SMILES: Study of Montelukast in Sickle Cell Disease

NCT04351698 | Unknown Phase 2 DMC

• 1 other identifier: Protocol Number: 16NC06
• Study Type: interventional
• Target: 200
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Anemia, Sickle Cell; Sleep-Disordered Breathing

Outcome Measures

Primary Outcomes (2)

• 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

• 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 Outcomes (6)

• 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.

  12 weeks

• 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.

  12 weeks

• 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.

  12 weeks

• Change in pain symptoms from baseline to 12 weeks for participants with sickle cell disease randomised to the control arm or the treatment arm.

  12 weeks

• Number of Participants With Treatment-Related Adverse Events from Montelukast

  12 weeks

Study Arms (2)

Investigation

ACTIVE COMPARATOR

Montelukast

Drug: Montelukast

Match Placebo

PLACEBO COMPARATOR

Matched Placebo

Other: Oral Placebo

Interventions

Montelukast DRUG

Montelukast is used to treat and prevent asthma. It will decrease the symptoms and the number of acute asthma attacks. However this medicine should not be used to relieve an asthma attack that has already started.

Also known as: singulair

Investigation

Oral Placebo OTHER

Inert chewable tablet with no therapeutic value

Match Placebo

Eligibility Criteria

• Age: 3 Years - 8 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• Other neurodevelopmental disorders
• Patient already on Montelukast
• Patient has had side effects on or an adverse reaction to Montelukast in the past

Sponsors & Collaborators

• Great Ormond Street Hospital for Children NHS Foundation Trust: lead
• Guy's and St Thomas' NHS Foundation Trust: collaborator
• University College London Hospitals: collaborator
• North Middlesex University Hospital: collaborator
• King's College Hospital NHS Trust: collaborator
• The Whittington Hospital NHS Trust: collaborator

Study Sites (1)

Great Ormond Street Hospital NHS Foundation Trust

London, WC1N 1EH, United Kingdom

RECRUITING

Related Publications (1)

• Hood AM, Stotesbury H, Kolbel M, DeHaan M, Downes M, Kawadler JM, Sahota S, Dimitriou D, Inusa B, Wilkey O, Pelidis M, Trompeter S, Leigh A, Younis J, Drasar E, Chakravorty S, Rees DC, Height S, Lawson S, Gavlak J, Gupta A, Ridout D, Clark CA, Kirkham FJ. Study of montelukast in children with sickle cell disease (SMILES): a study protocol for a randomised controlled trial. Trials. 2021 Oct 10;22(1):690. doi: 10.1186/s13063-021-05626-6.

  PMID: 34629091 DERIVED

MeSH Terms

Conditions

Anemia, Sickle Cell; Sleep Apnea Syndromes

Interventions

montelukast

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Apnea; Respiration Disorders; Respiratory Tract Diseases; Sleep Disorders, Intrinsic; Dyssomnias; Sleep Wake Disorders; Nervous System Diseases

Study Officials

• Fenella Kirkham, MA MB Bchir

  University College, London

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Fenella Kirkham, MA MB Bchir

CONTACT

+44 207 9052191; fenella.kirkham@ucl.ac.uk

Satwinder Sahota, MSc

CONTACT

+442079052191; s.sahota@ucl.ac.uk

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 2, 2019
• First Posted: April 17, 2020
• Study Start: October 16, 2023
• Primary Completion: September 30, 2024
• Study Completion: September 30, 2024
• Last Updated: November 1, 2023

Record last verified: 2023-10

Locations

GB (1)