AchalasiaNCT03450928
POEM: Long vs Short Myotomy for Achalasia. RCT
A study testing Peroral Endoscopic Myotomy (POEM) in people with Achalasia, run by Catholic University of the Sacred Heart at 1 site in Italy.
Trial summary
Per-Oral Endoscopic Myotomy (POEM) is increasingly used for the treatment of achalasia. In published series, a 12cm-POEM is usually performed. Surgical myotomy is typically shorter (8cm). The clinical efficacy of both procedure is comparable. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Achalasia
- What is being tested?
- Peroral Endoscopic Myotomy (POEM)
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 200 participants
- Where
- 1 siteItaly
- Study dates
- 6 Jun 2014to 1 Jan 2022
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of achalasia (type I and II)
- Age > 18
- Signed informed consent
- Patient accepts to undergo scheduled follow-up after POEM
- Eckardt score > 3
- ASA I-II-III
Who cannot join
- Prior surgical treatment of achalasia (previous endoscopic balloon dilation or botox injection ARE NOT exclusion criteria)
- Pregnancy or nursing
- Current alcohol or drug addiction.
- Mentally retarded or emotionally unstable, or exhibits psychological characteristics which, in the opinion of the investigator make the subject a poor candidate for this study.
- Severe congenital or acquired coagulopathy or INR > 1.6
- Participating in another ongoing clinical trial in which concomitant diagnosis or therapeutic intervention would adversely affect the integrity of the clinical trial.
Show all 9Show fewer
- Hepatic cirrhosis w/ or w/o portal hypertension w/ or w/o esophageal varices
- Eosinophilic esophagitis or Barrett Esophagus
- Esophageal Strictures
Trial locations and contacts
1 site in 1 country.
Italy1 site, 1 recruiting
- Recruiting
Fondazione Policlinico Universitario Agostino Gemelli
Roma, RM, 00168
Carolina Gualtieri, 00390630156580, carolina.gualtieri@policlinicogemelli.it
About this study
Official title: Peroral Endoscopic Myotomy (POEM) for the Treatment of Esophageal Achalasia: Long vs Short Myotomy. Prospective Randomized Controlled Study
Per-Oral Endoscopic Myotomy (POEM) is increasingly used for the treatment of achalasia. In published series, a 12cm-POEM is usually performed. Surgical myotomy is typically shorter (8cm). The clinical efficacy of both procedure is comparable.
This study intends to investigate if clinical outcomes of POEM depends on the length of esophageal myotomy, in patients with classic-type achalasia (type I and type II according to Chicago Classification)
Read the detailed description
Peroral Endoscopic Myotomy myotomy (POEM) has been recently introduced for treatment of achalasia, based on technical developments from NOTES (natural orifice translumenal surgery). The technique includes the incision of the mucosa in the esophageal body, the submucosal dissection of the distal esophagus and the creation of a submucosal tunnel in the distal esophagus and proximal gastric body, and the esophageal myotomy. The procedure is performed transorally, using a flexible endoscope.
In published series, a 12cm-POEM is usually performed. Surgical myotomy is typically shorter, being protracted for about 8cm. The clinical efficacy of POEM and surgical myotomy is more likely to be similar according to the most recent published series.
In this randomized controlled trial (non-inferiority trial) we evaluate the outcomes of POEM according to the length of the esophageal myotomy.
Patients with type I and II achalasia will be randomly assigned to one of the two groups, long--myotomy (LM) and short--myotomy (SM).
Patients in the LM-group will receive a 12cm-long POEM (including 3cm on the stomach); in the SM-group patients will undergo a POEM extended for 7 cm (including 3cm on the stomach).
During follow-up, High Resolution Manometry (HRM), Esophageal pH-monitoring study and Esophagogastroduodenoscopy (EGD) will be regularly performed and symptoms assessed with the use of the Eckardt score (ECKs).
The main hypothesis is that the results of a SM are not inferior to the results of a LM. Calculated sample size is 200 patients
- Conditions
- Achalasia
- Being tested
- Peroral Endoscopic Myotomy (POEM)
- Keywords
- Peroral Endoscopic Myotomy, Endoscopy, POEM, Submucosal endoscopy
- Medical subject headings
- Esophageal Achalasia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Peroral Endoscopic Myotomy (POEM)Procedure or surgery
Peroral endoscopic myotomy (POEM) is a minimally invasive intervention for the treatment of esophageal achalasia.
