2023-508541-41-00已完成2 期
A Phase 2a Safety, Tolerability, and Pharmacodynamic Study of OMT-28 in PMD patients with myopathy and/or cardiomyopathy and inflammation (PMD-OPTION)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 42
- 试验地点
- 9
- 主要终点
- Primary Efficacy Endpoint: Number of patients (responder rate) with a between phase difference in GDF-15 of at least 20% decrease after 12 weeks of treatment.
研究概览
简要总结
- To determine the responder rate of patients with a between phase difference in GDF-15 of at least 20% decrease after 12 weeks of treatment.
- To assess safety and tolerability of OMT-28 at 24 mg
入排标准
- 年龄范围
- 18 years 至 64 years（18-64 Years）
- 接受健康志愿者
- 否
入选标准
- •Any gender, age 18 to 60 years
- •Documented mutation resulting in mitochondrial disease: mitochondrial tRNA point mutations, including m3243A>G, m8344A>G, and single mtDNA deletions
- •Diagnosis of Cardiomyopathy defined as LV hypertrophy and/or LVEF<50% and/or late gadolinium enhancement on cardiac MRI and/or Myopathy as defined by the International Workshop: Outcome measures and clinical trial readiness in primary mitochondrial myopathies in children and adult (Mancuso et al. 2017)
- •GDF-15 between 1,200 to 10,000 pg/mL measured at screening
- •Ability to perform the exercise tests
- •Willing and able to provide a signed Informed Consent, as well as written documentation in accordance with country and local privacy requirements, e.g., written data protection consent
- •Able and willing to comply with the requirements of this study protocol
- •Both female patients, as well as, female partners of male patients who are of child- bearing potential must be willing to not become pregnant for the complete duration of the study (30 days after the last dose of study medication).
排除标准
- •Pregnant, breastfeeding, or unwilling to practice birth control during participation in the study
- •Regular use of steroid, non-steroidal anti-inflammatory drug (NSAID), or colchicine within 30 days before screening
- •Chronic use of Metformin
- •Use of fish oil / omega-3 fatty acid supplements within 2 weeks before screening
- •Drinking more than 9 standard cups of alcohol per week and/or more than 3 standard cups of alcohol per occasion
- •Positive drug and alcohol screen (including opiates, methadone, cocaine, amphetamines [including ecstasy], cannabinoids, barbiturates, benzodiazepines)
- •Any significant hepatic disease (defined as the presence of at least one of the following: AST, ALT, GGT, total bilirubin, or alkaline phosphatase >3x upper limit normal)
- •Receiving any investigational therapy or any approved therapy for investigational use within 30 days or 5 half-lives prior to screening (whichever is longer)
- •Received any vaccines (including the booster vaccination for Covid- 19) within two weeks prior to Visit 1
- •Females of childbearing potential (those who are not surgically sterilized or post- menopausal for at least 1 year) are excluded from participation in the study unless they agree to use adequate contraception as described in Appendix 11.4 of the protocol
- •Males (including sterilized subjects) and whose female partners have child-bearing potential, must agree to use male contraception (condoms) during the period from the time of signing the informed consent form (ICF) through 30 days after the last dose of study drug. They must agree to immediately inform the investigator if his partner becomes pregnant during the study.
- •Presence of a condition or abnormality that in the opinion of the Investigator would compromise the safety of the patient or the quality of the data
- •Subjects who have previously been exposed to OMT-28, whether responder or nonresponder
- •Any use of statins (HMG-CoA reductase inhibitors)
- •Use of quinine, tacrolimus, mycophenolate mofetil, ciclosporin, serotine receptortype 1 agonist, penicillin G, penicillamine (dpenicillamine), nicotinic acid (niacin), colchicine, isotretinoin, and amiodarone, PPAR activators, AMPK activators, Sirtuin activators, Steroids, COX-inhibitors
- •Subjects with a history of cancer in the last 5 years
- •Hypertension defined as systolic BP >160 mmHg or diastolic BP >100 mmHg at screening
- •Uncontrolled Diabetes mellitus according to investigator's assessment
- •Stroke-like episodes or seizures occurred within last 6 months
- •Motoric abnormalities other than related to the mitochondrial disease interfering with the outcome parameters
- •History or evidence of active tuberculosis (TB) infection, any co-disease with inflammatory condition (e.g. Inflammatory Bowel Disease (IBD) etc.)
- •Patients with a positive hepatitis panel and/or positive immunodeficiency virus test at screening
结局指标
主要结局
Primary Efficacy Endpoint: Number of patients (responder rate) with a between phase difference in GDF-15 of at least 20% decrease after 12 weeks of treatment.
Primary Efficacy Endpoint: Number of patients (responder rate) with a between phase difference in GDF-15 of at least 20% decrease after 12 weeks of treatment.
Primary Safety Endpoints: Incidence, severity, seriousness, reported causality, and duration of TEAEs, clinically significant changes in safety laboratory, vital signs, and 12-lead ECG
Primary Safety Endpoints: Incidence, severity, seriousness, reported causality, and duration of TEAEs, clinically significant changes in safety laboratory, vital signs, and 12-lead ECG
次要结局
未报告次要终点
研究者
Dr. Robert Fischer
Scientific
OMEICOS Therapeutics GmbH
研究点 (9)
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