ANZCTR - Registration

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Trial registered on ANZCTR

Registration number

ACTRN12619000927123

Ethics application status

Approved

Date submitted

17/06/2019

Date registered

3/07/2019

Date last updated

3/07/2019

Date data sharing statement initially provided

3/07/2019

Type of registration

Prospectively registered

Titles & IDs

Public title

Cricopharyngeal peroral endoscopic myotomy for cricopharyngeal achalasia: a pilot study on feasibility, efficacy, and safety

Scientific title

Cricopharyngeal peroral endoscopic myotomy for cricopharyngeal achalasia: a pilot study on feasibility, efficacy, and safety

Secondary ID [1]

none

Universal Trial Number (UTN)

U1111-1235-5211

Trial acronym

C-POEM Study

Linked study record

Health condition

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

Pharyngeal dysphagia

Upper oesophageal sphincter dysfunction

Condition category

Condition code

Oral and Gastrointestinal

Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon

Surgery

Surgical techniques

Neurological

Other neurological disorders

Intervention/exposure

Study type

Interventional

Description of intervention(s) / exposure

Dysphagic patients with manometric evidence of inadequate upper oesophageal sphincter relaxation will undergo cricopharyngeal per-oral endoscopic myotomy (C-POEM). C-POEM is a minimally invasive endoscopic technique designed to perform oesophageal sphincterotomy; its safety and efficacy are well-established in the treatment of achalasia cardia. C-POEM is performed under general anaesthesia, and the anticipated average duration of the procedure will be 60 minutes. Clinical efficacy is objectively determined by 1) patient-reported outcome - dysphagia questionnaire; 2) intra-procedural upper oesophageal sphincter distensibility measurement; and 3) upper oesophageal sphincter relaxation pressure by high-resolution manometry.

C-POEM will be performed by experienced therapeutic gastrointestinal endoscopists (Principal Investigator Dr Peter Wu; co-investigator Prof Philip Chiu). All standard of care monitoring for endoscopic procedures will be applied, including nursing and anaesthetic documentation. Endoscopy reports will also be generated as part of medical record.

Intervention code [1]

Treatment: Surgery

Comparator / control treatment

No control group

Control group

Uncontrolled

Outcomes

Primary outcome [1]

Dysphagia symptoms measured by Swallow Quality-of-Life questionnaire (Swal-QOL)

Timepoint [1]

1-month post-endoscopic myotomy

Secondary outcome [1]

Upper oesophageal sphincter distensibility measured by the endoluminal functional lumen imaging probe

Timepoint [1]

distensibility is measure intra-procedurally immediately before and after per-oral endoscopic myotomy

Secondary outcome [2]

Patients will be observed in the hospital for a minimum of 24 hours after the procedure for major procedure-related complications, including (but not exclusive of) 1) Lumen perforation; 2) Would infection; 3) Bleeding requiring transfusion and/or intervention. Following discharge, weekly phone calls and 1-month clinic follow-up will be conducted by investigators to monitor for delayed complications such as bleeding and symptoms of infection.

Timepoint [2]

Up to 30-day from the procedure

Eligibility

Key inclusion criteria

1) Known underlying neurological disorders that would result in upper oesophageal sphincter dysfunction, and
2) Self-reported pharyngeal dysphagia symptoms, and
3) Manometric evidence of upper oesophageal sphincter dysfunction

Minimum age

18 Years

Maximum age

No limit

Sex

Both males and females

Can healthy volunteers participate?

Key exclusion criteria

1. Oesophageal disorders known to cause dysphagia (e.g. achalasia cardia, peptic oesopahgeal stricture)
2. Known hypopharyngeal structural disorders (e.g. radiotherapy-induced stricture)
3. Unwilling or unable to provide informed consent

Study design

Purpose of the study

Treatment

Allocation to intervention

Non-randomised trial

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

not applicable

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

not applicable

Masking / blinding

Open (masking not used)

Who is / are masked / blinded?

Intervention assignment

Single group

Other design features

Phase

Not Applicable

Type of endpoint/s

Efficacy

Statistical methods / analysis

This is an open-label proof of principle study; there is no data to support a sample size calculation and the investigators believe n=30 will be sufficient to calculate variance estimates to derive a sample size for an adequately powered subsequent trial.
Hypothesis 1: C-POEM increase (improves) dysphagia severity score (SWAL-QOL)
Therapeutic response is defined as an increase (improvement) in SWAL-QOL score by at least 12 points after the C-POEM; this will be expressed as an percentage of response rate. To test the changes in patient-reported symptoms (SWAL-QOL score) with C-POEM, paired t-test will be used compare the two groups: 1) SWAL-POEM score at time of recruitment; 2) SWAL-POEM score at 3 months after the C-POEM. An equivalent non-parametric analysis will be performed if parametric assumptions are not met. Kaplan-Meier analysis of the follow-up (1-year) SWAL-QOL will be used to determine the rate and time to relapse of dysphagia.