POEM includes different steps:
- Lifting of the esophageal mucosa, with the injection of saline solution, and mucosal incision on the esophageal body (approximately 12 or 7 cm above the esophagogastric junction, for about 1-2cm)
- Advance of the endoscope into the submucosa, repeated submucosal injection and dissection of a submucosal tunnel into the distal esophagus up to 3 cm into the gastric wall.
- Myotomy of the distal esophagus, cardia and gastric wall, starting 3 cm below the mucosal incision
- After the completion of myotomy, and check for mucosal integrity, the mucosal incision is closed using endoscopic clips
| Study group | Role | What they receive |
|---|---|---|
| Short POEM | Active comparator | Patients in the Short POEM-group will undergo a Peroral Endoscopic Myotomy (POEM) extended for a total of 7 cm (including 4 cm above the esophago-gastric junction and 3cm on the stomach). Receives: Procedure: Peroral Endoscopic Myotomy (POEM) |
| Long POEM | Active comparator | Patients in the Long POEM-group will receive a 12cm-long Peroral Endoscopic Myotomy (POEM), including 9 cm on the esophagus and 3cm on the gastric wall Receives: Procedure: Peroral Endoscopic Myotomy (POEM) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Non-inferiority of a Short-POEM compared to a Long-POEM
Treatment success, defined as an Eckard score ≤ 3.
The Eckardt score is a clinical score for evaluating the severity of achalasia-related symptoms. Eckardt score varies between 0 and 12.
Questions are asked about the frequency of dysphagia, regurgitation and retrosternal pressure sensation. Depending on whether any of these symptom occurrs, never, occasionally, daily or with each meal, a symptom score between 0 and 3 is applied. In addition a symptom score of 0-3 is assigned to the degree of weight loss (0 - No weight loss; 1 - weight loss < 5Kg, 2 - weight loss between 5 and 10Kg, 3- weight loss > 10Kg).
Each symptom score (dysphagia, regurgitation, retrosternal pressure, and wieight loss) is summed to the other. Thus, a completely asymptomatic patient has a score of 0 and the most severely affected patient had a maximum score of 12.
Measured: 2 years
Secondary outcomes (7)
Basal Lower Esophageal Sphincter (LES) pressure
Basal LES pressure on esophageal manometry after Short-POEM compared to Long-POEM
Measured: Before POEM, 6 months and 2 years after the procedure
4-second Integrated Relaxation Pressure (4sIRP)
4sIRP on esophageal manometry after Short-POEM compared to Long-POEM
Measured: Before POEM, 6 months and 2 years after the procedure
Side effects and complications
Incidence of side effect and complications after Short-POEM compared to Long-POEM
Measured: Baseline to 2 years
Gastro-Esophageal Reflux (GER) symptom assessment
GerdQ questionnaire will be used to evaluate and compare Gastoesophageal reflux symptoms in the two groups.
GerdQ is a 6-item, validated, easy to use questionnaire. The GerdQ questionnaire asks patients to score the number of days with symptoms and use of over-the-counter (OTC) medications during the previous 7 days. It uses a four graded Likert scale (0-3) to score the frequency of four positive predictors of GERD (heartburn, regurgitation, sleep disturbance due to reflux symptoms or use of over-the-counter (OTC) medications for reflux symptoms) and a reversed Likert scale (3-0) for two negative predictors of GERD (epigastric pain and nausea) giving a total GerdQ score range of 0-18. The sleep disturbance and use of OTC medication are also used for assessment of the impact of GERD, giving a separate 'impact score' ranging from 0 to 6
Measured: baseline, 6 months and 2 years after POEM
Esophageal pH-monitoring study
patients will undergo esophageal pH-monitoring study during the follow-up. DeMeester score will be used for the diagnosis of GERD.
DeMeester score is a composite score based on 6 parameters (scored in comparison to mean values in normal subjects for each category below):
Supine reflux, Upright reflux, Total reflux, Number of episodes, Number of episodes longer than 5 min, Longest episode. A score of >14.72 shows significant reflux.
Prevalence of GERD will be compared in the two study groups
Measured: 6 months after POEM
Operating time
The duration of the endoscopic procedures for each patients will be calculated, in minutes, since the mucosal incision until the endoscopic closure of the mucosal entry with the last endoscopic clip.
