NCT04519567已完成1 期
A Phase 1 Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneous CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia
适应症
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 27
- 试验地点
- 2
- 主要终点
- Number of Participants with Treatment Emergent Adverse Events
研究概览
简要总结
To evaluate the safety and tolerability of multiple ascending doses of CTI-1601 in participants with Friedreich's ataxia
详细描述
Multiple Ascending Dose (MAD), Double-Blind, Placebo Controlled Study.
To evaluate the safety and tolerability of multiple ascending doses of CTI-1601 in subjects with Friedreich's ataxia.
Secondary Objectives:
- To evaluate the pharmacokinetics (PK) of CTI-1601 following, multiple, increasing, doses of subcutaneously (SC) administered CTI-1601.
- To evaluate the pharmacodynamics (PD) of CTI-1601 following, multiple, increasing, doses of SC administered CTI-1601.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subject has genetically confirmed Friedreich's ataxia diagnosis manifested by homozygous GAA repeat expansions, with repeat sizing (if available) included on diagnostic report.
- •Subject is male or female, 18 years of age or older at screening
- •Subject must have a mFARS_neuro score ≥ 20 and be able to traverse a distance of 25 feet with or without some assistive device (cane, walker, crutches, self-propelled wheelchair) and (a) be able to sit upright with thighs together and arms crossed without requiring support on more than two sides; (b) be able to transfer from bed to chair independently or with assistance if, in the opinion of the principal investigator, the degree of physical disability does not result in undue risk to the subject while participating in the study; and (c) perform basic daily care, such as feeding themselves and personal hygiene, with minimal assistance.
- •Subjects must weigh > 40 kilograms (kg).
排除标准
- •Subjects who had a serious adverse event (SAE), an adverse event (AE) that is Grade 3 or higher according to the Common Terminology Criteria for Adverse Events (CTCAE), version 5.0 (or higher), or an AE considered clinically significant during participation in CLIN-1601-101 (NCT04176991).
- •Subjects who are confirmed as compound heterozygous (GAA repeat expansion on only one allele) for Friedreich's ataxia.
- •Subject use of investigational drug (other than CTI-1601) or device within 90 days prior to screening.
- •Subject requires use of amiodarone.
- •Subject used erythropoietin, etravirine, or gamma interferon within 3 months prior to screening.
- •Subject use of daily biotin supplementation that exceeds 30 mcg/day, either as part of a multivitamin or as a standalone supplement, within 7 days prior to study drug administration and/or throughout the entire study.
- •Subject has clinically significant arrhythmia on electrocardiogram (ECG), or evidence of predisposition to significant ventricular arrhythmia on ECG, or evidence of active and unstable coronary artery disease.
- •Male subject who has a QT interval corrected for heart rate using Fridericia's formula (QTcF) > 450 milliseconds or female subject who has a QTcF > 470 milliseconds on an ECG.
- •Subject has a screening echocardiogram left ventricular ejection fraction < 45 percent.
- •Subject has a history of aspiration, aspiration pneumonia, or recurrent episodes of pneumonia (greater than or equal to 2 episodes of pneumonia) within the last 12 months.
结局指标
主要结局
Number of Participants with Treatment Emergent Adverse Events
时间窗: Through study completion, an average of 75 days
Overall summary of Participants with Treatment Emergent Adverse Events
Number of Participants with Treatment Emergent Adverse Events by System Organ Classification and Preferred Term
时间窗: Through study completion, an average of 75 days
Overall summary of Participants with Treatment Emergent Adverse Events by System Organ Classification (MedDRA version 23.0)
次要结局
- Changes from Baseline in Frataxin Levels in Platelets(At baseline and up to 13 days)
- Changes from Baseline in Gene Expression in Whole Blood(At baseline and up to 16 days)
- Pharmacokinetics - Minimum or "trough" plasma concentration after multiple doses(At baseline and up to 15 days)
- Pharmacokinetics - Area under the concentration time curve (AUC) from time 0 through the last measurable time point(At baseline and up to 15 days)
- Changes from Baseline in Gene Expression in Buccal Cells(At baseline and up to 43 days)
- Changes from Baseline in Levels of Defined Protein Markers in Blood(At baseline and up to 16 days)
- Changes from Baseline in Levels of Specialized Lipids in Blood(At baseline and up to 16 days)
- Pharmacokinetics - Maximum observed plasma concentration after multiple doses(At baseline and up to 15 days)
- Pharmacokinetics - Terminal half-life estimation(At baseline and up to 15 days)
- Changes from Baseline in Frataxin Levels in Buccal Cell(At baseline and up to 43 days)
- Changes from Baseline in Levels of Protein Markers in Buccal Cell(At baseline and up to 43 days)
- Changes from Baseline in Frataxin Levels in Skin Punch Cells(At baseline and up to 13 days)
研究者
研究点 (2)
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