A Phase 2/3, Prospective, Open-label Trial Evaluating the Efficacy, Safety, and Pharmacokinetics of Remimazolam for Intravenous Sedation in Paediatric Patients Undergoing Diagnostic and/or Therapeutic Procedures
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 100
- 试验地点
- 8
- 主要终点
- Success of the procedure
研究概览
简要总结
To assess the efficacy of intravenous remimazolam in inducing and maintaining suitable sedation levels for paediatric patients undergoing diagnostic and/or therapeutic procedures
详细描述
This trial is part of the European Paediatric Investigational Plan and the US Pediatric Study Plan and has been developed in line with guidance from the EMA Paediatric Committee and the US FDA.
The trial will commence with cohort 1 (aged ≥6 and <18 years) and proceed to lower age groups: cohort 2 (≥3 and <6 years); and, in European sites only, cohort 3 (full-term birth to <3 years). The Data Monitoring committee (DMC) may convene any time after at least half of the subjects in cohort 1 or 2 have completed the study, to review PK,safety and efficacy data. If there are no concerns, the DMC may recommend to initiate concurrent dosing in the next younger cohort (cohort 2 or 3) in parallel to dosing the remaining subjects in cohort 1 or 2 Dosing for cohort 3 will be predicted based on PK modelling as well as efficacy and safety outcomes of the older age groups. Enrolment of patients aged <2 years will not be permitted until supported by adequate juvenile toxicity data.
The trial will consist of three visits: Screening (Day -21 to day 1),Treatment (Day 1), and Follow-up (Day 4 [+3/-1 days]).
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Supportive Care
- 盲法
- None
入排标准
- 年龄范围
- 0 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Signed informed consent form and/or assent and willingness of patient and parent(s) to participate in the trial.
- •In US sites: Paediatric male or female patients, aged ≥3 and <18 years scheduled to undergo a diagnostic or therapeutic procedure, which is medically indicated and independent from the trial.
- •In European sites: Paediatric male or female patients, aged full term birth to <18 years scheduled to undergo a diagnostic or therapeutic procedure, which is medically indicated and independent from the trial.
- •Maximum planned duration of procedure: 2 hours
- •ASA Physical Status I-III
- •Planned spontaneous breathing during sedation
- •A female who is of child bearing potential (i.e. after menarche) and sexually active must use a highly effective method of birth control during the trial period (from the time of consent until all specified observations are completed)
- •Negative pregnancy test at screening and on treatment day -
排除标准
- •Emergency procedures
- •Condition/procedure that requires planned airway control via endotracheal tube or LMA/IGEL insertion
- •Cranio-facial malformation, which would severely limit the possibilities for emergency airway rescue
- •Other abnormalities relating to the airway (including large tonsils and anatomical abnormalities of upper airway or lower airway) which may compromise emergency airway rescue
- •Known hypersensitivity to benzodiazepines, flumazenil, dextran or any of the ingredients of the drug product
- •Known paradoxical reactions to benzodiazepines
- •History of sleep apnoea
- •Active respiratory failure
- •Active neuromuscular disease
- •Active cardiac failure
- •Active hepatic failure
- •Breast feeding females
- •Prohibited medication
- •Any patient judged by the Principal Investigator (PI) or Sub-Investigator to be inappropriate for the trial for any other reason
研究组 & 干预措施
All Patients
All paediatric patients undergoing diagnostic and/or therapeutic procedures
干预措施: Remimazolam (Drug)
结局指标
主要结局
Success of the procedure
时间窗: 2 hours
Success of the procedure defined as: Completion of the procedure AND no requirement for rescue sedative medication AND no requirement for more than the permitted bolus or infusion regimen
次要结局
- Signs of re-sedation(2 hours)
- Time to start of procedure(2 hours)
- Procedure success excluding cases where the procedure could not be completed for non sedative reasons(2 hours)
- Percentage of time within target range of sedation(2 hours)
- Safety: AEs(4 days)
- Safety: emergence of delirium(2 hours)
- Target depth of sedation achieved(2 hours)
- Adequacy of sedation(2 hours)
- Time to fully alert(2 hours)
- Target range of sedation achieved during 80% of procedure duration(2 hours)
- Time to ready for discharge(2 hours)
- Safety: need for reversal(2 hours)
- PK: assessment of plasma concentration-time relationship(3.5 hours)
- Safety: need for ventilation(2 hours)