Esophageal AchalasiaNCT01768091
POEM vs. Pneumatic Dilation for Esophageal Achalasia
A study testing POEM and Pneumatic dilation in people with Esophageal Achalasia, run by Nanfang Hospital, Southern Medical University at 1 site in China.
Trial summary
The purpose of this study is to determine the efficacy and safety of peroral endoscopic myotomy (POEM) compared with pneumatic dilation in the treatment of esophageal achalasia. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Esophageal Achalasia
- What is being tested?
- POEM and Pneumatic dilation
- 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 siteChina
- Study dates
- Dec 2011to Dec 2013
- 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 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Between 18 and 75 years of age
- Patient with esophageal achalasia
- Eckardt score > 3
- Signed informed consent
Who cannot join
- Severe cardio-pulmonary disease or other serious disease leading to unacceptable surgical risk
- Pseudo-achalasia, Mega-oesophagus (greater than 7 cm), or Oesophageal diverticula in the distal oesophagus
- Previous endoscopic Botox injection
- Previous oesophageal or gastric surgery
- Pregnancy or lactation women, or ready to pregnant women
- Not capable of filling out questionnaires
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Nanfang Hospital of Southern Medical University
Guangzhou, Guangdong, 510515
Xiaobing Cui, M.D., +86 13631312723, xbing119@gmail.com
About this study
Official title: Peroral Endoscopic Myotomy Versus Pneumatic Dilation for Esophageal Achalasia: a Prospective Randomized Controlled Trial
The purpose of this study is to determine the efficacy and safety of peroral endoscopic myotomy (POEM) compared with pneumatic dilation in the treatment of esophageal achalasia.
Read the detailed description
Esophageal achalasia is an esophageal motor disorder, which is characterized by the absence of esophageal peristalsis combined with a defective relaxation of the lower esophageal sphincter (LES). The major symptoms of esophageal achalasia are dysphagia, chest pain, and regurgitation of undigested food.
Currently, treatment options mainly focus on relief of the symptoms by reducing the LES pressure. Pneumatic dilation is the main endoscopic therapies for esophageal achalasia. However, the patients need repeat treatment to maintain therapeutic success and there is a risk of perforation (1%-3%). For surgery approaches, the laparoscopic Heller's myotomy (LHM) combined with Dor's antireflux procedure has gained considerable interest. The LHM can sustain therapeutic effects for long-term in approximately 80% of patients.
Recently, Inoue et al. succeeded in treating achalasia endoscopically with a method called peroral endoscopic myotomy (POEM) and achieved promising results in short-term. Technically, POEM derived from natural orifice transluminal endoscopic surgery (NOTES) and endoscopic submucosal dissection (ESD), in which a submucosal tunnel is created after submucosal injection, and then an endoscopic myotomy was made at the gastroesophageal junction.
However, the long-term efficacy and safety of POEM were not determined, and there was no prospective study that compared the POEM with other conventional treatment. Therefore, we aim to determine the efficacy and safety of POEM, compared with the pneumatic dilation, in the treatment of esophageal achalasia.
- Conditions
- Esophageal Achalasia
- Being tested
- POEM, Pneumatic dilation
- Keywords
- Esophageal achalasia, Peroral endoscopic myotomy, POEM, Pneumatic dilation
- Medical subject headings
- Esophageal Achalasia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
POEMProcedure or surgery
Also called Peroral endoscopic myotomy
- Entry to submucosal space. After submucosal injection, a 2-cm longitudinal mucosal incision is made at approximately 13 cm proximal to the gastroesophageal junction (GEJ).
- Submucosal tunnelling. A long submucosal tunnel is created to 3 cm distal to the GEJ.
- Endoscopic myotomy is begun at 3 cm distal to the mucosal entry point, and is carried out in a proximal to distal direction to a total length of 10 cm.
- Long endoscopic myotomy of inner circular muscle bundles is done, leaving the outer longitudinal muscle layer intact. The expected end point of myotomy is 2 cm distal to the GEJ.
- Closure of mucosal entry: the mucosal incision is closed using hemostatic clips. (Inoue H et al. POEM for esophageal achalasia… Endoscopy 2010; 42: 265-271)
Pneumatic dilationProcedure or surgery
A Rigiflex balloon (30 mm) was positioned at the esophagogastric junction and dilated at a pressure of 5 PSI for 1 minute, followed by 8 PSI for 1 minute.
(N Engl J Med 2011;364:1807-16.)
| Study group | Role | What they receive |
|---|---|---|
| POEM | Experimental | POEM for patients with esophageal achalasia Receives: Procedure: POEM |
| Pneumatic dilation | Active comparator | Pneumatic dilation for patients with esophageal achalasia Receives: Procedure: Pneumatic dilation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Therapeutic success
Therapeutic success is defined as a symptom control to an Eckardt score of 3 or less.
The Eckardt score is the sum of the symptom scores for dysphagia, regurgitation, and chest pain (with a score of 0 indicating the absence of symptoms, 1 indicating occasional symptoms, 2 indicating daily symptoms, and 3 indicating symptoms at each meal) and weight loss (with 0 indicating no weight loss, 1 indicating a loss of <5 kg, 2 indicating a loss of 5 to 10 kg, and 3 indicating a loss of >10 kg) (Eckardt, V. Gastroenterology, 1992. 103(6): p. 1732-8.)
