SMILES: Study of Montelukast in Sickle Cell Disease

• Status: Not yet recruiting
• Trial end date: 2022-08-31
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: Accepts Healthy Volunteers

Details

• Lead Sponsor: Great Ormond Street Hospital for Children NHS Foundation Trust
• Collaborators: Guy's and St Thomas' NHS Foundation Trust; King's College Hospital NHS Trust; North Middlesex University Hospital; The Whittington Hospital NHS Trust; University College London Hospitals
• Treatments: Montelukast

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