The average duration of the procedure in the Short-POEM group will be compared wth the dureation of the procedures in the Long-POEM-groups
Measured: During the endoscopic procedure
Quality of life
Improvement of quality of life after Short-POEM compared to Long-POEM, assessed by using the SF-36 questionnaires' scores
The Short Form (36) Health Survey is a 36-item, patient-reported survey of patient health.
The SF-36 consists of eight scaled scores, which are the weighted sums of the questions in their section. Each scale is directly transformed into a 0-100 scale on the assumption that each question carries equal weight. The lower the score the more disability. The higher the score the less disability i.e., a score of zero is equivalent to maximum disability and a score of 100 is equivalent to no disability. The eight sections are: vitality, physical functioning, bodily pain, general health perceptions, physical role functioning, emotional role functioning, social role functioning, mental health
Measured: baseline, 6 months and 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Mar 2018
- Study start6 Jun 2014
- Primary completion1 Jan 2020 (estimated)
- Study completion1 Jan 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for AchalasiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 200 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Catholic University of the Sacred Heart
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Guido Costamagna, Catholic University of the Sacred Heart
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03450928
- Sponsor's protocol ID
- POEMLvS
- Status last verified
- Feb 2018
- First submitted
- 7 Nov 2017
- Last updated
- 1 Mar 2018
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Familiari P, Borrelli de Andreis F, Landi R, Mangiola F, Boskoski I, Tringali A, Perri V, Costamagna G. Long versus short peroral endoscopic myotomy for the treatment of achalasia: results of a non-inferiority randomised controlled trial. Gut. 2023 Aug;72(8):1442-1450. doi: 10.1136/gutjnl-2021-325579. Epub 2023 Apr 18.
PubMed 37072180
Study at a glance
From the registry record, last updated by the sponsor on 1 Mar 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Peroral Endoscopic Myotomy (POEM) for the Treatment of Esophageal Achalasia: Long vs Short Myotomy. Prospective Randomized Controlled Study
- ClinicalTrials.gov ID
- NCT03450928
- Acronym / sponsor ID
- POEMLvS
- Conditions
- Achalasia
- Keywords
- Peroral Endoscopic Myotomy, Endoscopy, POEM, Submucosal endoscopy
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 200 participants (estimated)
- Lead sponsor
- Catholic University of the Sacred HeartUniversity, hospital or other
- Study start
- 6 Jun 2014 (actual)
- Primary completion
- 1 Jan 2020 (estimated)
- Study completion
- 1 Jan 2022 (estimated)
- First posted
- 1 Mar 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Per-Oral Endoscopic Myotomy (POEM) is increasingly used for the treatment of achalasia. In published series, a 12cm-POEM is usually performed. Surgical myotomy is typically shorter (8cm). The clinical efficacy of both procedure is comparable.
This study intends to investigate if clinical outcomes of POEM depends on the length of esophageal myotomy, in patients with classic-type achalasia (type I and type II according to Chicago Classification)
Detailed description
Peroral Endoscopic Myotomy myotomy (POEM) has been recently introduced for treatment of achalasia, based on technical developments from NOTES (natural orifice translumenal surgery). The technique includes the incision of the mucosa in the esophageal body, the submucosal dissection of the distal esophagus and the creation of a submucosal tunnel in the distal esophagus and proximal gastric body, and the esophageal myotomy. The procedure is performed transorally, using a flexible endoscope.
In published series, a 12cm-POEM is usually performed. Surgical myotomy is typically shorter, being protracted for about 8cm. The clinical efficacy of POEM and surgical myotomy is more likely to be similar according to the most recent published series.
In this randomized controlled trial (non-inferiority trial) we evaluate the outcomes of POEM according to the length of the esophageal myotomy.
Patients with type I and II achalasia will be randomly assigned to one of the two groups, long--myotomy (LM) and short--myotomy (SM).
Patients in the LM-group will receive a 12cm-long POEM (including 3cm on the stomach); in the SM-group patients will undergo a POEM extended for 7 cm (including 3cm on the stomach).
During follow-up, High Resolution Manometry (HRM), Esophageal pH-monitoring study and Esophagogastroduodenoscopy (EGD) will be regularly performed and symptoms assessed with the use of the Eckardt score (ECKs).
