除标准
- •1. Participation in a prior REN001 study.
- •2. Currently taking or anticipated to need a PPAR agonist during the study.
- •3. Bone deformities or motor abnormalities other than related to the
- •mitochondrial myopathy that may interfere with the outcome measures.
- •4. Treatment with an investigational drug within 3 months or 5 drug half-lives,
- •whichever is longer, prior to Day 1.
- •5. Anticipated to need a prescription and/or non-prescription drug that might
- •interfere with the study endpoints
- •6. Currently taking drugs with a narrow therapeutic index and BCRP mediated
- •7. Clinically significant kidney disease or impairment with an eGFR less than
- •60ml/min/1.73m2 using the CKD-EPI creatinine equation at Screening.
- •8. Clinically significant liver disease or impairment of AST or ALT Grade 2 or
- •above (>2.5 x ULN), or Total bilirubin > 1.6 x ULN or >ULN with other
- •signs and symptoms of hepatotoxicity at Screening.
- •9. Uncontrolled diabetes and/or a Screening HbA1c of >=11%.
- •10. Uncontrolled epilepsy.
- •11. Evidence of significant concomitant clinical disease that may need a change
- •in management during the study or could interfere with the conduct or safety of
- •this study.
- •12. A history of cancer. A history of in situ basal cell carcinoma in the skin
- •is allowed.
- •13. Have been hospitalized within the 3 months prior to Screening for any major
- •medical condition (as deemed by the Investigator).
- •14. Clinically significant cardiac disease and/or clinically significant ECG
- •abnormalities including a screening QTcF of >= 450 msec, 2nd degree heart
- •block, symptomatic tachyarrhythmia or unstable arrythmia that in the opinion of
- •the Investigator should exclude the subject from completing exercise tests
- •(i.e. study 12 MWT and 30 STS tests).
- •15. Any condition possibly reducing drug absorption.
- •16. Evidence of hospitalization for rhabdomyolysis within the year prior to
- •17. Positive HBsAg and HBcAb at screening or positive for hepatitis C or HIV at
- •Screening..
- •18. Pregnant or nursing females.
- •19. History of sensitivity to PPAR agonists.
- •20. Donation or intent to donate blood, or blood components during the study or
- •within one month after completion of the study.
- •21. A history of drug dependency. Use of opiates/cannabis for medical reasons
- •is acceptable with prescription evidence or at the Investigators discretion.
- •22. A history of alcohol dependency.
- •23. Significant impairment due to central or peripheral nervous system
- •involvement that would interfere with the exercise tests.
- •24. Significant weakness not caused by the underlying primary muscle disease
- •such as post stroke or neurogenic weakness.
- •25. Have had an organ transplant.
- •26. Are not eligible or have a contraindication for cataract surgery.
- •27. A history of osteoporosis as evidenced by non-traumatic (stress) fractures
- •or a prior T-score of -2.5 or worse which has not been adequately addressed.
- •28. Inability to comprehend or unwilling to follow the study requirements
- •including restrictions on treatments, attendance at the study center,
- •completion of questionnaires and participation in laboratory testing as called
- 另有 4 项未显示
研究者
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