Hypothesis 2: C-POEM increases (improves) CP distensibility.
Objectively measured CP distensibility is expressed as an index calculated by narrowest cross-sectional area (mm2) at maximal distension divided by corresponding intra-bag pressure (mm2/Hg). To test the changes of CP distensibility with C-POEM, paired t-test will be used compare: 1) CP distensibility index immediately before C-POEM; 2) CP distensibility index immediately after C-POEM. An equivalent non-parametric analysis will be performed if parametric assumptions are not met.

Hypothesis 3: C-POEM reduces (improves) CP deglutitive nadir pressure.
To test the changes in the CP deglutitive nadir pressure (mmHg) with C-POEM, paired t-test will be used compare the two groups: 1) CP pressure at time of recruitment; 2) CP pressure at 1 months after the C-POEM. An equivalent non-parametric analysis will be performed if parametric assumptions are not met.

Recruitment

Recruitment status

Not yet recruiting

Date of first participant enrolment

Anticipated

8/07/2019

Actual

Date of last participant enrolment

Anticipated

10/07/2020

Actual

Date of last data collection

Anticipated

14/08/2020

Actual

Sample size

Target

Accrual to date

Final

Recruitment outside Australia

Country [1]

Hong Kong

State/province [1]

HKSAR

Funding & Sponsors

Funding source category [1]

Self funded/Unfunded

Name [1]

not applicable - unfunded

Address [1]

not application - unfunded

Country [1]

Primary sponsor type

University

Name

The Chinese University of Hong Kong

Address

4M, S. H. Ho Centre for Digestive Health, Day Treatment Block and Children Wards, Prince of Wales Hospital, 30-32 Ngan Shing Street, Shatin, New Territories.

Country

Hong Kong

Secondary sponsor category [1]

None

Name [1]

Address [1]

Country [1]

Ethics approval

Ethics application status

Approved

Ethics committee name [1]

The Joint Chinese University of Hong Kong – New Territories East Cluster Clinical Research Ethics Committee

Ethics committee address [1]

8/F, Lui Che Woo Clinical Sciences Building,
Prince of Wales Hospital
Shatin, Hong Kong

Ethics committee country [1]

Hong Kong

Date submitted for ethics approval [1]

15/03/2019

Approval date [1]

09/04/2019

Ethics approval number [1]

IRB 2019.183

Summary

Brief summary

Cricopharyngeal (CP) achalasia is a motility disorder where the upper oesophageal sphincter does not relax completely to allow passage of swallowed food and water from the pharynx into the oesophagus. It is associated with several neurological disorders including Parkinson's Disease, Lateral Medullary Stroke, and brain stem tumour. Patients with this condition suffer from troublesome swallowing, often resulting in both reduced quality of life and increase risk for malnourishment and chest infection. 
Conventional treatment (cricopharyngeal myotomy) is a morbid open surgery that carries significant risk, thus often not offered to patients. Per-oral endoscopic myotomy (POEM) is a minimally invasive endoscopic technique initially designed to treat lower oesophageal sphincter dysfunction (achalasia cardia). This technique has potentially to be adapted in the treatment of CP achalasia while mimimising the risk associated with conventional open surgery.. In this pilot study, we aim to gather preiliminary clinical efficacy and safety of cricopharyngeal POEM in the treatment of swallowing difficulties related to upper oesophageal sphincter dysfunction.

Trial website

Trial related presentations / publications

Public notes

Contacts

Principal investigator

Name

Dr Peter Wu

Address

4M, S. H. Ho Centre for Digestive Health, Day Treatment Block and Children Wards, Prince of Wales Hospital, 30-32 Ngan Shing Street, Shatin, New Territories.

Country

Hong Kong

Phone

+852 5239 2518

Fax

p.wu@unsw.edu.au

Contact person for public queries

Name

Thomas Lam

Address

4M, S. H. Ho Centre for Digestive Health, Day Treatment Block and Children Wards, Prince of Wales Hospital, 30-32 Ngan Shing Street, Shatin, New Territories.

Country

Hong Kong

Phone

+852 2637 0355

Fax

thomaslam@cuhk.edu.hk

Contact person for scientific queries

Name

Thomas Lam

Address

4M, S. H. Ho Centre for Digestive Health, Day Treatment Block and Children Wards, Prince of Wales Hospital, 30-32 Ngan Shing Street, Shatin, New Territories.

Country

Hong Kong

Phone

+85226370355

Fax

thomaslam@cuhk.edu.hk

Data sharing statement

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

No/undecided IPD sharing reason/comment

What supporting documents are/will be available?

No Supporting Document Provided

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	Cricopharyngeal peroral endoscopic myotomy improves oropharyngeal dysphagia in patients with Parkinson's disease.	2021	https://dx.doi.org/10.1055/a-1562-7107

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

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Documents added manually

Documents added automatically