POEM: Endoscopic Peroral Myotomy for Treatment of Achalasia

• Sponsor: Universitätsklinikum Hamburg-Eppendorf (Other)
• Overall Status: Completed
• CT.gov ID: NCT01405469
• Collaborator: (none)
• Enrollment: 16
• Location: 1
• Arm: 1
• Duration (Months): 72
• Patients Per Site Per Month: 0.2

Study Details

Study Description

Brief Summary

This study intends to investigate the feasibility, safety and efficacy of peroral endoscopic myotomy for the treatment of achalasia in a single center setting in Europe.

Condition or Disease	Intervention/Treatment	Phase
Achalasia	Other: Peroral Endoscopic Myotomy	N/A

Detailed Description

This pilot study intends to investigate the feasibility, safety and efficacy of peroral endoscopic myotomy for the treatment of achalasia in a single center setting in Europe.

16 patients are enrolled to evaluate feasibility, safety and efficacy of peroral endoscopic myotomy. Main outcome measurement is the eckhardt symptom score 3 month after therapy.

Primary outcome:

-eckhardt symptom score 3 month after therapy.

Secondary outcomes:

• Lower esophageal sphincter pressure at 3 month after therapy.

• Reflux symptoms at 3 month after therapy.

For this prospective study, inclusion criteria are achalasia, as diagnosed by established methods (contrast fluoroscopy, manometry, esophagal-gastro-duodenoscopy) and age greater than 18 years. Previous therapy, such as esophageal surgery or previous myotomy are exclusion criterion.

A forward-viewing upper endoscope is used with a transparent distal cap attachment. Carbon dioxide gas is necessary for insufflation during the procedures. An endoscopic knife is used to access the submucosa, dissect the submucosal tunnel and also to divide circular muscle bundles over a length of approximately 10cm, extending 2-3cm onto the cardia. An electric generator is used with spray coagulation mode. A coagulating forceps is used for hemostasis as needed. Closure of the mucosal entry site is performed using standard endoscopic clips.

Study Design

• Study Type: Interventional
• Actual Enrollment: 16 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Endoscopic Peroral Myotomy for Treatment of Achalasia: Pilot Study
• Study Start Date: Nov 1, 2010
• Actual Primary Completion Date: Jun 1, 2011
• Actual Study Completion Date: Nov 1, 2016

Arms and Interventions

Arm

Intervention/Treatment

Experimental: Peroral Endoscopic Myotomy Patients with achalasia who are designed to either get balloon dilatation or have botulinum toxin injection, or to have surgical intervention (Heller myotomy)for treatment

Other: Peroral Endoscopic Myotomy; A forward-viewing upper endoscope is used with a transparent distal cap attachment. Carbon dioxide gas is necessary for insufflation during the procedures. An endoscopic knife is used to access the submucosa, dissect the submucosal tunnel and also to divide circular muscle bundles over a length of approximately 10cm, extending 2-3cm onto the cardia. An electric generator is used with spray coagulation mode. A coagulating forceps is used for hemostasis as needed. Closure of the mucosal entry site is performed using standard endoscopic clips

Outcome Measures

Primary Outcome Measures

1. Treatment Success Defined as Symptom Relief 3 Months After Treatment Based on an Eckhardt Score ≤ 3 [3 months after treatment]

   eckhardt score is a score to evaluate achalasia discomfort in patients. Patients are being interrogated for dysphagia, regurgitation, and retrosternal pain , correlated with the time frame of occurrence. "with every meal" giving 3 points," daily" (2 points), "sometimes" (1 Point) or "no" (0 Points), as well as weight loss,(>10 kg= 3 points, 5-10 kg=2 points, 0-5 kg=1 point, None=0 points. Scale range is from 0 Points (no achalasia) up to 12 points for the worst achalasia symptoms.

Secondary Outcome Measures

1. mmHg of the Lower Esophageal Sphincter 3 Months After POEM Procedure [manometry at 3 month after therapy]

   esophageal manometry is done 3 months after POEM procedure to evaluate resting lower esophageal sphincter pressure

2. Number of Participants With Reflux Symptoms [during procedure, and 3 and 6 months, and 1, 2 and 5 years after treatment]

   Number of Participants with Reflux Symptoms during procedure, and 3 and 6 months, and 1, 2 and 5 years after treatment

3. Number of Participants With Procedure-related Adverse Events [procedure to 3 months post procedure]

   procedure-related adverse events per protocol

4. Medication 3 Months After POEM [3 months]

   proton pump inhibitor (PPI) use at 3 months after POEM procedure

5. Duration Time Procedure [procedure]

   duration time of POEM procedures in minutes

6. Days Duration Hospitalization [days of hospitalization for POEM procedure, an average of 4 days]

   participants were followed for the duration of hospital stay, an average of 4 days

7. cm Myotomy Length [POEM procedure]

   myotomy length in cm

8. Percentage of Participants Who Achieved Treatment Success 3 Months After Treatment [3 months after treatment]

   eckhardt score is a score to evaluate achalasia discomfort in patients. Patients are being interrogated for dysphagia, regurgitation, and retrosternal pain , correlated with the time frame of occurrence. "with every meal" giving 3 points," daily" (2 points), "sometimes" (1 Point) or "no" (0 Points), as well as weight loss,(>10 kg= 3 points, 5-10 kg=2 points, 0-5 kg=1 point, None=0 points. Scale range is from 0 Points (no achalasia) up to 12 points for the worst achalasia symptoms. Post-myotomy eckhardt score ≤ 3 n individuals has been reached in 15 individuals.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion criteria:

• Patient with symptomatic achalasia and pre-op barium swallow, manometry and esophagal-gastro-duodenoscopy which are consistent with the diagnosis

• persons of age > 18 years with medical indication for surgical myotomy or Endoscopic balloon dilatation

• Signed written informed consent.

