ANZCTR - Registration

Please note the ANZCTR will be unattended from Friday 20 December 2024 for the holidays. The Registry will re-open on Tuesday 7 January 2025. Submissions and updates will not be processed during that time.

Registering a new trial?

To achieve prospective registration, we recommend submitting your trial for registration at the same time as ethics submission.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers

Trial registered on ANZCTR

Registration number

ACTRN12615000563561

Ethics application status

Approved

Date submitted

7/05/2015

Date registered

1/06/2015

Date last updated

8/03/2021

Date data sharing statement initially provided

30/07/2019

Type of registration

Prospectively registered

Titles & IDs

Public title

Bell's Palsy in Children: A Multi-centre, double-blind, Randomised, Placebo-controlled Trial to Determine Whether Prednisolone Improves Recovery at 1 Month.

Scientific title

A multi-centre randomised controlled trial of children aged between 6 months and 18 years presenting to emergency departments with symptoms of Bell's palsy, to determine whether treatment with oral prednisolone versus a placebo, increases the proportion of children who have complete recovery at 1 month, where complete recovery is defined as grade 1 on the House Brackmann scale.

Secondary ID [1]

Nil

Universal Trial Number (UTN)

U1111-1169-9918

Trial acronym

BellPIC

Linked study record

Health condition

Health condition(s) or problem(s) studied:

Bell's Palsy

Condition category

Condition code

Neurological

Other neurological disorders

Inflammatory and Immune System

Other inflammatory or immune system disorders

Intervention/exposure

Study type

Interventional

Description of intervention(s) / exposure

Intervention: Prednisolone.
Prednisolone is a corticosteroid that has strong evidence for effectiveness in reducing the length of facial palsy symptoms in adults suffering from Bell's palsy.
Participants assigned to the intervention will receive 1mg/kg/day of prednisolone (dosing is based on weight categories) up to a maximum of 50mg/day for 10 days.
The prednisolone will be administered as an oral solution as a once daily dose.
Strategies used to monitor adherence will be a survey relating to compliance at the end of the 10 day course of treatment, in addition to the return of the bottles.

Intervention code [1]

Treatment: Drugs

Comparator / control treatment

control treatment: placebo.
The placebo will be supplied in glass bottles identical to the intervention by the same supplier that produces the intervention. It will be manufactured to look and taste identical to that of the intervention
The placebo will be an oral liquid with the same ingredients as the intervention solution minus the active drug, prednisolone.

Control group

Placebo

Outcomes

Primary outcome [1]

The primary outcome is complete recovery at one month post randomisation, where recovery is defined as a House Brackmann facial grading score of 1. Recovery will be assesed by a specialist clinician in a face to face setting

Timepoint [1]

One month post randomisation

Secondary outcome [1]

Complete recovery at 1 month using the Sunnybrook facial grading scale assessed by a specialist physician

Timepoint [1]

One month post randomisation

Secondary outcome [2]

Complete recovery at 3 months using the Sunnybrook facial grading scale assessed by a research assistant and/or specialist physician

Timepoint [2]

Three months post randomisation

Secondary outcome [3]

Complete recovery at 3 months using the House Brackmann facial grading scale assessed by a research assistant and/or specialist physician

Timepoint [3]

Three months post randomisation

Secondary outcome [4]

Complete recovery at 6 months using the Sunnybrook facial grading scale assessed by a research assistant and/or specialist physician

Timepoint [4]

Six months post randomisation

Secondary outcome [5]

Complete recovery at 6 months using the House Brackmann facial grading scale assessed by a research assistant and/or specialist physician

Timepoint [5]

6 months post randomisation

Secondary outcome [6]

Parent/guardian and participant (where aged >8 years) perception of facial nerve recovery at 1, 3 and 6 months using a lay translation of the House Brackmann facial grading scale

Timepoint [6]

One, three and six months post randomisation

Secondary outcome [7]

Quality of Life: Emotional and functional wellbeing of the participant assessed by the parent/guardian and participant using the Pediatric Quality of Life Inventory scale

Timepoint [7]

One, three and six months post randomisation

Secondary outcome [8]

Pain assessed using child assigned visual analogue scale or Faces Pain Scale Revised (for participants aged 5 and older) and using parent assigned VAS for participants at any age. Pain scales are numbered 0 to 10

Timepoint [8]

One, three and six months post randomisation

Secondary outcome [9]

