EUCTR2017-002447-15-DK进行中（未招募）1 期
A Phase 3 Randomized, Double-Blind, Parallel-Group, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects with Primary Mitochondrial Myopathy Followed by an Open-Label Treatment Extension
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 218
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. Willing and able to provide a signed informed consent form (ICF) prior to participation in any trial-related procedures.
- •2. Agrees and is able to adhere to the trial requirements for the length of the trial, including the use of the elamipretide delivery system.
- •3. Subject is =16 and =80 years of age. In Germany, subjects must be =18 years of age.
- •4. Enrolled (signed ICF) in SPIMM-300 or have prior approval from the Sponsor to enroll without SPIMM-300 participation.
- •5. Diagnosed with PMM in the opinion of the Investigator, consisting of:
- •a. Molecular genetic abnormality of the mitochondrial respiratory chain, and
- •b. Subject reported symptoms (i.e., exercise intolerance, fatigue, muscle weakness) or physical examination findings of myopathy that are the predominant symptoms of the subject’s mitochondrial respiratory chain disorder.
- •6. The subject’s molecular genetic abnormality is consistent with PMM as confirmed by the Adjudication Committee.
- •7. Women of childbearing potential must agree to use 1 of the following methods of birth control from the date they sign the ICF until 28 days after the last dose of IMP:
- •a. Abstinence, when it is in line with the preferred and usual lifestyle of the subject. Subject agrees to use a highly effective method of contraception should they become sexually active.
- •b. Relationships with male partners who have been surgically sterilized by vasectomy (the vasectomy procedure must have been conducted at least 60 days prior to the Screening Visit).
- •c. Barrier method (e.g., condom or occlusive cap) with spermicidal foam/gel/film/cream AND either hormonal contraception (oral, implanted, or
- •injectable) or an intrauterine device or system.
- •Note: Non-childbearing potential is defined as surgical sterilization (e.g., bilateral oophorectomy, hysterectomy, or tubal ligation) or postmenopausal (defined as permanent cessation of menstruation for at least 12 consecutive months prior to the Screening Visit).
- •8. Male subjects with female partners of child-bearing potential must be willing to use a highly effective method of contraception from the date they sign the ICF until 28 days after the last dose of IMP.
- •PART 2 Subject Continuation Criteria
- •A subject must meet all of the following PART 2 Continuation Criteria at the Week 24 Visit in PART 1 to be eligible for PART 2:
- •1. Subjects must continue to be able and willing to adhere to the trial requirements.
- •2. Subject is appropriate to continue in PART 2 (i.e. subject was compliant in SPIMM-301), in the opinion of the Investigator.
- •3. Subject has not had a serious adverse event (SAE)/serious adverse device effect (SADE) attributed to the elamipretide delivery system.
- •4. Subject has not permanently discontinued the elamipretide delivery system.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 6
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 194
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 2
排除标准
- •1. Subject has myopathic signs and/or symptoms due to a neuropathic process (i.e. cerebellar dysfunctions and peripheral neuropathies) or a gait problem that would interfere with the 6MWT, in the opinion of the Investigator.
- •2. Female subjects who are pregnant, planning to become pregnant, or breastfeeding/lactating.
- •3. Walks < 100 meters or > 450 meters during the 6MWT at either the Screening Visit OR Baseline Visit.
- •4. At the Baseline Visit, the estimated glomerular filtration rate (eGFR) < 30 mL/min/1.73 m2, using the Screening Visit value with the Modification of Diet in Renal Disease (MDRD) Study equation.
- •5. Subject has undergone an in-patient hospitalization within the 30 days prior to the Baseline Visit or has a planned hospitalization or a surgical procedure during the trial.
- •6. Subject has clinically significant respiratory disease and/or cardiac disease (medical history or current clinical findings), in the opinion of the Investigator, or prior interventional cardiac procedure (e.g., cardiac catheterization, angioplasty/percutaneous coronary intervention, balloon valvuloplasty, etc.) within 3 months of the Baseline Visit.
- •7. Subject has a pacemaker, implantable cardioverter-defibrillator, or cardiac resynchronization therapy device OR QTc elongation (using the correction factor utilized at the clinical site) defined as a QTc >450 msec in male subjects and >480 msec in female subjects.
- •Note: At the initial electrocardiogram (ECG), if QTc exceeds these parameters, the ECG may be repeated 2 more times (during the same visit), and the average of the 3 QTc values used to determine the subject’s eligibility.
- •8. ECG evidence of acute ischemia, atrial fibrillation, or active conduction system abnormalities with the exception of any of the following:
- •a. First degree AV-block
- •b. Second degree AV-block Type 1 (Mobitz Type 1 / Wenckebach type)
- •c. Right bundle branch block
- •9. Subject has severe vision impairment that, in the opinion of the Investigator, may interfere with their ability to complete all trial requirements
- •10. Subject has a seizure disorder that, in the opinion of the Investigator, may interfere with their ability to complete all trial requirements.
- •11. Active malignancy or any other cancer from which the subject has been disease-free for < 2 years.
- •12. Subject has a solid organ transplant and/or is currently receiving treatment with therapy for immunosuppression, in the opinion of the Investigator.
- •13. Subject has been previously diagnosed with human immunodeficiency virus (HIV), hepatitis B, or hepatitis C infection.
- •14. Subject has a history of a systemic eosinophilic illness and/or an eosinophil count >1,000 cells x106/L at the Screening Visit.
- •15. Subject is currently participating or has participated in an interventional clinical trial (i.e., investigational product or device, stem cell therapy, gene therapy) within 30 days of the Baseline Visit; or is currently enrolled in a non-interventional clinical trial (except for SPIMM-300) at the Baseline Visit which, in the opinion of the Investigator, may be potentially confounding to the results of the current trial (e.g. exercise therapy trial).
- •16. Subject has previously received elamipretide (MTP-131), for any reason.
- •17. Subject has a history of active substance abuse during the year before the Baseline Visit, in the opinion of the Investigator.
- •18. Subject has any prior or current medical condition that, in the judgment of the Investigator, would prevent the subject
研究者
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