Montelukast treatment for children with sickle cell disease
Official title: Study of Montelukast In ChiLdrEn With Sickle Cell Disease (SMILES)
Run by Great Ormond Street Hospital for Children NHS Foundation Trust · NCT04351698 · Data updated 2026-05-14
This study is no longer in our active list. It may have stopped recruiting or finished. The details below are as of 2026-05-14 and are kept for reference. Check ClinicalTrials.gov for its current status, or see similar studies below.
This study is testing if the asthma medication Montelukast can help young children with sickle cell disease and sleep problems think better and improve their brain function.
Quick facts
| Phase | Phase2; Phase3 |
|---|---|
| Study type | Interventional |
| Ages | 3 Years to 8 Years |
| Sex | All |
| Enrollment | 200 (estimated) |
| Sponsor | Great Ormond Street Hospital for Children NHS Foundation Trust Academic / other |
| Locations | 1 site (London) |
| Trial ID | NCT04351698 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates are children aged 3 to 8 years with sickle cell disease and a history of sleep-disordered breathing.
✕Probably not a fit
Patients with other neurodevelopmental disorders or those who have previously experienced adverse reactions to Montelukast may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This trial investigates the effects of Montelukast, a medication commonly used for asthma, on young children with sickle cell disease who also experience sleep-disordered breathing. The study aims to determine if Montelukast can improve cognitive processing speed and overall brain function in these patients. A total of 200 participants aged 3 to 8 years will be recruited from various hospitals and will be randomly assigned to receive either Montelukast or a placebo for three months. Assessments will include brain speed tests and optional MRI scans before and after the treatment period.
Why it matters
Potential benefit: If successful, this treatment could enhance cognitive function and quality of life for children with sickle cell disease.
How similar studies have gone: Previous studies have shown that Montelukast can improve symptoms of sleep-disordered breathing in other populations, suggesting potential for success in this novel application.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: * 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 Exclusion Criteria: * Other neurodevelopmental disorders * Patient already on Montelukast * Patient has had side effects on or an adverse reaction to Montelukast in the past
Where it's running
-
Great Ormond Street Hospital NHS Foundation Trust
London, United Kingdom Recruiting
Study contacts
- Principal investigator: Fenella Kirkham, MA MB Bchir — University College, London
- Study coordinator: Fenella Kirkham, MA MB Bchir
- Email: fenella.kirkham@ucl.ac.uk
- Phone: +44 207 9052191
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.