Anemia, Sickle Cell Clinical Trial
— SMILESOfficial title:
Study of Montelukast In ChiLdrEn With Sickle Cell Disease (SMILES)
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.
Status | Recruiting |
Enrollment | 200 |
Est. completion date | September 30, 2024 |
Est. primary completion date | September 30, 2024 |
Accepts healthy volunteers | Accepts Healthy Volunteers |
Gender | All |
Age group | 3 Years to 8 Years |
Eligibility | 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 HbSß0 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 |
Country | Name | City | State |
---|---|---|---|
United Kingdom | Great Ormond Street Hospital NHS Foundation Trust | London |
Lead Sponsor | Collaborator |
---|---|
Great Ormond Street Hospital for Children NHS Foundation Trust | 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 |
United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | 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. | NIH toolbox processing speed | 12 weeks | |
Primary | 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. | Cancellation (Wechsler scales and paper-and-pencil) | 12 weeks | |
Secondary | 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. | Dimensional Change Card Sort Test: There are 40 trials, and scoring was based on a combination of accuracy and reaction time.
Flanker Inhibitory Control and Attention Test: There are 40 trials. For children aged 3-7.99 years scores of = 90% using preliminary fish stimuli were followed that an additional 20 trials using arrows. Scoring was based on a combination of accuracy and reaction time. List Sorting Working Memory Test: The number of items in series increases from 2 to a maximum of 8. The test was discontinued when 2 trials at the same length were failed. Responses were entered by the examiner on a wireless keyboard. Children under 7 years did not complete this test. Scoring was based on the total number of items correctly recalled across all trials. Each measure will be analysed separately and as a combined composite. Higher scores indicate better executive function (mean = 100, standard deviation = 15, range = 50 - 150). |
12 weeks | |
Secondary | 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. | Behavioral Rating Inventory of Executive Function (BRIEF-2) is an 63-item questionnaire that assesses EF behaviours in the school and home environments. The Global Executive Composite, Behavioural Regulation, and Emotion Regulation indices will be used in analyses. Higher T-scores (> 65) indicate more clinical concern (range = 0 - 100). | 12 weeks | |
Secondary | 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. | Children's Sleep Habit questionnaire (CSHQ): a 45-item parent-rated questionnaire that assesses the frequency of behaviors associated with common paediatric sleep difficulties (range = 0 - 135).
Pediatric Sleep Questionnaire: a 22-item parent-rated questionnaire that asks about snoring frequency, loud snoring, observed apneas, difficulty breathing during sleep, daytime sleepiness, inattentive or hyperactive behaviour (range = 0 - 22). Total sleep time/total scores will be used in analyses. Higher scores are of greater clinical concern. |
12 weeks | |
Secondary | Change in pain symptoms from baseline to 12 weeks for participants with sickle cell disease randomised to the control arm or the treatment arm. | Pain and hurt and pain management and control scales from the PedsQL - Sickle Cell Module: The PedsQL is a 43-item questionnaire with 9 dimensions that assesses health-related quality of life in individuals with SCD (pain and hurt range = 0 - 36, pain management and control range = 0 - 8).
Total scores will be used in analyses. Lower scores are of greater concern. |
12 weeks | |
Secondary | Number of Participants With Treatment-Related Adverse Events from Montelukast | All AEs (adverse events), hospital attendances, days of illness, and days lost from pre-school/school to be recorded on CRFs (case report forms) by coordinator and encrypted and stored separately | 12 weeks | |
Secondary | Change in Brain MRI from baseline to 12 weeks for participants with sickle cell disease randomised to the control arm or the treatment arm. | Adenoidal size, volumetrics, white-matter integrity, structural and functional connectivity, perfusion) and nocturnal oximetry will be measured. | 12 weeks |
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