Condition category
Digestive System
Date applied
01/09/2020
Date assigned
14/09/2020
Last edited
14/09/2020
Prospective/Retrospective
Prospectively registered
Overall trial status
Ongoing
Recruitment status
Not yet recruiting

Plain English Summary

Background and study aims
Oesophageal Achalasia (OA) means that a person has difficulty swallowing food or liquids due to the muscles in their food-pipe not working properly. Patients may have chest pain, trouble swallowing food or liquids, weight loss, and may complain about food coming back into their mouth after eating. The incidence of OA is relatively low (1-2.5 new cases per 100.000 people per year), but because it is a chronic disease, it is estimated that total cases are around 10 patients per 100.000 people. OA is a chronic disease and currently, all treatment options are for the control of symptoms only and patients may require multiple interventions during their lifetime. There are currently three treatment options for OA: surgical or endoscopic division of the muscle between the oesophagus and the stomach, forceful pneumatic dilatation, and paralysis of the muscle by injecting botulinum toxin. The outcomes of these treatments are measured by symptom improvement; however, currently, there are no validated patient-reported outcome tools to measure achalasia symptoms.

With the help of seventeen international experts and cognitive interviews with patients, a new Patient Reported Outcome Tool has been created to quantify the symptoms in achalasia patients and to assess the efficacy of achalasia treatments. This is necessary for a period when different treatment options need to be compared in randomized controlled trials

Before embarking in a large study, a pilot study has been planned with the main aim of establishing the willingness of patients to fill in questionnaires related to their symptoms. This will allow the design of a larger study to test the capacity of the tool for detecting the success or failure of treatments. It is expected that the acceptance rate in the pilot study will be over 80%. If this target is achieved, a larger study to validate the I-PASS questionnaire will be created.

Who can participate?
Adult patients after their diagnosis has been confirmed by the usual tests for achalasia (High-resolution manometry, barium swallow, and UGI endoscopy). Patients younger than 18 years, not fluent in English, or with psychiatric or neurological disorders will be excluded. This study has been discussed and approved by the Association of Achalasia Patients in the UK.

What does the study involve?
Participants will be invited via an invitation letter, the study information leaflet, and consent form being posted to them. They will have a pre-paid envelope to return the documents. They will be provided with a contact number of the research team if they have any queries regarding the study. Participants will be given at least 1 month to decide whether on not they would like to participate in the study.

The patient will receive the I-PASS questionnaire and the short form 36 of the quality of life questionnaire once informed consent has been taken. The questionnaires will be posted to the participant with a prepaid envelope. Patients will fill in the two questionnaires individually (without any involvement of the nurse) however, the research team contact details will be available if the patient requires any further explanations. On completion of the questionnaires, the time needed to fill in the forms will be recorded by the participant. It is expected that the time needed to complete the questionnaires will be around 10 to 20 minutes.

The last questionnaire will then be filled to establish if the patient was satisfied with the questionnaires, if they understood all the questions, and whether they would be available to review the questionnaires again, after treatment.

What are the possible benefits and risks of participating?
There will be no direct or indirect benefit for the patients from their participation in the study, and there are not risks foreseen as a result of participation.

Where is the study run from?
Three NHS centers in London, Imperial College, University College, and Barts Health (UK)

When is the study starting and how long is it expected to run for?
From November 2019 to December 2022

Who is funding the study?
Fondazione Morgagni ONLUS (Italy)

Who is the main contact?
Dr. Sheraz Markar
s.markar@imperial.ac.uk

Trial website

Contact information

Type

Scientific

Primary contact

Dr Sheraz Markar

ORCID ID

http://orcid.org/0000-0001-8650-2017

Contact details

Imperial College London
10th floor QEQM Building
St Mary’s Hospital
South Warf Road
London
W2 1NY
United Kingdom
+44 (0)20 3312 2125
s.markar@imperial.ac.uk

