Condition category
Respiratory
Date applied
08/05/2018
Date assigned
12/06/2018
Last edited
12/06/2018
Prospective/Retrospective
Retrospectively registered
Overall trial status
Ongoing
Recruitment status
Recruiting

Plain English Summary

Background and study aims
Recurrent Respiratory Papillomatosis (RRP) causes wart-like growths in the airway which make it difficult to breathe, speak and carry out normal everyday activities. It is a rare condition but is more common and aggressive in children than in adults, affecting 4 in every 100,000 children. There is no known cure for RRP, but symptoms are checked through regular hospital visits, with multiple treatments or procedures under general anaesthetic needed to remove or shrink the growths which can grow back quickly. The problem is that nobody knows which treatments or procedures work best. The aim of this study is to collect information on airway procedures currently being used in UK NHS hospitals to determine which of the treatments are the safest and most effective.

Who can participate?
All patients (adults and children) with RRP receiving at least one treatment in a UK NHS hospital

What does the study involve?
Data is collected on all treatments for RRP in a secure, online database over a 28-month period from 01/04/2018 to 31/08/2020. By determining the most effective treatments for RRP, the aim is to increase the time interval between surgical treatments needed to control symptoms, and reduce the overall number of treatments, the severity and spread of papillomas in the airway, hospital visits, and medications, and ultimately improve quality of life in those with RRP. The safety of the different treatments are assessed to identify those which minimise the spread of disease. From the data collected, patient subgroups are identified who respond better to specific treatments, along with risk factors which contribute to complications (such as tracheostomy).

What are the possible benefits and risks of participating?
The main benefit of participation is the opportunity to address the current lack of evidence around the relative safety and efficacy of the various treatments used to manage RRP, which will improve outcomes for all RRP patients. As this is an observational study, there are no risks of participation, as the clinical treatment a patient receives will be exactly the same regardless of whether they consent to their data being entered to the registry or not.

Where is the study run from?
1. Alder Hey Children’s NHS Foundation Trust (UK)
2. Basildon & Thurrock University Hospitals NHS Foundation Trust (UK)
3. Blackpool Teaching Hospitals NHS Foundation Trust (UK)
4. Great Ormond Street Hospital for Children NHS Foundation Trust (UK)
5. North Cumbria University Hospitals NHS Trust (UK)
6. Nottingham University Hospitals NHS Trust (UK)
7. The Newcastle upon Tyne Hospitals NHS Foundation Trust (UK)
8. NHS Greater Glasgow and Clyde (UK)

When is the study starting and how long is it expected to run for?
September 2017 to August 2020

Who is funding the study?
National Institute for Health Research (NIHR) (UK)

Who is the main contact?
Mrs Kim Fairbairn
kim.keltie@nuth.nhs.uk

Trial website

www.rrp.org.uk

Contact information

Type

Scientific

Primary contact

Mrs Kim Fairbairn

ORCID ID

http://orcid.org/0000-0001-5108-6279

Contact details

Freeman Hospital
Newcastle upon Tyne
NE7 7DN
United Kingdom
+44 (0)191 213 8636
kim.keltie@nuth.nhs.uk

Additional identifiers

EudraCT number

ClinicalTrials.gov number

Protocol/serial number

38013

Study information

Scientific title

Airway Intervention Registry (AIR) extension: Recurrent Respiratory Papillomatosis

Acronym

AIR: RRP

Study hypothesis

Recurrent Respiratory Papillomatosis (RRP) causes wart like growths in the airway which make it difficult to breathe, speak and carry out normal everyday activities. It is a rare condition but is more common and aggressive in children than in adults, affecting 4 in every 100,000 children. There is no known cure for RRP, but symptoms are checked through regular hospital visits, with multiple therapies or procedures under general anaesthetic needed to remove or shrink the growths which can grow back quickly. The problem is that nobody knows which therapies or procedures work best.