Prevalence of sykinesis or autonomic dysfunction using the Sunnybrook scale augmented by a synkinesis assessment questionnaire, assessed by a specialist clinician and/or research assistant

Timepoint [9]

One, three and six months post randomisation

Secondary outcome [10]

Health utilisation costs assessed via Child Health Utility 9D and via capture of cost information from the parent/guardian/participant related to in-patient, out-patient, or ED visits and to any other health facilities including general practitioner attendance for treatment or investigation

Timepoint [10]

6 months following randomisation

Secondary outcome [11]

Quality of Life: Emotional and functional wellbeing of the participant assessed by the parent/guardian and participant using the Child Health Utility 9D scale and sections of the Harter scale

Timepoint [11]

One, three and six months post randomisation

Secondary outcome [12]

Quality of life: Emotional and functional wellbeing of the participant assessed by the parent/guardian and participant using sections of the Harter scale

Timepoint [12]

One, three and six months post randomisation

Eligibility

Key inclusion criteria

Patients must be aged between 6 months to less than 18 years, weigh greater than or equal to 5kg, be diagnosed with Bell's Palsy by their treating doctor and have an acute onset of symptoms of Bell's palsy for less than 72 hours prior to randomisation

Minimum age

6 Months

Maximum age

18 Years

Sex

Both males and females

Can healthy volunteers participate?

Key exclusion criteria

- Previous episode of Bell's Palsy or previously randomised in BellPIC study
- Contraindication to prednisolone: active or latent tuberculosis, systemic fungal infection, known hypersensitivity to prednisolone, diminished cardiac function, diabetes mellitus, peptic ulcer or chronic renal function, multiple sclerosis or recent active herpes zoster or chickenpox
- current use of systemic or inhaled steroid, or use within 2 weeks prior to the onset symptoms
- current or past oncological diagnosis
- FBE result (if obtained during this illness) may indicate leukaemia
- Pregnancy and/or lactating
- Currently receiving medications for which prednisolone is contraindicated
- Immunisation with a live vaccine within the previous 1 month
- Requirement for live vaccine within 6 weeks of first dose of prednisolone
- Signs of upper motor neuron VII nerve palsy (weakness of lower half of the face only)
- Current or recent (1 week prior to Bell's palsy symptoms) otitis media
- Evidence of vesicles on the ear from or vesicles/ulcers elsewhere on the body suggestive of recent herpes simplex, herpes zoster or chickenpox
- Known significant facial trauma within 1 week prior to symptoms appearing
- Referred to GP clinic in Emergency Department or failed to wait
- Unable to attend a follow up visit in one month time
- Any other condition at risk of being influenced by study treatment or completion of study
- Parents will not be able to comply with the study, or have enough understanding of the study.

Study design

Purpose of the study

Treatment

Allocation to intervention

Randomised controlled trial

Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)

in the event of a child being diagnosed with Bell's palsy, a senior doctor from the ED or a member of the resarch team will review the medical history for initial determination of eligibility. They will then approach the potential participant to explain the study to them, and conduct the informed consent process.
Once written informed consent has been obtained, demographic information and medical history will be elicited. Study eligibility will be confirmed and the patient will be randomised.
To randomise the patient, the senior doctor or the research team will select the lowest number study pack. This study pack will either contain prednisolone or placebo made up by the central study pharmacist according to a computer generated randomisation schedule.

Methods used to generate the sequence in which subjects will be randomised (sequence generation)

Participants will be randomised stratified by site in a 1:1 ratio to one of two treatment arms: intervention (prednisolone) or matched placebo.
An independant statistician will generate a randomisation schedule using variable block sizes, stratified by site.
This schedule will be used by the central phyarmacist at RCH to undertake the blinding, labelling and distribution of the study drugs to participating sites by putting together study packs of the required drug labelled with sequential study numbers for each site.
Randomisation at the site will be undertaken at the site by the doctor or research team by selecting the lowest numbered study pack (next pack system) from the study drug store in the ED

Masking / blinding

Blinded (masking used)

Who is / are masked / blinded?

