E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Otitis media with effusion. |
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E.1.1.1 | Medical condition in easily understood language |
Otitis media with effusion is commonly known as glue ear. It is a common childhood condition in which the middle ear becomes filled with fluid. The main symptom of glue ear is hearing loss. |
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E.1.1.2 | Therapeutic area | Diseases [C] - Ear, nose and throat diseases [C09] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10040113 |
E.1.2 | Term | Serous otitis media (glue ear) |
E.1.2 | System Organ Class | 100000004862 |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To determine the clinical and cost effectiveness of a 7 day course of oral prednisolone (steroid) on improving hearing in the short term in children with bilateral OME with confirmed hearing loss for at least 3 months. |
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E.2.2 | Secondary objectives of the trial |
To assess the longer–term (up to 12 months) effect of the oral steroid intervention on hearing loss, resolution of OME and grommet surgery rates. To assess the short-term (up to five weeks) effectiveness of oral steroid on subjective severity, adverse events, impact on family, and daily symptoms. To asses the short and long-term effects of treating OME with oral steroids, on functional health status and quality of life. To assess the short and long-term cost-effectiveness of treating OME with oral steroids, in terms of cost per unit reduction in subjective outcome severity. To undertake an exploratory cost utility analysis using a condition specific measure of health for children. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
•2-8 years of age (reached 2nd birthday and not yet reached 9th birthday), •Had symptoms of hearing loss attributable to OME for at least 3 months (or had audiometry proven hearing loss for at least 3 months), •Diagnosis of bilateral OME made in an ENT clinic on the day of recruitment or during the preceding week. •Audiometry confirming hearing loss of more than 20 dB averaged at 0.5, 1, 2, and 4 KHz in the better ear by pure tone audiometry in children 3 years of age or more or hearing loss of more than 25 dB averaged over 0.5, 1, 2, and 4 KHz by sound field visual reinforcement audiometry (VRA) in children less than 3 years of age, on the day of recruitment or in the preceding week. |
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E.4 | Principal exclusion criteria |
•Children with cleft palate, •Children with Down’s syndrome, •Children with confirmed, major developmental difficulties (e.g. are tube fed, have chromosomal abnormalities), •Children with current systemic infection (including Varicella), •Children with renal failure, hypertension or congestive heart failure, •Children with diabetes mellitus, •Children who have taken oral steroids in the preceding four weeks, •Children with a condition that increases their risk of adverse effects from oral steroids (i.e. on treatment likely to modify the immune system or who are immunocompromised), •Children with no prior history of Varicella (Chicken Pox) infection or immunisation and who have been in close contact with someone known or suspected to have Varicella or active Zoster (Shingles) during the three weeks prior to recruitment, •Children who are currently involved in another CTIMP or have participated in a CTIMP during the last 4 months. Or if they do not have: • A parent or legal guardian who is available to provide written informed consent. • A parent/legal guardian (or a person delegated by the parent/legal guardian) who expects to be available for follow up visits at 5 weeks, 6 and 12 months and who understands English well enough to complete simple verbal and written questionnaires. |
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary outcome will be acceptable hearing at five weeks from randomisation (four weeks after conclusion of treatment), where acceptable hearing is defined as ‘less than 20 dB averaged at 0.5, 1, 2 and 4 kHz in at least one ear in children aged 3-8 years, and less than 25 dB averaged at 0.5, 1, 2 and 4 KHz by sound field VRA in children aged under 3 years. These thresholds are based on national guidelines (Newborn hearing screening and assessment Programme (NHSP) Clinical Group 2008). |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
The primary endpoint is satisfactory hearing. At baseline, 5 week, 6 month and 12 month follow ups, participants will attend the ENT outpatients clinic for an audiology assessment. This will determine whether hearing loss has been resolved. |
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E.5.2 | Secondary end point(s) |
- Daily symptoms (reported by parent and/or child) - Adverse events - Medication use - Tympanometry (using calibrated standardised tympanometers and modified Jeger classification Types B and C2) - Otoscopy findings - Grommet surgery - Health-related quality of life (PedsQL) - Functional Health status (OM8-30 and HUI3) - Healthcare consultations related to OME, and other resource use - Short and long-term cost effectiveness |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
- Daily symptoms, adverse events and medication use - during study treatment and subsequent 4 weeks. - Tympanometry and otoscopy- baseline and 5 weeks, 6 months, 12 month follow ups. - Grommet surgery - 5 weeks, 6 months, 12 month follow ups. - PedsQL, OM8-30 and HUI3 - baseline and 5 weeks, 6 months, 12 month follow ups. -Healthcare consultations related to OME, and other resource use. Parent reported use at 5 weeks, 6 months, 12 month follow ups, with data extraction from secondary care notes review at 12 months post recruitment. - Short and long-term cost effectiveness conducted at data analysis stage. |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
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E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 7 |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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LVLS. This represents the last participant consented and randomised to receive IMP, who completes all the data collection time points as stated in the protocol, including the 12 month follow up. |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 3 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 3 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |