Study of Montelukast In Children with Sickle Cell Disease

Phase 1

• Conditions: Sleep-disordered breathing; MedDRA version: 20.0Level: LLTClassification code 10078294Term: Obstructive sleep apnoea hypopnoea syndromeSystem Organ Class: 100000004855; MedDRA version: 21.0Level: LLTClassification code 10040977Term: Sleep apnoeaSystem Organ Class: 100000004855; Therapeutic area: Psychiatry and Psychology [F] - Psychological processes [F02]

• Registration Number: EUCTR2017-004539-36-GB
• Lead Sponsor: Great Ormond Street Hospital for Children NHS Foundation Trust

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-10-14
• Last Update Posted: 19 October 2020

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 200

Inclusion Criteria

•Aged between 3 and <8 years
•Informed consent with assent in accordance with institutional policies and European guidelines; ICF must be signed by patients/guardian
•HbSS or HbSß0 thalassaemia diagnosed by standard techniques (HPLC, IEF, MS 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<93%; mean SO2<96%))
•In steady state i.e. haemoglobin not decreased by >10% in previous year, no painful crisis requiring opioids for at least 1 month and at least 1 month after any hospital admission
•Likely to comply with treatment for 3 months as determined by the local paediatrician or hematologist
•Able to speak and understand English

Are the trial subjects under 18? yes
Number of subjects for this age range: 200
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range 0
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0

Exclusion Criteria

•Other developmental or psychiatric disorders including craniofacial abnormalities, neuromuscular disorder and other chronic conditions.
•Patient already on montelukast
•Patient has had side effects on or an adverse reaction to montelukast in the past
•Presence or history of another hemoglobinopathy or blood dyscrasia
•Concomitant treatment with any other leukotriene antagonist within a window period of enrolment
•Participation on other SCD/Montelukast trials

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To assess the effect of Montelukast on neurocognition, specifically processing speed measured as Cancellation (Wechsler scales) and NIH toolbox processing speed.;Secondary Objective: •To assess the effect of Montelukast on <br>-adenoid:nasopharynx size <br>-brain MRI<br>-overnight oximetry,<br>-symptoms of sleep-disordered breathing, e.g. snoring<br>-sleep duration and <br>-Pain <br>•To assess side-effects of Montelukast<br>•To assess feasibility of MRI in children aged 3-<8 years.<br><br><br>;Primary end point(s): The trial is powered to look at 2 primary outcomes<br>1. Cancellation (paper-and-pencil test of processing speed and attention from the Wechsler scales)<br>2. Processing speed on the NIH toolbox<br>;Timepoint(s) of evaluation of this end point: Baseline before treatment and after 12 weeks of treatment

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): oNIH toolbox executive function <br>oDuration of Sleep from Children’s Sleep Habit questionnaire (CSHQ)<br>oSymptoms of Sleep Disordered Breathing from Pediatric Sleep questionnaire (PSQ)<br>oPain burden from sickle module of PEDSQL<br>oAEs & drug side-effects<br><br>* Additional secondary endpoints in patients willing to undergo optional additional tests (MRI without sedation (awake/asleep); overnight oximetry):<br><br>oSelf-rated pain diaries via trial smartphone app<br>oCore MRI endpoints: Multiparametric maps (MPM) for adenoidal and brain size, Volumetrics(T1 MRI), White matter integrity and structural connectivity (DWI), <br><br>*Additional MRI endpoints if tolerated:<br> <br>Perfusion (ASL), Functional Connectivity (rsFMRI), Multiparametric maps (MPM), Oxygen-extraction fraction (TRUST)<br>;Timepoint(s) of evaluation of this end point: Baseline before treatment and after 12 weeks of treatment