Measured: From date of randomization until the follow-up ended, assessed up to 2 years
Secondary outcomes (4)
Procedure related complication
Perforation, Delayed bleeding, Pneumothorax, Subcutaneous emphysema, Anastomotic leak etc.
Measured: From date of randomization until the follow-up ended, assessed up to 2 years
Time of treatment failure
Time of treatment failure is defined as when the Eckardt score of patients are more than 3
Measured: From date of randomization until the follow-up ended, assessed up to 2 years
Pressure at the lower esophageal sphincter
The basal LES pressure and the swallow-induced LES relaxation will be monitored and measured at the end of expiration. After introduction and equilibration, basal pressure is monitored during at least 5 minutes. Swallow-induced relaxation of the sphincter is assessed by 5 ml wet swallows, at least 30 s apart.
Measured: From date of randomization until the follow-up ended, assessed up to 2 years
Quality of life
Patients will complete the quality-of-life questionnaires (the Medical Outcomes Study 36-Item Short-Form Health Survey, SF-36) for assessing quality of life
Measured: From date of randomization until the follow-up ended, assessed up to 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jan 2013 (estimated)
- Study startDec 2011
- Primary completionDec 2013 (estimated)
- Study completionDec 2013 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Esophageal AchalasiaStudies of the same condition, open and under way.
- More trials run by Nanfang Hospital, Southern Medical UniversityThe sponsor's other registered studies.
- 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
- Nanfang Hospital, Southern Medical University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Wei Gong, M.D., Principal investigator, Department of Gastroenterology, Nanfang Hospital of Southern Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01768091
- Sponsor's protocol ID
- NFEC-201211-K2
- Other IDs
- 201120
- Status last verified
- Jan 2013
- First submitted
- 5 Jan 2013
- Last updated
- 15 Jan 2013
- Results posted
- No
- Data monitoring committee
- No
Publications
Inoue H, Minami H, Kobayashi Y, Sato Y, Kaga M, Suzuki M, Satodate H, Odaka N, Itoh H, Kudo S. Peroral endoscopic myotomy (POEM) for esophageal achalasia. Endoscopy. 2010 Apr;42(4):265-71. doi: 10.1055/s-0029-1244080. Epub 2010 Mar 30.
PubMed 20354937Boeckxstaens GE, Annese V, des Varannes SB, Chaussade S, Costantini M, Cuttitta A, Elizalde JI, Fumagalli U, Gaudric M, Rohof WO, Smout AJ, Tack J, Zwinderman AH, Zaninotto G, Busch OR; European Achalasia Trial Investigators. Pneumatic dilation versus laparoscopic Heller's myotomy for idiopathic achalasia. N Engl J Med. 2011 May 12;364(19):1807-16. doi: 10.1056/NEJMoa1010502.
PubMed 21561346
Study at a glance
From the registry record, last updated by the sponsor on 15 Jan 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Peroral Endoscopic Myotomy Versus Pneumatic Dilation for Esophageal Achalasia: a Prospective Randomized Controlled Trial
- ClinicalTrials.gov ID
- NCT01768091
- Acronym / sponsor ID
- NFEC-201211-K2
- Conditions
- Esophageal Achalasia
- Keywords
- Esophageal achalasia, Peroral endoscopic myotomy, POEM, Pneumatic dilation
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 200 participants (estimated)
- Lead sponsor
- Nanfang Hospital, Southern Medical UniversityUniversity, hospital or other
- Study start
- Dec 2011
- Primary completion
- Dec 2013 (estimated)
- Study completion
- Dec 2013 (estimated)
- First posted
- 15 Jan 2013 (estimated)
- 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.
The purpose of this study is to determine the efficacy and safety of peroral endoscopic myotomy (POEM) compared with pneumatic dilation in the treatment of esophageal achalasia.
Detailed description
Esophageal achalasia is an esophageal motor disorder, which is characterized by the absence of esophageal peristalsis combined with a defective relaxation of the lower esophageal sphincter (LES). The major symptoms of esophageal achalasia are dysphagia, chest pain, and regurgitation of undigested food.
Currently, treatment options mainly focus on relief of the symptoms by reducing the LES pressure. Pneumatic dilation is the main endoscopic therapies for esophageal achalasia. However, the patients need repeat treatment to maintain therapeutic success and there is a risk of perforation (1%-3%). For surgery approaches, the laparoscopic Heller's myotomy (LHM) combined with Dor's antireflux procedure has gained considerable interest. The LHM can sustain therapeutic effects for long-term in approximately 80% of patients.
Recently, Inoue et al. succeeded in treating achalasia endoscopically with a method called peroral endoscopic myotomy (POEM) and achieved promising results in short-term. Technically, POEM derived from natural orifice transluminal endoscopic surgery (NOTES) and endoscopic submucosal dissection (ESD), in which a submucosal tunnel is created after submucosal injection, and then an endoscopic myotomy was made at the gastroesophageal junction.
