Study of Montelukast In ChiLdrEn With Sickle Cell Disease (SMILES)

Study point of contact

Satwinder Sahota, MSc
[email protected]
Fenella Kirkham, MA MB Bchir
+44 207 9052191
[email protected]


4 United Kingdom sites


3 to 8 Years




Phase 2/Phase 3

Study type









About the study

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.

participation requirements

– 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

participation restrictions

– Other neurodevelopmental disorders

– Patient already on Montelukast

– Patient has had side effects on or an adverse reaction to Montelukast in the past


  • London, United Kingdom, North Middlesex University Hospital NHS Trust, N18 1QX
  • London, United Kingdom, University College London Hospitals NHS Trust, NW1 2BU
  • London, United Kingdom, Guys & St Thomas Hospital NHS Trust, SE1 9RT
  • London, United Kingdom, King's College Hospital NHS Foundation Trust, SE5 9RS