2024-517436-22-00已完成2 期
A Phase 2 Study of the Safety, Efficacy, and Pharmacodynamics of RTA 408 in the Treatment of Friedreich's Ataxia (MOXIe)
Reata Pharmaceuticals Inc., Reata Pharmaceuticals Inc., Reata Pharmaceuticals Inc.2 个研究点 分布在 2 个国家目标入组 21 人开始时间: 2024年11月15日最近更新:
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 21
- 试验地点
- 2
- 主要终点
- Part 1: To evaluate the change in peak work during maximal exercise testing
研究概览
简要总结
Part 1 • To evaluate the change in peak work during maximal exercise testing • To evaluate the safety and tolerability of RTA 408
Part 2 • To evaluate the change in the modified Friedreich's ataxia rating scale (mFARS) score at Week 48 • To evaluate the safety and tolerability of RTA 408
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 0 years 至 64 years（0-17 Years, 18-64 Years）
- 接受健康志愿者
- 是
入选标准
- •Part 1 and Part 2: Patients must have genetically confirmed Friedreich’s ataxia
- •Part 1 and Part 2: Patients must be willing to practice medically acceptable methods of birth control
- •Part 1 and Part 2: Patients must provide written informed consent for study participation, approved by the appropriate Institutional Review Board
- •Extension eligibility: Patients must complete 12 weeks of treatment in Part 1 or 24 weeks of treatment in Part 2, have no major protocol deviations, and meet inclusion criteria as follows
- •Extension eligibility: Patients must have adequate kidney function defined as an estimated glomerular filtration rate (eGFR) ≥ 60 mL/min/1.73 m^2 using the Modification of Diet in Renal Disease (MDRD) 4-variable formula
- •Extension eligibility: Patients must have a left ventricular ejection fraction ≥ 40% (based on echocardiogram performed at Screening Visit or within 90 days prior to Screening Visit)
- •Extension eligibility: Patients must be able to swallow capsules
- •Extension eligibility: Patients must be willing and able to cooperate with all aspects of the extension
- •Extension eligibility: Patients must be willing to practice medically acceptable methods of birth control
- •Extension eligibility: Patients must provide written informed consent for study participation, approved by the appropriate Institutional Review Board
- •Extension eligibility: Patients must have been enrolled in Part 1 or Part 2 and completed assessments through the follow-up visit with no major protocol deviations
- •Part 1 and Part 2: Patients must have an mFARS score ≥ 20 and ≤
- •The average of the two mFARS values collected at Screening and Day 1 visits must fall within the allowable range, and they must be within 4.5 points of each other
- •Part 1 and Part 2: Patients must be male or female and ≥ 16 years of age and ≤ 40 years of age
- •Part 1 and Part 2: Patients must have no changes to their exercise regimen within 30 days prior to Study Day 1 and be willing to remain on the same exercise regimen during the study period
- •Part 1 and Part 2: Patients must have the ability to complete maximal exercise testing
- •Part 1 and Part 2: Patients must have adequate kidney function defined as an estimated glomerular filtration rate (eGFR) ≥ 60 mL/min/1.73 m^2 using the Modification of Diet in Renal Disease (MDRD) 4-variable formula
- •Part 1 and Part 2: Patients must have a left ventricular ejection fraction ≥ 40% (based on echocardiogram performed at Screening Visit or within 90 days prior to Screening Visit)
- •Part 1 and Part 2: Patients must be able to swallow capsules
- •Part 1 and Part 2: Patients must be willing and able to cooperate with all aspects of the protocol
排除标准
- •Part 1 and Part 2: Patients must not have uncontrolled diabetes (HbA1c > 11.0%)
- •Part 1 and Part 2: Patients must not have participated in any other interventional clinical study within 30 days prior to Study Day 1
- •Part 1 and Part 2: Patients must not have a cognitive impairment that may preclude ability to comply with study procedures
- •Part 1 and Part 2: Patients must not be unable to comply with the requirements of the study protocol or be unsuitable for the study for any reason, in the opinion of the investigator
- •Part 1 and Part 2: Patients must not have used antioxidant supplements, including but not limited to idebenone, coenzyme Q10, nicotinamide, and vitamin E above the recommended daily allowance, within 14 days prior to Study Day 1, or plan to take any of these supplements during the time of study participation
- •Part 1 and Part 2: Patients must not have previously documented mitochondrial respiratory chain disease
- •Part 1 and Part 2: Patients must not have a history of thromboembolic events within the past 5 years
- •Part 1 and Part 2: Patients must not have taken anticoagulant therapy within 30 days prior to Study Day 1
- •Part 1 and Part 2: Patients must not have scheduled surgical treatment for scoliosis or foot deformity during the study
- •Part 1 and Part 2: Patients must not have had significant suicidal ideation within 1 month prior to Screening Visit as per investigator judgment or any history of suicide attempts
- •Part 1 and Part 2: Patients must not be pregnant or breastfeeding
- •Part 1 and Part 2: Patients must not have BNP level > 200 pg/mL