The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data

Intervention assignment

Parallel

Other design features

Phase

Phase 3

Type of endpoint/s

Efficacy

Statistical methods / analysis

Sample size estimation:
The primary outcome is the proportion of subjects who have complete recovery at 1 month post randomisation. Based on our observational data, we expect 60% of children without prednisolone to have complete recovery at 1 month. A study in adults found an improvement of 12% with prednisolone compared with placebo which was deemed to represent a clinically important difference.
To enable us to identify an increase in recovery from 60% to 72% or larger with 80% power requires a study with 244 subjects in each treatment group based on a two-sided test with a=0.05. Hence we aim to recruit 270 per group (540 in total) to allow for 10% loss to follow-up at 1 month.
Statistical Analysis:
Data will be analysed on an intention-to-treat basis where primary outcome data are available.
Primary outcome will be presented as a difference in proportions in each treatment group, with a comparison between the groups presented as a difference in proportions and as an odds ratio from a logistic regression model adjusted for site, with a 95% CI and p-value.
Secondary outcomes will be summarised by treatment group. Binary outcomes will be presented as proportions, with comparisons betwen the groups presented as a difference in proportions and as odds ratios from logistic regression adjusted for site, with 95% CIs and p-values.

Recruitment

Recruitment status

Recruiting

Date of first participant enrolment

Anticipated

1/07/2015

Actual

13/10/2015

Date of last participant enrolment

Anticipated

Actual

Date of last data collection

Anticipated

Actual

Sample size

Target

540

Accrual to date

175

Final

Recruitment in Australia

Recruitment state(s)

NSW,QLD,SA,WA,VIC

Recruitment hospital [1]

The Royal Childrens Hospital - Parkville

Recruitment hospital [2]

Monash Medical Centre - Clayton campus - Clayton

Recruitment hospital [3]

The Children's Hospital at Westmead - Westmead

Recruitment hospital [4]

Womens and Childrens Hospital - North Adelaide

Recruitment hospital [5]

Gold Coast Hospital - Southport

Recruitment hospital [6]

Logan Hospital - Meadowbrook

Recruitment hospital [7]

Queensland Children's Hospital - South Brisbane

Recruitment hospital [8]

Perth Children's Hospital - Nedlands

Recruitment postcode(s) [1]

3052 - Parkville

Recruitment postcode(s) [2]

3168 - Clayton

Recruitment postcode(s) [3]

2145 - Westmead

Recruitment postcode(s) [4]

5006 - North Adelaide

Recruitment postcode(s) [5]

4215 - Southport

Recruitment postcode(s) [6]

4131 - Meadowbrook

Recruitment postcode(s) [7]

6009 - Nedlands

Recruitment outside Australia

Country [1]

New Zealand

State/province [1]

Auckland

Funding & Sponsors

Funding source category [1]

Government body

Name [1]

National Health and Medical Research Council

Address [1]

16 Marcus Clarke Street
Canberra
ACT 2601

Country [1]

Australia

Funding source category [2]

Charities/Societies/Foundations

Name [2]

Emergency Medicine Foundation

Address [2]

2/15 Lang Parade Milton QLD 4064

Country [2]

Australia

Primary sponsor type

Individual

Name

Franz Babl

Address

Murdoch Children's Research Institute
Flemington Road
Parkville
VIC 3052

Country

Australia

Secondary sponsor category [1]

None

Name [1]

Address [1]

Country [1]

Ethics approval

Ethics application status

Approved

Ethics committee name [1]

The Royal Children's Hospital, Melbourne- Human Research Ethics Committee

Ethics committee address [1]

Flemington Road
Parkville VIC 3052

Ethics committee country [1]

Australia

Date submitted for ethics approval [1]

Approval date [1]

30/03/2015

Ethics approval number [1]

35035A

Summary

Brief summary

Children diagnosed with Bell's palsy have a sudden weakness of facial muscles on one side of their face. Bell's palsy usually improves in children in about four to six weeks without treatment. However, in some children it may be a year before the facial weakness has completely resoved and in a small number of cases, the facial muscle weakness is permanent. 
Recent research in adults with Bell's palsy has shown that a short course of treamtent with steroids (prednisolone) led to improved recovery time. Prednisolone is believed to reduce the irritation to the facial nerve. There are no similar studies in children and, because children's bodies respond different to adults', it is not known whether a short course of prednisolone will help children to recover more quickly. Currently, some doctors treat with prednisolone and others do not resulting in confusion and variability in medical care. 

The study aims to answer the following question: In children presenting to Emergency Departments with recent onset of Bell's palsy, does treatment with prednisolone result in a higher proportion of children with recovery at 1 month compared with placebo. 