The Airway Intervention Registry (AIR) platform was designed to capture information on airway procedures. It was set up originally to capture information from any patient aged 18 years or less undergoing routine treatment for airway stenosis using balloon dilatation, or treatment for recurrent respiratory papillomatosis (RRP) with radiofrequency cold ablation, in order to answer the research questions highlighted by NICE as absent from the current evidence base on each procedure (see guidance 1.3 in IPG425 and IPG434). Registry data will help to address this gap.

AIR has been collecting data on balloon dilatation for airway stenosis since 01/04/2015 and we are due to report back to NICE by 30/06/2018 to inform the IPG425 guidance update. As part of a national survey (Donne et al., 2016) we gathered clinical advice that radiofrequency cold ablation is not commonly used to treat RRP, and what was needed was safety and efficacy data gathered for all treatments for this condition. As RRP is such a rare disease we have extended data collection to include both adults and paediatrics. We secured funding from the NIHR’s RfPB programme to support the development of the RRP registry in paediatrics, which will be hosted on the same AIR platform (both ENT procedures, same clinical staff entering data but different patient population). Funding was received on 1st September 2017 for a 3 year fixed period.

Ethics approval

Newcastle and North Tyneside 1, 29/12/2014, IRAS ID: 164160, REC ref: 14/NE/1200

Study design

Observational; Design type: Cohort study

Primary study design

Observational

Secondary study design

Cohort study

Trial setting

Hospitals

Trial type

Treatment

Patient information sheet

See additional files

Condition

Specialty: Children, Primary sub-specialty: Respiratory and Cystic Fibrosis; UKCRC code/ Disease: Respiratory/ Other diseases of upper respiratory tract

Intervention

The trialists are collecting observational data on all interventions for RRP in a secure, online database over a 28-month period from 01/04/2018 to 31/08/2020. The objective is to improve the care of patients with RRP by analysing the registry data to determine which of the RRP interventions currently being used in UK NHS hospitals are the safest and most effective (information which is currently lacking).

By determining the most effective interventions for RRP, the trialists will be able to increase the time interval between surgical interventions to maintain symptomatic control, reduce overall number of RRP interventions, severity and spread of papillomas in the airway, hospital visits, medications and ultimately improve quality of life in those suffering from RRP. By also capturing peri- and post-procedural details they will be able to determine the relative safety of the different interventions available and identify those which minimise the spread of disease. From the data collected they will also be able to identify patient subgroups (based on patient characteristics such as age, gender, human papillomavirus (HPV) type, location of papillomas, RRP severity and spread, comorbidities) who respond better to specific interventions, and also identify patient risk factors which contribute to the complication outcomes (such as tracheostomy).

Intervention type

Other

Phase

Drug names

Primary outcome measure

1. Effectiveness of the interventions, assessed using the Derkay staging system to quantify RRP severity based on involvement of laryngeal structures. As this is observational data, the study will not require the patients to attend additional hospital appointments. They will be assessed at their routine hospital visits which will be scheduled according to clinical need over the course of the 28-month data collection period.
2. Quality of life, measured using the Voice Handicap Index (VHI) questionnaire (for patients aged 16 or over) or the paediatric Voice Handicap Index (pVHI) for patients under 16. Patients currently complete these questionnaires as part of their standard care at hospital appointments. Patients who have consented to taking part in the study will also be given a unique QoL ID so that they can submit additional QoL forms via a secure patient website at their convenience.

Secondary outcome measures

1. Peri-procedural complications; Timepoint(s): At hospital discharge
2. Airway related A&E visits; Timepoint(s): After hospital discharge
3. Airway related GP visits; Timepoint(s): After hospital discharge
4. Change in Derkay score (disease severity and spread); Timepoint(s): Over 28-month data collection period
5. Change in voice assessment scores; Timepoint(s): Over 28-month data collection period
6. Inter-surgical interval (time between interventions to achieve symptomatic control); Timepoint(s): Over 28-month data collection period
7. Nature and timing of surgical intervention; Timepoint(s): Over 28-month data collection period
8. Nature and timing of adjuvant therapy; Timepoint(s): Over 28-month data collection period
9. Histology results (including HPV type); Timepoint(s): Over 28-month data collection period
10. No. of hospital visits/admissions (inpatient, outpatient); Timepoint(s): Over 28-month data collection period
11. Post-operative discharge location (planned and actual); Timepoint(s): After procedure (following RRP intervention)

