Study of Montelukast In ChiLdrEn With Sickle Cell Disease (SMILES)
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 200
- 试验地点
- 1
- 主要终点
- Change in processing speed scores from baseline to 12 weeks for participants with sickle cell disease randomised to the control arm or the treatment arm.
研究概览
简要总结
Sickle cell disease (SCD) is a genetic blood condition causing long term health problems including pain and brain problems which affect quality of life. These may be made worse if patients have low night-time oxygen levels when the upper airways close repeatedly during the night (obstructive sleep apnoea). This is associated with increased pain, poorer concentration and increased kidney problems. Montelukast, widely used in the treatment of Asthma, has been shown to improve symptoms of obstructive sleep apnoea in patients without sickle cell anaemia. Investigators think this treatment could be useful in patients with sickle cell disease too. Early intervention with Montelukast could help prevent deterioration in concentration and thinking skills.
The aim of this trial is to see whether young children with sickle cell disease randomised (randomise: the same as tossing a coin and not knowing whether it will come up heads or tails) to Montelukast treatment have better thinking skills compared with people randomised to placebo (tablet with no active medical ingredients - i.e. "sugar pill"). This means that the child could be on Montelukast treatment or he/she might be on placebo tablets.
详细描述
This trial, funded by Action Medical Research, will involve 200 SCD patients recruited from haematology clinics and ENT (ear, nose and throat) surgery waiting lists or their local sleep laboratory at Evelina Children's hospital (Guy's & St Thomas), University College London hospital, North Middlesex hospital and Whittington Hospitals. Patients are eligible if they are between 3 and <8 years old w SCD, give informed consent, have any Sleep-Disordered Breathing, e.g. snoring, and/or any abnormality on overnight oximetry. After consenting, the PI/Coordinator will organise a simple app-based questionnaire and baseline testing of brain speed, with an optional brain scan, and randomisation to chewable Montelukast tablets or Placebo for 3 months after which the brain speed tests, questionnaires and MRI will be repeated.
Purpose and design:
This is designed as a phase 2/3 trial of Montelukast therapy to investigate whether this drug improves processing speed, which appears to underpin the difference in full scale IQ (intelligence quotient) United Kingdom.
between children with sickle cell disease and their siblings.
Recruitment:
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
- Single (Outcomes Assessor)
入排标准
- 年龄范围
- 3 Years 至 8 Years（Child）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Aged between 3 and <8 years
- •Informed consent with assent in accordance with institutional policies and European guidelines; ICF (informed consent form) must be signed by patients/guardian
- •HbSS 9homozygous SS disease) or HbSβ0 thalassaemia diagnosed by standard techniques (HPLC, IEF (Isoelectric focusing), MS (Mass spectrometry) or AlkE)
- •History of Sleep-Disordered Breathing, (i.e. parent-reported any degree of snoring (CHSQ questionnaire) and/or any abnormality on overnight oximetry compared with published data in children of the same age (e.g. nadir SO2 (oxygen saturation) <93%; mean SO2<96%))
- •Able to speak and understand English
排除标准
- •Other neurodevelopmental disorders
- •Patient already on Montelukast
- •Patient has had side effects on or an adverse reaction to Montelukast in the past
研究组 & 干预措施
Investigation
Montelukast
干预措施: Montelukast (Drug)
Match Placebo
Matched Placebo
干预措施: Oral Placebo (Other)
结局指标
主要结局
Change in processing speed scores from baseline to 12 weeks for participants with sickle cell disease randomised to the control arm or the treatment arm.
时间窗: 12 weeks
Cancellation (Wechsler scales and paper-and-pencil)
次要结局
- Change in executive function (from NIH Toolbox® Cognition Battery) from baseline to 12 weeks for participants with sickle cell disease randomised to the control arm or the treatment arm.(12 weeks)
- Change in executive function (parent reported questionnaire) from baseline to 12 weeks for participants with sickle cell disease randomised to the control arm or the treatment arm.(12 weeks)
- Change in sleep and respiratory symptoms from baseline to 12 weeks for participants with sickle cell disease randomised to the control arm or the treatment arm.(12 weeks)
- Change in pain symptoms from baseline to 12 weeks for participants with sickle cell disease randomised to the control arm or the treatment arm.(12 weeks)
- Number of Participants With Treatment-Related Adverse Events from Montelukast(12 weeks)
- Change in Brain MRI from baseline to 12 weeks for participants with sickle cell disease randomised to the control arm or the treatment arm.(12 weeks)