Up to 13 hospitals in Australia and New Zealand will participate in enrolling 540 children/young people, aged 6 months to 18 years, who present to ED's within 72 hours of symptom onset.

Trial website

Trial related presentations / publications

1. Babl FE, Mackay MT,Borland ML, Herd DW, Kochar A, Hort J, Rao A, Cheek JA, Furyk J, Barrow L, George S, Zhang ,, Gardiner K, Lee KJ, Davidson A, Berkowitz R, Sullivan F, Porrello E, Dalziel KM, Anderson V, Oakley E, Hopper S, Williams F, Wilson C, Williams A, Dalziel SR; PREDICT (Paediatric Research In Emergency Departments International Collaborative) research network. 
Bell's Palsy in Children (BellPIC): protocol for a multicentre, placebo-controlled randomized trial. BMC Pediatr. 2017 Feb 13;17(1):53. doi: 10.1186/s12887-016-0702-y.

2. Babl FE, Gardiner KK, Kochar A, Wilson CL, George SA, Zhang M, Furyk J, Thosar D, Cheek JA, Krieser D, Rao AS, Borland ML, Cheng N, Phillips NT, Sinn KK, Neutze JM, Dalziel SR; PREDICT (Paediatric Research In Emergency Departments International Collaborative).
Bell's palsy in children: Current treatment patterns in Australia and New Zealand. A PREDICT study. J Paediatr Child Health. 2017 Feb 8. doi: 10.1111/jpc.13463.

Public notes

Contacts

Principal investigator

Name

Prof Franz Babl

Address

Emergency Research Department
Murdoch Childrens Research Institute
Royal Children's Hospital
Flemington Road
Parkville VIC 3052

Country

Australia

Phone

+61399366748

Fax

franz.babl@rch.org.au

Contact person for public queries

Name

Franz Babl

Address

Emergency Research Department
Murdoch Childrens Research Institute
Royal Children's Hospital
Flemington Road
Parkville VIC 3052

Country

Australia

Phone

+61399366748

Fax

franz.babl@rch.org.au

Contact person for scientific queries

Name

Franz Babl

Address

Emergency Research Department
Murdoch Childrens Research Institute
Royal Children's Hospital
Flemington Road
Parkville VIC 3052

Country

Australia

Phone

+61399366748

Fax

franz.babl@rch.org.au

Data sharing statement

Will individual participant data (IPD) for this trial be available (including data dictionaries)?

No/undecided IPD sharing reason/comment

IPD will not be available, data will be summarised by treatment group and primary outcome presented as a proportion in each treatment groups.

What supporting documents are/will be available?

Doc. No.	Type	Citation	Link	Email	Other Details	Attachment
3572	Study protocol	Babl FE, Mackay MT, Borland ML, Herd DW, Kochar A, Hort J, Rao A, Cheek JA, Furyk J, Barrow L, George S, Zhang M, Gardiner K, Lee KJ, Davidson A, Berkowitz R, Sullivan F, Porrello E, Dalziel KM, Anderson V, Oakley E, Hopper S, Williams F, Wilson C, Williams A, Dalziel SR; PREDICT (Paediatric Research In Emergency Departments International Collaborative) research network. Bell’s Palsy in Children (BellPIC): Protocol for a Multicentre, Placebo-Controlled Randomized Trial. BMC Pediatrics 2017 Feb 13;17(1):53. doi: 10.1186/s12887-016-0702-y.

Results publications and other study-related documents

Documents added manually

No documents have been uploaded by study researchers.

Documents added automatically

Source	Title	Year of Publication	DOI
Embase	Corticosteroids for Bell's palsy (idiopathic facial paralysis).	2016	https://dx.doi.org/10.1002/14651858.CD001942.pub5
Embase	Efficacy of Prednisolone for Bell Palsy in Children: A Randomized, Double-Blind, Placebo-Controlled, Multicenter Trial.	2022	https://dx.doi.org/10.1212/WNL.0000000000201164
Embase	Cost-effectiveness of Prednisolone to Treat Bell Palsy in Children: An Economic Evaluation Alongside a Randomized Controlled Trial.	2023	https://dx.doi.org/10.1212/WNL.0000000000207284

N.B. These documents automatically identified may not have been verified by the study sponsor.

Trial Review

Registering a new trial?

Documents added manually

Documents added automatically