Additional identifiers

EudraCT number

Nil known

ClinicalTrials.gov number

Nil known

Protocol/serial number

IRAS 26741

Study information

Scientific title

International patients-reported-outcome for Achalasia Symptom Score

Acronym

I-PASS

Study hypothesis

Oesophageal achalasia is a relatively rare disease characterized by the absence of opening of the cardia and the absence of peristaltic contractions along the oesophageal body. Consequently, when an achalasia patient swallows solid food or liquid, the bolus cannot pass into the stomach and he/she experiences dysphagia, regurgitation of undigested food into the mouth, or chest pain. It has been estimated that the incidence of OA is between 1 to 2.5 per 100.000 habitants per year, but the prevalence of the disease is ten times higher (10/100.000).
Most patients will require more than one treatment during their lifetime. The medical community treats the symptoms caused by OA (no etiological therapy exists) by lowering the resting pressure of the cardia. This is achieved by cutting the muscle, either during surgery or endoscopy or by forceful stretching of the muscle or by “poisoning” the muscle by injecting botulinum toxin into it. The efficacy of any of these treatment options is measured by symptom improvement, however, there are no validated patient-reported outcome questionnaires for achalasia symptoms. At present, the so-called “Eckardt score” is used, but this tool is not based on patient self-reporting, and therefore may be influenced by the interviewer and more importantly, it has never been properly validated. When we developed the International Guidelines for Oesophageal Achalasia, the lack of a validated tool that helps to define the success/failure of treatment was highlighted. A validated patient report outcome tool is also essential to compare the different treatment methods in randomized clinical trials and may facilitate the medical community accurately selecting the most appropriate therapy option.
We have created an international group of “achalasia experts” comprising of seventeen gastroenterologists and surgeons from the UK, USA, Canada, Australia, Sweden, Norway, the Netherlands, Belgium, Germany, and Italy with the aim of creating this new tool (International Patient-reported outcome for Achalasia Symptom Scoring – I-PASS). Through a 3-round Delphi process, we have prepared a questionnaire, translated it into lay words, and refined it with cognitive interviews with 10 achalasia patients. Our hypothesis is that this new tool will allow to objectively measure the outcome of the different type of treatments of achalasia.

Ethics approval

Pending

Study design

Observational pilot study

Primary study design

Observational

Secondary study design

Cross sectional study

Trial setting

Hospitals

Trial type

Diagnostic

Patient information sheet

Condition

Oesophageal achalasia

Intervention

Achalasia patients will be recruited in high-volume Upper Gastrointestinal (UGI) surgical and gastroenterological units. The recruitment of the patients will occur once the patients have completed their investigations (high-resolution manometry testing and barium swallow) and have a confirmed diagnosis of achalasia. UGI endoscopy will exclude any malignancies in the esophagus and the stomach.

Participants will be asked if they would like to participate in this research study via an invitation letter, the study information leaflet, and consent form being posted to them. They will have a pre-paid envelope to return the documents. They will be provided with a contact number of the research team if they have any queries regarding the study. Participants will be given at least 1 month to decide whether on not they would like to participate in the study. Once informed consent has been taken, questionnaires will be posted to the participant with a prepaid envelope.

The patient will then receive the I-PASS questionnaire and the short form 36 of the quality of life questionnaire. The I-PASS questionnaire has been developed through a 3-round Delphi process and then translated into lay words and refined through cognitive interviews with 10 achalasia patients. Patients will fill in the two questionnaires individually (without any involvement of the nurse), however, the research team contact details will be available if the patient requires any further explanations. On completion of the questionnaires, the time needed to fill in the forms will be recorded by the participant. The last questionnaire will then be filled to establish if the patient was satisfied with the questionnaires, if they understood all the questions, and whether they would be available to review the questionnaires again, after treatment.

It is expected that the time needed to complete the questionnaires will be around 10 to 20 mins.

Intervention type

Other

Phase

Drug names

Primary outcome measure

The willingness of naïve achalasia patients to fill in questionnaires related to their symptoms measured by the proportion of the number of eligible patients invited to participate who complete the questionnaires

Secondary outcome measures

1. The time required for the patient to fill in the questionnaires recorded by patients on completion of the questionnaires
2. Patient satisfaction with the questionnaires measured using a follow-up questionnaire

Overall trial start date

01/11/2019

Overall trial end date

01/12/2022

Reason abandoned (if study stopped)

Eligibility

Participant inclusion criteria

1. Aged ≥18 years
2. Primary achalasia confirmed by High-Resolution manometry, Barium swallow and endoscopy
3. Not previously treated for achalasia

Participant type

Patient

Age group

Adult

Gender

Both

Target number of participants

50

Participant exclusion criteria

1. Not fluent in English
2. Diagnosis of neurological or cognitive impairment, and/or severe psychiatric disorder

Recruitment start date

15/11/2020

Recruitment end date

15/11/2022

Locations

Countries of recruitment

United Kingdom

Trial participating centre

Imperial College NHS Trust
St Mary's Hospital South Warf Road
London
W2 1BL
United Kingdom

Trial participating centre

University College London Hospital NHS Foundation trust
250 Euston Road
London
NW1 2PG
United Kingdom

Trial participating centre

Barts Health NHS Trust
The Royal London Hospital Whitechapel
London
E1 1BB
United Kingdom

Sponsor information

Organisation

Imperial College Healthcare NHS Trust

Sponsor details

Medical School Building
Norfolk Place
London
W1 PG
United Kingdom
+44 (0)2075941862
r.nicholson@imperial.ac.uk

Sponsor type

University/education

Website

http://www.imperial.nhs.uk/

Funders

Funder type

Charity

Funder name

Fondazione Morgagni ONLUS - Fondazione Morgagni Charity

Alternative name(s)

Funding Body Type

Funding Body Subtype

Location

Results and Publications

Publication and dissemination plan

Planned publication of results in a high impact-factor journal

IPD sharing statement:
The datasets generated and/or analysed during the current study will be included in the subsequent results publication. Data collected will be anonymized and stored in password-protected Imperial College PC.

Intention to publish date

01/06/2023

Participant level data

Other

Basic results (scientific)

Publication list

Publication citations

Additional files

Editorial Notes

04/09/2020: Trial’s existence confirmed by the Morgagni Foundation.