However, the long-term efficacy and safety of POEM were not determined, and there was no prospective study that compared the POEM with other conventional treatment. Therefore, we aim to determine the efficacy and safety of POEM, compared with the pneumatic dilation, in the treatment of esophageal achalasia.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| POEM | Experimental | Procedure: POEMPOEM for patients with esophageal achalasia |
| Pneumatic dilation | Active comparator | Procedure: Pneumatic dilationPneumatic dilation for patients with esophageal achalasia |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Between 18 and 75 years of age
- Patient with esophageal achalasia
- Eckardt score > 3
- Signed informed consent
Key exclusion criteria (6 listed)
- Severe cardio-pulmonary disease or other serious disease leading to unacceptable surgical risk
- Pseudo-achalasia, Mega-oesophagus (greater than 7 cm), or Oesophageal diverticula in the distal oesophagus
- Previous endoscopic Botox injection
- Previous oesophageal or gastric surgery
- Pregnancy or lactation women, or ready to pregnant women
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 |
|---|---|---|
| POEMAlso: Peroral endoscopic myotomy1. Entry to submucosal space. After submucosal injection, a 2-cm longitudinal mucosal incision is made at approximately 13 cm proximal to the gastroesophageal junction (GEJ). 2. Submucosal tunnelling. A long submucosal tunnel is created to 3 cm distal to the GEJ. 3. Endoscopic myotomy is begun at 3 cm distal to the mucosal entry point, and is carried out in a proximal to distal direction to a total length of 10 cm. 4. Long endoscopic myotomy of inner circular muscle bundles is done, leaving the outer longitudinal muscle layer intact. The expected end point of myotomy is 2 cm distal to the GEJ. 5. Closure of mucosal entry: the mucosal incision is closed using hemostatic clips. (Inoue H et al. POEM for esophageal achalasia… Endoscopy 2010; 42: 265-271) | Procedure or surgery | POEM |
| Pneumatic dilationA Rigiflex balloon (30 mm) was positioned at the esophagogastric junction and dilated at a pressure of 5 PSI for 1 minute, followed by 8 PSI for 1 minute. (N Engl J Med 2011;364:1807-16.) | Procedure or surgery | Pneumatic dilation |
Primary outcome
Therapeutic success
Time frame: From date of randomization until the follow-up ended, assessed up to 2 years
Therapeutic success is defined as a symptom control to an Eckardt score of 3 or less. The Eckardt score is the sum of the symptom scores for dysphagia, regurgitation, and chest pain (with a score of 0 indicating the absence of symptoms, 1 indicating occasional symptoms, 2 indicating daily symptoms, and 3 indicating symptoms at each meal) and weight loss (with 0 indicating no weight loss, 1 indicating a loss of <5 kg, 2 indicating a loss of 5 to 10 kg, and 3 indicating a loss of >10 kg) (Eckardt, V. Gastroenterology, 1992. 103(6): p. 1732-8.)
4 secondary outcomes
Procedure related complication
Time frame: From date of randomization until the follow-up ended, assessed up to 2 years
Time of treatment failure
Time frame: From date of randomization until the follow-up ended, assessed up to 2 years
Pressure at the lower esophageal sphincter
Time frame: From date of randomization until the follow-up ended, assessed up to 2 years
Quality of life
Time frame: From date of randomization until the follow-up ended, assessed up to 2 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Jan 2013.
- Background: PubMed 20354937Inoue H, Minami H, Kobayashi Y, Sato Y, Kaga M, Suzuki M, Satodate H, Odaka N, Itoh H, Kudo S. Peroral endoscopic myotomy (POEM) for esophageal achalasia. Endoscopy. 2010 Apr;42(4):265-71. doi: 10.1055/s-0029-1244080. Epub 2010 Mar 30.
- Background: PubMed 21561346Boeckxstaens GE, Annese V, des Varannes SB, Chaussade S, Costantini M, Cuttitta A, Elizalde JI, Fumagalli U, Gaudric M, Rohof WO, Smout AJ, Tack J, Zwinderman AH, Zaninotto G, Busch OR; European Achalasia Trial Investigators. Pneumatic dilation versus laparoscopic Heller's myotomy for idiopathic achalasia. N Engl J Med. 2011 May 12;364(19):1807-16. doi: 10.1056/NEJMoa1010502.
- Homepage of Nanfang Hospital of Southern Medical UniversityLink provided by the sponsor.
- Homepage of Department of Gastroenterology, Nanfang Hospital of Southern Medical UniversityLink provided by the sponsor.
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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.
Wei Gong, M.D. principal investigator
Department of Gastroenterology, Nanfang Hospital of Southern Medical University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing POEM and Pneumatic dilation in people with Esophageal Achalasia, run by Nanfang Hospital, Southern Medical University at 1 site in China.
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 4 inclusion and 6 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 15 Jan 2013. 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 China. 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 Wei Gong, M.D. (+86 15820290385, gwei203@yahoo.com.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Therapeutic success (time frame: From date of randomization until the follow-up ended, assessed up to 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 NCT01768091, which also lists 2 linked publications.
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 15 Jan 2013.