- •Part 1 and Part 2: Prior participation in a trial with RTA 408
- •Extension eligibility: Patients must not have uncontrolled diabetes (HbA1c > 11.0%)
- •Extension eligibility: Patients must not have B-type natriuretic peptide (BNP) level > 200 pg/mL
- •Extension eligibility: Patients must not have a history of clinically significant left-sided heart disease and/or clinically significant cardiac disease, with the exception of mild to moderate cardiomyopathy associated with Friedreich's ataxia
- •Extension eligibility: Patients must not have a history of clinically significant liver disease or has, at screening, clinically relevant deviations in laboratory tests
- •Extension eligibility: Patients must not have a cognitive impairment that may preclude ability to comply with study procedures
- •Extension eligibility: Patients must not be unable to comply with the requirements of the study protocol or be unsuitable for the study for any reason, in the opinion of the investigator
- •Extension eligibility: Patients must not have a history of thromboembolic events within the past 5 years
- •Extension eligibility: Patients must not have taken anticoagulant therapy within 30 days prior to Extension Day 1
- •Extension eligibility: Patients must not be pregnant or breastfeeding
- •Part 1 and Part 2: Patients must not have a history of clinically significant left-sided heart disease and/or clinically significant cardiac disease, with the exception of mild to moderate cardiomyopathy associated with Friedreich’s ataxia, including but not limited to any of the following: a. Clinically significant congenital or acquired valvular disease; b. Pericardial constriction (based on echocardiogram performed at Screening Visit or within 90 days prior to Screening Visit); c. Restrictive or congestive cardiomyopathy (based on echocardiogram performed at Screening Visit or within 90 days prior to Screening Visit); d. Symptomatic coronary disease (prior myocardial infarction, percutaneous coronary intervention, coronary artery bypass graft surgery, or angina); e. History of hospitalization for heart failure in the last five years; f. Cardiac insufficiency, defined as New York Heart Association Class > 2; g. History of atrial fibrillation; h. History of unstable arrhythmias
- •Extension eligibility: Patients must not have an ongoing SAE from a clinical study that is assessed by the investigator as related to RTA 408
- •Extension eligibility: Patients must not have discontinued treatment early in Part 1 or Part 2
- •Part 1 and Part 2: Patients must not have known active fungal, bacterial, and/or viral infection, including human immunodeficiency virus (HIV) or hepatitis virus (B or C)
- •Part 1 and Part 2: Patients must not have known or suspected active drug or alcohol abuse, as per investigator judgment
- •Part 1 and Part 2: Patients must not have clinically significant abnormalities of clinical hematology or biochemistry, including but not limited to elevations greater than 1.5 times the upper limit of normal (ULN) of AST or ALT. Levels above this threshold are allowable if attributable to muscle injury
- •Part 1 and Part 2: Patients must not have any abnormal laboratory test value or clinically significant pre-existing medical condition that, in the opinion of the investigator, would put the patient at risk by study enrollment
- •Part 1 and Part 2: Patients must not have taken any of the following drugs within 7 days prior to Study Day 1 or plan to take any of these drugs during the time of study participation: a. Sensitive substrates for cytochrome P450 2C8 or 3A4 (e.g., repaglinide, midazolam, sildenafil); b. Moderate or strong inhibitors or inducers of cytochrome P450 3A4 (e.g., carbamazepine, phenytoin, ciprofloxacin, grapefruit juice); c. Substrates for p-glycoprotein transporter (e.g., ambrisentan, digoxin)
- •Part 1 and Part 2: Patients must not have a history of clinically significant liver disease (e.g., fibrosis, cirrhosis, hepatitis), or has, at screening, clinically relevant deviations in laboratory tests including any one of the following: a. ALT and/or AST > 1.5-fold ULN; b. bilirubin > 1.2-fold ULN; c. ALP > 2-fold ULN; d. albumin < lower limit of normal (LLN)
结局指标
主要结局
Part 1: To evaluate the change in peak work during maximal exercise testing
Part 1: To evaluate the change in peak work during maximal exercise testing
Part 1: To evaluate the safety and tolerability of RTA 408
Part 1: To evaluate the safety and tolerability of RTA 408
Part 2: To evaluate the change in the mFARS score at Week 48
Part 2: To evaluate the change in the mFARS score at Week 48
Part 2: To evaluate the safety and tolerability of RTA 408
Part 2: To evaluate the safety and tolerability of RTA 408
次要结局
- Part 1: To evaluate the change in the modified Friedreich’s ataxia rating scale (mFARS) score
- Part 2: To evaluate the change in peak work during maximal exercise testing at Week 48
- Part 2: To evaluate the Patient Global Impression of Change at Week 48
- Part 2: To evaluate the Clinical Global Impression of Change at Week 48
研究者
CTA Lead Dept
Scientific
Reata Pharmaceuticals Inc.
研究点 (2)
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