NCT05308472已完成2 期
(AURORA) A Randomized, Double-blind, Placebo-Controlled Study of Bitopertin to Evaluate the Safety, Tolerability, Efficacy, and Protoporphyrin IX (PPIX) Concentrations in Participants With Erythropoietic Protoporphyria (EPP)
适应症
干预措施
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 75
- 试验地点
- 17
- 主要终点
- Percent Change From Baseline in Whole Blood Metal-free PPIX Levels
研究概览
简要总结
This is a Phase 2, multi-center, double-blind, placebo-controlled, parallel group study of bitopertin to evaluate the safety, tolerability, efficacy, and PPIX concentration change in participants with EPP. Participants may roll over to an open label extension portion after completing the double-blind treatment period.
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
- Triple (Participant, 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
- •Aged 18 years or older at the time of signing the informed consent form (ICF).
- •Diagnosis of EPP, based on medical history by ferrochelatase ( FECH) genotyping or by biochemical porphyrin analysis.
- •Body weight ≥50 kg.
- •Washout of at least 2 months prior to Screening of afamelanotide and dersimelagon, if applicable.
- •Aspartate aminotransferase (AST) and alanine transaminase (ALT) <2× upper limit of normal (ULN) and total bilirubin <ULN (unless documented Gilbert syndrome) at Screening. Albumin >lower limit of normal (LLN).
排除标准
- •Medical History:
- •Major surgery within 8 weeks before Screening or incomplete recovery from any previous surgery.
- •Other than EPP, an inherited or acquired red cell disease associated with anemia.
- •A history or known allergic reaction to any investigational product excipients or history of anaphylaxis to any food or drug.
- •History of liver transplantation.
- •History of alcohol dependence or excessive alcohol consumption, as assessed by the Investigator.
- •Human immunodeficiency virus (HIV), active Hepatitis B, or C.
- •Other medical or psychiatric condition or laboratory finding not specifically noted above that, in the judgment of the Investigator or Sponsor, would put the participant at unacceptable risk or otherwise preclude the participant from participating in the study
- •Condition or concomitant medication that would confound the ability to interpret clinical, clinical laboratory, or participant diary data, including a major psychiatric condition that has had an exacerbation or required hospitalization in the last 6 months.
- •Treatment History:
- •Concurrent or planned treatment with afamelanotide or dersimelagon during the study period.
- •Treatment with opioids for any period >7 days in the 2 months prior to screening or anticipated to require opioid use for >7 days at any point during the study.
- •New treatment for anemia, including initiation of iron supplementation, in the 2 months prior to Screening.
- •Current or planned use of any drugs or herbal remedies known to be strong inhibitors or inducers of CYP3A4 enzymes for 28 days prior to the first dose and throughout the study.
- •Laboratory Exclusions:
- •Hemoglobin <10 g/dL at Screening.
研究组 & 干预措施
Placebo
Placebo Comparator
干预措施: Placebo (Drug)
DISC-1459 oral dose level 1
Experimental
干预措施: DISC-1459 (Drug)
DISC-1459 oral dose level 2
Experimental
干预措施: DISC-1459 (Drug)
结局指标
主要结局
Percent Change From Baseline in Whole Blood Metal-free PPIX Levels
时间窗: 121 days
Percent change from baseline in PPIX concentration was analyzed using a mixed model repeated measures analysis in the ITT population.
次要结局
- Total Hours of Sunlight Exposure to Skin on Days With no Pain From 1000 to 1800 Hours (10:00am to 6:00pm)(121 days)
- Daily Sunlight Exposure Time (Minutes) to First Prodromal Symptom (Burning, Tingling, Itching, or Stinging) Associated With Sunlight Exposure Between 1 Hour Post-sunrise and 1 Hour Pre-sunset(121 days)
- Total Pain Intensity(Sum of Day 1 to Day 121)
- Incidence of Treatment-emergent Adverse Events(121 days)
- Erythrocyte Total PPIX Concentrations(121 days)
- Plasma Total PPIX Concentrations(121 days)
- Whole Blood Total PPIX Concentrations(121 days)
- Plasma Bitopertin Concentrations(Day 29)
研究者
研究点 (17)
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