NCT02170025终止2 期
Multi-center Phase 2 Study to Assess the Safety, Tolerability and Early Signs of Efficacy of Tid Orally Administered BAY63-2521 in Adult Delta F508 Homozygous Cystic Fibrosis Patients
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- Bayer
- 入组人数
- 21
- 主要终点
- Change of Sweat Chloride Content From Baseline
研究概览
简要总结
Assessment of the safety, tolerability and early signs of efficacy of three times a day orally administered BAY63-2521 in adult delta F508 homozygous Cystic Fibrosis patients not on treatment with Orkambi
详细描述
The study consists of two parts. The first part is double-blind, randomized and placebo-controlled. The second part has an open-label study design. In part 1 patients on Orkambi or other CFTR-modulators are excluded. In part 2 patients on Orkambi are allowed to be included under certain conditions.
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 informed consent available before any study specific tests or procedures are performed
- •Patients must be at least 18 years of age at time of inclusion (i.e. upon signature of informed consent)
- •Patient diagnosed with Cystic Fibrosis according to standard criteria (i.e. either elevated sweat chloride content above 60 mmol/ L and/ or genetic testing)
- •Patient is homozygous for the deltaF508 mutation
- •Patient has a mild-to-moderate stage of lung disease as determined by FEV1 (FEV1 between 40 and 100% predicted)
- •Patient has a stable condition of lung disease (no ongoing or recent pulmonary exacerbation and no change in current treatment) within the last 4 weeks prior to screening
- •Ability and willingness to understand and follow study procedures for the entire study
- •Patients do not smoke. Patients with a history of smoking can be included, if they have refrained from smoking for the last 3 months. If a patients starts smoking during the study participation, he/ she needs to be excluded and considered to be a drop out
- •Body mass index (BMI): ≥ 16 kg/ m² (calculated by dividing the patient's weight by the square of his/ her height [kg/ m2])
- •Inclusion criterion valid for study part 1 only:
- •Women of childbearing potential must agree to use adequate contraception when sexually active. 'Adequate contraception' is defined as one highly effective form of contraception (intrauterine devices [IUD], contraceptive implants or tubal sterilization) or a combination of methods (hormone method with a barrier method ). If a partner's vasectomy is the chosen method of contraception or if a partner has documented azoospermia, a hormone or barrier method must be used in combination. Adequate contraception is required from the signing of the informed consent form up until 4 weeks after the last study drug administration
- •Inclusion criteria valid for study part 2 only:
- •Women of childbearing potential must agree to use adequate contraception when sexually active. 'Adequate contraception' is defined as one highly effective form of contraception (intrauterine devices [IUD], contraceptive implants or tubal sterilization) or a combination of methods (hormone method with a barrier method). For patients on Orkambi hormonal methods (including hormonal oral contraceptives) cannot be accepted in this study. They need to choose non-hormonal methods. If a partner's vasectomy is the chosen method of contraception or if a partner has documented azoospermia, a hormone or barrier method must be used in combination. Adequate contraception is required from the signing of the informed consent form up until 4 weeks after the last study drug administration
- •Patients receiving Orkambi (Lumcaftor + Ivacaftor) as part of their standard care need to be on stable Orkambi treatment for at least 3 months prior to screening (patients on Lumacaftor and/or Ivacaftor are excluded in part 1)
排除标准
- •Patients with Cystic Fibrosis with any background other than homozygous deltaF508 mutation
- •Exclusion criterion only valid for study part 1: Patients receiving treatment with Lumacaftor and/ or Ivacaftor
- •Active state of hemoptysis or pulmonary hemorrhage, including those events managed by bronchial artery embolization. Also any history of moderate hemoptysis within the 3 months prior to inclusion
- •Any history of pneumothorax, bronchial artery embolization or massive hemoptysis. Massive hemoptysis being defined as acute bleeding >240 mL in a 24-hour period or recurrent bleeding >100 mL/ d over several days
- •A positive sputum culture for Burkholderia cenocepacia, Burkholderia dolosa, and/ or Mycobacterium abscessus either currently or within the previous year
- •Active allergic broncho-pulmonary aspergillosis
- •Current pulmonary exacerbation
- •Known history of solid organ transplantation
- •Known history of any form of pulmonary hypertension
- •Clinically relevant deviations of the screened laboratory parameters from reference ranges outside of expected changes for Cystic Fibrosis patients, especially a hemoglobin value below 110 g/L or a creatinine clearance based on the Cockcroft-Gault formula < 15 ml/ min
研究组 & 干预措施
Riociguat (Adempas, BAY63-2521)
Experimental
Participants received 0.5 mg BAY63-2521 three times daily (tid) for 14 days. The dose would be increased to 1 mg BAY63-2521 for an additional 14 days, if this was considered safe and tolerable on the basis of the available data for a given patient.
干预措施: Riociguat (Adempas, BAY63-2521) (Drug)
Placebo
Experimental
Participants received matching placebo tid.
干预措施: Placebo (Drug)
结局指标
主要结局
Change of Sweat Chloride Content From Baseline
时间窗: Baseline, at day 14 and day 28 in study part 1
Sweat chloride samples were obtained by using a Macroduct induction and collection device according to standard procedures.
次要结局
- Change of FEV1 From Baseline(From Baseline to Day 14, Day 28 and Follow-up)
研究者
相似试验
已完成
2 期
Safety and Efficacy of Dexlansoprazole Delayed-Release Capsules in Treating Symptomatic Non-Erosive Gastroesophageal Reflux Disease in AdolescentsGastroesophageal Reflux DiseaseNCT01642602Takeda104
已完成
2 期
BTK Inhibitor BGB-3111 in Chinese Participants With Diffuse Large B-Cell Lymphoma (Non-GCB) and Indolent Lymphoma (FL and MZL)Marginal Zone LymphomaDiffuse Large B-Cell LymphomaFollicular LymphomaNCT03520920BeiGene41
终止
2 期
Efficacy, Safety and Tolerability of AG013 in Oral Mucositis Compared to Placebo When Administered Three Times Per DayOral MucositisNCT03234465Oragenics, Inc.200
已完成
2 期
KN046 in Subjects With Late Stage Esophageal Squamous Cell CarcinomaEsophageal Squamous Cell CarcinomaNCT03925870Jiangsu Alphamab Biopharmaceuticals Co., Ltd45
已完成
2 期
Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary AngioedemaGenetic DisordersNCT00262288Pharming Technologies B.V.14