SMILES: Study of Montelukast in Sickle Cell Disease

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

Interventions

DRUG

Montelukast

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.

OTHER

Oral Placebo

Inert chewable tablet with no therapeutic value

Sponsors & Collaborators

• Guy's and St Thomas' NHS Foundation Trust

  collaborator OTHER

• University College London Hospitals

  collaborator OTHER

• North Middlesex University Hospital

  collaborator OTHER

• King's College Hospital NHS Trust

  collaborator OTHER

• The Whittington Hospital NHS Trust

  collaborator OTHER_GOV

• Great Ormond Street Hospital for Children NHS Foundation Trust

  lead OTHER

Principal Investigators

• Fenella Kirkham, MA MB Bchir · University College, London

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

SINGLE

Model

PARALLEL

Eligibility

Min Age

3 Years

Max Age

8 Years

Sex

ALL

Healthy Volunteers

Yes

Timeline & Regulatory

Start

2023-10-16

Primary Completion

2024-09-30

Completion

2024-09-30

Countries

• United Kingdom

Study Locations