Overall trial start date

01/09/2017

Overall trial end date

31/08/2020

Reason abandoned (if study stopped)

Eligibility

Participant inclusion criteria

All patients (adults and paediatrics) with a diagnosis of RRP receiving at least one intervention for RRP in a UK NHS hospital

Participant type

Patient

Age group

Adult

Gender

Both

Target number of participants

Planned Sample Size: 400; UK Sample Size: 400

Participant exclusion criteria

Patient/parent/guardian unable/unwilling to provide written informed assent/consent

Recruitment start date

01/04/2018

Recruitment end date

31/08/2020

Locations

Countries of recruitment

United Kingdom

Trial participating centre

Alder Hey Children’s NHS Foundation Trust
L12 2AP

Trial participating centre

Basildon & Thurrock University Hospitals NHS Foundation Trust
SS16 5NL

Trial participating centre

Blackpool Teaching Hospitals NHS Foundation Trust
FY3 8NR

Trial participating centre

Great Ormond Street Hospital for Children NHS Foundation Trust
WC1N 3JH

Trial participating centre

North Cumbria University Hospitals NHS Trust
CA2 7HY

Trial participating centre

Nottingham University Hospitals NHS Trust
NG5 1PB

Trial participating centre

The Newcastle upon Tyne Hospitals NHS Foundation Trust
NE7 7DN

Trial participating centre

NHS Greater Glasgow and Clyde
G12 0XH

Sponsor information

Organisation

The Newcastle upon Tyne Hospitals NHS Foundation Trust

Sponsor details

Freeman Hospital
Freeman Road
High Heaton
Newcastle-upon-Tyne
NE7 7DN
United Kingdom

Sponsor type

Hospital/treatment centre

Website

Funders

Funder type

Government

Funder name

NIHR Central Commissioning Facility (CCF); Grant Codes: PB-PG-0416-20037

Alternative name(s)

Funding Body Type

Funding Body Subtype

Location

Results and Publications

Publication and dissemination plan

Results will be available to clinicians via open-access peer-reviewed manuscripts (e.g. Clinical Otolaryngology) and presentations/posters at appropriate clinical conferences (with attendance at three conferences specifically included in the funding application: ENT UK 2018 annual conference to promote RRP database, ENT UK 2019 annual conference to share initial results and data completeness/quality issues, EPSO 2020 international conference to share results of 3 year study). This will enable results to reach clinicians in acute care, and national bodies (e.g. NICE) so that they can be used to update national guidance and inform commissioners. The trialists will also liaise with professional bodies (e.g. ENT UK, British Association for Paediatric Otolaryngology (BAPO)) to display the published report on their website and share with their members via emailed newsletters (both ENT UK and BAPO were engaged with the UK cross-sectional survey and contributed to publicising results using the methods already described). The trialists have included costs for three focus groups with their local YPAGne group during the study duration (one each year of the study). The local PPI group will guide writing and dissemination of lay summaries of our findings, which will be shared on patient forums, charity websites, social media and by any other appropriate means suggested. The trialists have also acted on feedback provided by their local YPAGne group to share recruitment rates and interim results on a dedicated study website to ensure that results reach patients and are presented in a format which is appropriate to young patients.

IPD sharing statement
Any external researchers requesting data from the registry will be required to submit a formal application form describing which datafields are required, the time period of interest, if updates are required (i.e. monthly/annually), what the information will be used for and how the results of any analysis will be shared with the public. Only formal applications with appropriate ethical approvals in place (reviewed by an independent Ethics Committee) will be reviewed by the established AIR Steering Group committee. If successful, a data sharing agreement between applying researcher and Steering Group committee will be signed. If approval is given, only anonymised data will be shared for the purposes of external research studies.

Intention to publish date

31/08/2021

Participant level data

Available on request

Basic results (scientific)

Publication list

Publication citations

Editorial Notes

12/06/2018: Uploaded protocol Version 1.5 17 April 2018 (not peer-reviewed).