The main hypothesis is that the results of a SM are not inferior to the results of a LM. Calculated sample size is 200 patients
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Short POEM | Active comparator | Procedure: Peroral Endoscopic Myotomy (POEM)Patients in the Short POEM-group will undergo a Peroral Endoscopic Myotomy (POEM) extended for a total of 7 cm (including 4 cm above the esophago-gastric junction and 3cm on the stomach). |
| Long POEM | Active comparator | Procedure: Peroral Endoscopic Myotomy (POEM)Patients in the Long POEM-group will receive a 12cm-long Peroral Endoscopic Myotomy (POEM), including 9 cm on the esophagus and 3cm on the gastric wall |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Diagnosis of achalasia (type I and II)
- Age > 18
- Signed informed consent
- Patient accepts to undergo scheduled follow-up after POEM
- Eckardt score > 3
Key exclusion criteria (9 listed)
- Prior surgical treatment of achalasia (previous endoscopic balloon dilation or botox injection ARE NOT exclusion criteria)
- Pregnancy or nursing
- Current alcohol or drug addiction.
- Mentally retarded or emotionally unstable, or exhibits psychological characteristics which, in the opinion of the investigator make the subject a poor candidate for this study.
- Severe congenital or acquired coagulopathy or INR > 1.6
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Peroral Endoscopic Myotomy (POEM)Peroral endoscopic myotomy (POEM) is a minimally invasive intervention for the treatment of esophageal achalasia. POEM includes different steps: 1. Lifting of the esophageal mucosa, with the injection of saline solution, and mucosal incision on the esophageal body (approximately 12 or 7 cm above the esophagogastric junction, for about 1-2cm) 2. Advance of the endoscope into the submucosa, repeated submucosal injection and dissection of a submucosal tunnel into the distal esophagus up to 3 cm into the gastric wall. 3. Myotomy of the distal esophagus, cardia and gastric wall, starting 3 cm below the mucosal incision 4. After the completion of myotomy, and check for mucosal integrity, the mucosal incision is closed using endoscopic clips | Procedure or surgery | Long POEM, Short POEM |
Primary outcome
Non-inferiority of a Short-POEM compared to a Long-POEM
Time frame: 2 years
Treatment success, defined as an Eckard score ≤ 3. The Eckardt score is a clinical score for evaluating the severity of achalasia-related symptoms. Eckardt score varies between 0 and 12. Questions are asked about the frequency of dysphagia, regurgitation and retrosternal pressure sensation. Depending on whether any of these symptom occurrs, never, occasionally, daily or with each meal, a symptom score between 0 and 3 is applied. In addition a symptom score of 0-3 is assigned to the degree of weight loss (0 - No weight loss; 1 - weight loss < 5Kg, 2 - weight loss between 5 and 10Kg, 3- weight loss > 10Kg). Each symptom score (dysphagia, regurgitation, retrosternal pressure, and wieight loss) is summed to the other. Thus, a completely asymptomatic patient has a score of 0 and the most severely affected patient had a maximum score of 12.
7 secondary outcomes
Basal Lower Esophageal Sphincter (LES) pressure
Time frame: Before POEM, 6 months and 2 years after the procedure
4-second Integrated Relaxation Pressure (4sIRP)
Time frame: Before POEM, 6 months and 2 years after the procedure
Side effects and complications
Time frame: Baseline to 2 years
Gastro-Esophageal Reflux (GER) symptom assessment
Time frame: baseline, 6 months and 2 years after POEM
Esophageal pH-monitoring study
Time frame: 6 months after POEM
Operating time
Time frame: During the endoscopic procedure
Quality of life
Time frame: baseline, 6 months and 2 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Mar 2018.
- Derived from this study: PubMed 37072180Familiari P, Borrelli de Andreis F, Landi R, Mangiola F, Boskoski I, Tringali A, Perri V, Costamagna G. Long versus short peroral endoscopic myotomy for the treatment of achalasia: results of a non-inferiority randomised controlled trial. Gut. 2023 Aug;72(8):1442-1450. doi: 10.1136/gutjnl-2021-325579. Epub 2023 Apr 18.
- Individual participant dataSharing planned: No
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Achalasia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Peroral Endoscopic Myotomy (POEM) in people with Achalasia, run by Catholic University of the Sacred Heart at 1 site in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 1 Mar 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Pietro Familiari, MD (00390630156580, pietrofamiliari@tiscali.it), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Non-inferiority of a Short-POEM compared to a Long-POEM (time frame: 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03450928, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Mar 2018.