SMILES: Study of Montelukast in Sickle Cell Disease

Launched by GREAT ORMOND STREET HOSPITAL FOR CHILDREN NHS FOUNDATION TRUST · Apr 15, 2020

Keywords

ClinConnect Summary

The SMILES trial is studying whether a medication called Montelukast can help improve thinking skills in young children with sickle cell disease (SCD) who also have sleep-disordered breathing, which means they have trouble breathing normally during sleep. Sickle cell disease can cause serious health issues, including pain and problems with thinking, especially when a child’s oxygen levels drop at night. Montelukast is commonly used to treat asthma and has shown promise in improving sleep-related breathing issues in other patients. The researchers hope that giving Montelukast to children with SCD might help them think better and reduce some of their health problems.

To participate in this trial, children must be between 3 and 8 years old and diagnosed with a specific type of sickle cell disease. They should also have a history of sleep-disordered breathing, like snoring. To join, parents or guardians will need to sign a consent form. Participants will be randomly assigned to receive either Montelukast or a placebo (a sugar pill that does not have the active medication) and will be monitored for changes in their thinking skills. The trial is currently looking for participants, and all children, regardless of gender, are welcome to apply.

Gender

ALL

Eligibility 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