A randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability and pharmacokinetics of single and multiple ascending doses of LTP001 in healthy adult participants (Part A) and to evaluate the efficacy and safety of LTP001 for the treatment of participants with pulmonary arterial hypertension (Part B)
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 68
- 试验地点
- 50
- 主要终点
- Part A: All safety endpoints, including vital signs, ECG, central lab evaluations and AEs up to and including the EoS visit. Part B: (Period 1) Change in PVR from baseline to 24 weeks (Period 2): All safety endpoints, including vital signs, ECG, central lab evaluations, treatment-emergent AEs and discontinuations due to AEs.
研究概览
简要总结
Part A: To assess the safety and tolerability of single and multiple doses of LTP001 in healthy participants Part B: Period 1: To characterize the dose-response relationship of LTP001 plus standard-of-care (SoC) compared to placebo plus SoC in participants with WHO functional class (FC) II-III PAH Period 2: To assess the safety and tolerability of LTP001
入排标准
- 年龄范围
- 18 years 至 65+ years（18-64 Years, 65+ Years）
- 接受健康志愿者
- 是
入选标准
- •Part A: Signed informed consent must be obtained prior to participation in the study. Part B: Confirmed diagnosis of pulmonary arterial hypertension
- •Part A: Healthy male and non-childbearing potential female participants Part B: Pre-randomization PVR ≥400 dyn.sec.cm
- •Part A: In good health in the opinion of the investigator and as determined by medical history, physical examination, vital signs, ECG, and laboratory tests. Part B: Treatment with stable doses of standard-of-care PAH therapies
- •Part A: Able to read, speak, and understand the local language, to understand and comply with the requirements of the study. Part B: 6MWD ≥ 150 m and ≤450 m
排除标准
- •Part A: History or current diagnosis of clinically significant ECG or cardiac abnormalities indicating significant risk of safety for participants. Part B: Any surgical or medical condition which may place the participant at higher risk from his/her participation in the study
- •Part A: Use of any investigational drugs within 5 half-lives or within 30 days or until the expected pharmacodynamic effect has returned to baseline prior to initial dosing, whichever is longer. Part B: Women of child-bearing potential unless they are using highly effective methods of contraception
- •Part A: History of hypersensitivity to the investigational compound/compound class or excipients being used in this study. Part B: Sexually active males unwilling to use a condom during intercourse while taking study treatment and for 24 hours after stopping study treatment.
- •Part B: History of hypersensitivity to any of the study treatments or excipients
结局指标
主要结局
Part A: All safety endpoints, including vital signs, ECG, central lab evaluations and AEs up to and including the EoS visit. Part B: (Period 1) Change in PVR from baseline to 24 weeks (Period 2): All safety endpoints, including vital signs, ECG, central lab evaluations, treatment-emergent AEs and discontinuations due to AEs.
Part A: All safety endpoints, including vital signs, ECG, central lab evaluations and AEs up to and including the EoS visit. Part B: (Period 1) Change in PVR from baseline to 24 weeks (Period 2): All safety endpoints, including vital signs, ECG, central lab evaluations, treatment-emergent AEs and discontinuations due to AEs.
次要结局
- Part A: Cmax, Tmax, AUClast, AUCinf and T1/2 of LTP001 Part B: (Period 1) Change in 6MWD from baseline to 24 weeks. (Period 2) Change in 6MWD from baseline to 18 months
- Part B Period 2: Number of participants experiencing an improvement in WHO FC from baseline to 18 months
- Part B (Period 1): Change in WHO FC from baseline to 24 weeks
- Part B (Period 1): All safety endpoints, including vital signs, ECG, clinical laboratory evaluations, treatment-emergent adverse events and discontinuations due to adverse events
研究者
Novartis Pharma Arzneimittel GmbH
Scientific
Novartis Pharma AG