NCT05883748Enrolling By Invitation2 期
HELIOS: An Open-Label, Long-Term Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- Enrolling By Invitation
- 入组人数
- 230
- 试验地点
- 34
- 主要终点
- Incidence of clinically abnormal vital signs
研究概览
简要总结
This is an open-label, long-term extension study to investigate the safety, tolerability and efficacy of DISC-1459 in participants with EPP.
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
- None
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants with diagnosis of EPP who are participating (or who have participated) in a prior Disc Medicine bitopertin study and who have completed the randomized treatment phase and End-of-Study visit
- •Aged ≥12 years upon study consent
- •Body weight ≥32 kg for participants <18 years of age and BMI ≥18.5 kg/m2 for adult participants
- •Willing to practice highly effective methods of birth control (both males who have partners of childbearing potential and females of childbearing potential during the study, while taking study drug, and for at least 30 days after the last dose of study drug.
- •Negative urine or serum pregnancy test (females of childbearing potential).
- •Able to understand the study aims, procedures, and requirements, and provide written informed consent (and assent if necessary).
- •Able to comply with all study procedures.
排除标准
- •Participants who have an ongoing SAE from a clinical study that is assessed by the investigator as related to bitopertin
- •Other medical or psychiatric condition or laboratory finding not specifically noted above that, in the judgement of the Investigator or Sponsor, would put the participant at unacceptable risk or otherwise preclude participation 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
- •Planned treatment with afamelanotide or dersimelagon during the study
- •Planned use of any drugs or herbal remedies known to be strong inhibitors or inducers of cytochrome p450 (CYP)3A4 enzymes throughout the study
- •If female, pregnant, or breastfeeding
- •Participation in any other clinical protocol or investigational trial, other than Disc Medicine bitopertin trials, that involves administration of experimental therapy and/or therapeutic devices within 30 days of Day 1
- •Score of PHQ-8 ≥10 at screening or imminent suicidal risk identified by the C-SSRS as defined as suicidal ideation with intent (Grade 4 or 5) within the last year or any suicidal behavior within the last 5 years.
- •Consumption of grapefruit/Seville orange and products containing these for 14 days prior to first dose of study drug and throughout the study
研究组 & 干预措施
DISC-1459 Oral Dose Level 1
Experimental
Oral dose, once a day
干预措施: DISC-1459 (Drug)
DISC-1459 Oral Dose Level 2
Experimental
Oral dose, once a day
干预措施: DISC-1459 (Drug)
结局指标
主要结局
Incidence of clinically abnormal vital signs
时间窗: up to 5 Years
Incidence of clinically abnormal physical exam
时间窗: up to 5 Years
Incidence of abnormal laboratory test results
时间窗: up to 5 Years
Incidence of treatment-emergent adverse events
时间窗: up to 5 Years
Assessment of Patient Health Questionnaire (PHQ-8)
时间窗: up to 5 Years
The Patient Health Questionnaire (PHQ-8), an 8-item participant-report measure for screening for depression and for establishing depression severity. The total score ranges from 0-24, with a higher score indicating greater depression symptom severity.
Assessment of C-SSRS
时间窗: up to 5 Years
The C-SSRS is a clinician-rated scale that assesses suicidality from ideation to behaviors and monitors the potential emergence of suicidality in clinical studies.
次要结局
- Change from baseline in daily daylight tolerance, as assessed by total hours spent in the sunlight without pain and average time to first prodromal syndrome in sunlight(up to 5 Years)
- Plasma Bitopertin Concentrations(up to 5 Years)
- Change from baseline in whole blood metal-free PPIX levels(up to 5 Years)
研究者
研究点 (34)
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