A Double-blind, Randomised, Placebo-controlled Trial Evaluating the Efficacy and Safety of Oral Nerandomilast Treatment in Patients With Systemic Sclerosis (SSc)
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 448
- 试验地点
- 431
- 主要终点
- Time to the first occurrence of disease progression or all-cause death
研究概览
简要总结
Nerandomilast is being developed to help people with systemic sclerosis by potentially improving symptoms and slowing disease progression. This study is open to adults who are at least 18 years old and have systemic sclerosis (SSc). People can join the study if they have limited or diffuse cutaneous SSc with disease onset within 7 years of the first non-Raynaud's symptom. The purpose of this study is to find out whether a medicine called nerandomilast helps people with systemic sclerosis. This study also aims to find out how well nerandomilast is tolerated in people with systemic sclerosis.
Participants are put into 2 groups randomly, which means by chance. One group takes nerandomilast tablets and the other group takes placebo tablets. Placebo tablets look like nerandomilast tablets but do not contain any medicine. Participants take the tablets twice a day.
Participants are in the study for 1 to about 4 years. During this time, they visit the study site regularly and get phone calls from the site staff. During study visits participants regularly have blood samples taken and doctors check changes in skin thickening, lung function, and internal organs, overall health and the safety and tolerability of study treatment in people with SSc. The results are compared between the groups to see whether the treatment works. The doctors also regularly check participants' health and take note of any unwanted effects.
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
- •Signed and dated written informed consent in accordance with ICH-GCP and local legislation prior to admission to the trial.
- •Patients must be at least 18 years of age and fulfil the 2013 American College of Rheumatology/European Alliance of Associations for Rheumatology (ACR/EULAR) criteria for SSc.
- •Patients must be diagnosed with limited cutaneous SSc (lcSSc) or diffuse cutaneous SSc (dcSSc), as defined by LeRoy et al. (1988).
- •Disease onset (defined by first non-RP [Raynaud's phenomenon] symptom) must be within 7 years of Visit
- •Trial participants with dcSSc must have evidence of active disease during screening.
- •Trial participants with lcSSc must have evidence of active disease during screening. LcSSc patients must be anti-centromere antibody (ACA) negative.
- •FVC % predicted ≥45% at Visit
- •Diffusing Capacity of the Lungs for Carbon Monoxide (DLCO) % predicted ≥25% corrected for haemoglobin (Hb) at Visit
- •Women of childbearing potential (WOCBP) must be ready and able to use highly effective methods of birth control.
- •Patients may be either untreated or on stable treatment with permitted immunosuppressive/immunomodulatory agents and/or nintedanib. All treatments must remain stable prior to Visit 2 and during the screening period
排除标准
- •Active, unstable, or uncontrolled vasculitis within 8 weeks prior to Visit 1 or during the screening period.
- •Any suicidal behaviour in the past 2 years.
- •Any suicidal ideation of type 4 or 5 on the C-SSRS in the past 3 months. Further exclusion criteria apply.
研究组 & 干预措施
Placebo
干预措施: Placebo matching nerandomilast formulation 1 (Drug)
Placebo
干预措施: Placebo matching nerandomilast formulation 2 (Drug)
Nerandomilast
干预措施: Nerandomilast formulation 1 (Drug)
Nerandomilast
干预措施: Nerandomilast formulation 2 (Drug)
结局指标
主要结局
Time to the first occurrence of disease progression or all-cause death
时间窗: up to 4 years
次要结局
- Time to all-cause death(up to 4 years)
- Time to first occurrence of absolute increase in mRSS ≥5 points and relative increase from baseline in mRSS ≥25% or death(up to 4 years)
- Time to first occurrence of adjudicated SSc-related related clinically meaningful disease progression or complication or death(up to 4 years)
- Change from baseline in mRSS at Week 52(At baseline and at Week 52.)
- Change from baseline in HAQ-DI score at Week 52(At baseline and at Week 52.)
- Change from baseline in FVC [mL] at Week 52(At baseline and at Week 52.)
- Disease improvement as defined by rCRISS-25 at Week 52(At baseline and at Week 52.)
- Time to first occurrence of confirmed absolute decline from baseline in FVC % predicted ≥5% (for patients with ILD at baseline) or newly diagnosed ILD (for patients without ILD at baseline) or death(up to 4 years)
- Change from baseline in Systemic Sclerosis Impact of Disease (ScleroID) score at Week 52(At baseline and at Week 52.)
- Change from baseline in digital ulcer total burden at Week 52(At baseline and at Week 52.)