Exclusion criteria:

• Patients with previous surgery of the stomach or esophagus

• Patients with known coagulopathy

• Previous achalasia-treatment with surgery

• Patients with liver cirrhosis and/or esophageal varices

• Active esophagitis

• Eosinophilic esophagitis

• Barrett's esophagus

• Pregnancy

• Stricture of the esophagus

• Malignant or premalignant esophageal lesion

• Candida esophagitis

• Hiatal hernia > 2cm

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Universitätsklinikum Hamburg-Eppendorf, Klinik für Interdisziplinäre Endoskopie	Hamburg		Germany	20246

Sponsors and Collaborators

• Universitätsklinikum Hamburg-Eppendorf

Investigators

• Principal Investigator: Thomas Roesch, Prof. Dr., Universitätsklinikum Hamburg-Eppendorf

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

• University Hospital Hamburg-Eppendorf, Endoscopy Department

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.

• Responsible Party: Universitätsklinikum Hamburg-Eppendorf
• ClinicalTrials.gov Identifier: NCT01405469
• Other Study ID Numbers: UKE Hamburg Endoscopy PV3725
• First Posted: Jul 29, 2011
• Last Update Posted: Oct 15, 2019
• Last Verified: Oct 1, 2019

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Keywords provided by Universitätsklinikum Hamburg-Eppendorf: Achalasia; Heller myotomy; Dysphagia; Peroral Endoscopic Myotomy
• Additional relevant MeSH terms: Esophageal Achalasia

Study Results

Participant Flow

Recruitment Details	Patients with symptomatic achalasia
Pre-assignment Detail	16 patients were included as per protocol, no patient was lost during follow-up over a 2 year period

Arm/Group Title	POEM Patients
Arm/Group Description	pilot group of achalasia patients who received POEM Treatment: Endoscopic Myotomy of the Lower Esophageal Sphincter
Period Title: Overall Study
STARTED	16
COMPLETED	16
NOT COMPLETED	0

Baseline Characteristics

Arm/Group Title	POEM Patients
Arm/Group Description	pilot study, first 16 patients who received POEM for treatment of achalasia
Overall Participants	16
Age (Count of Participants)
<=18 years	0 0%
Between 18 and 65 years	13 81.3%
>=65 years	3 18.8%
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	45 (16.22)
Sex: Female, Male (Count of Participants)
Female	4 25%
Male	12 75%
Region of Enrollment (participants) [Number]
Germany	16 100%

Outcome Measures

1. Secondary Outcome

Title	mmHg of the Lower Esophageal Sphincter 3 Months After POEM Procedure
Description	esophageal manometry is done 3 months after POEM procedure to evaluate resting lower esophageal sphincter pressure
Time Frame	manometry at 3 month after therapy

Outcome Measure Data

Analysis Population Description

Pilot study group to evaluate feasibility and safety of POEM procedure before initiating studies evaluating long-term efficacy.Patients with primary achalasia, diagnosed by established methods(contrast fluoroscopy, manometry, esophagogastroduodenoscopy(EGD)) and age greater than 18 years were included.

Arm/Group Title	POEM Patients
Arm/Group Description	pilot group of achalasia patients who received POEM treatment
Measure Participants	16
Mean (Standard Deviation) [mmHg]	11.8 (9.016200316)

2. Secondary Outcome

Title	Number of Participants With Reflux Symptoms
Description	Number of Participants with Reflux Symptoms during procedure, and 3 and 6 months, and 1, 2 and 5 years after treatment
Time Frame	during procedure, and 3 and 6 months, and 1, 2 and 5 years after treatment

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	POEM Patients
Arm/Group Description	pilot group of achalasia patients who received POEM treatment
Measure Participants	16
Number [participants]	1 6.3%

3. Primary Outcome

Title	Treatment Success Defined as Symptom Relief 3 Months After Treatment Based on an Eckhardt Score ≤ 3
Description	eckhardt score is a score to evaluate achalasia discomfort in patients. Patients are being interrogated for dysphagia, regurgitation, and retrosternal pain , correlated with the time frame of occurrence. "with every meal" giving 3 points," daily" (2 points), "sometimes" (1 Point) or "no" (0 Points), as well as weight loss,(>10 kg= 3 points, 5-10 kg=2 points, 0-5 kg=1 point, None=0 points. Scale range is from 0 Points (no achalasia) up to 12 points for the worst achalasia symptoms.
Time Frame	3 months after treatment

Outcome Measure Data

Analysis Population Description

Pilot study group to evaluate feasibility and safety of POEM procedure before initiating studies evaluating long-term efficacy.Patients with primary achalasia, diagnosed by established methods(contrast fluoroscopy, manometry, esophagogastroduodenoscopy(EGD)) and age greater than 18 years were included.

Arm/Group Title	POEM Patients
Arm/Group Description	pilot group of achalasia patients who received POEM treatment
Measure Participants	16
symptom relief 3 months after treatment	1.4 (2.217355783)
eckhardt score baseline	8.8 (1.28)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	POEM Patients
	Comments	this pilot study was created ro evaluate sample size for the following multicenter study, based on manometric outcomes. Mean values between baseline and follow-up were compared using Student ' s t -test for paired samples. P values less then 0.05, two-sided, were considered significant.R 2.13.1(R Development Core Team (2011). Subgroups (partial vs. complete myotomy) were compared using an analysis of variance test adjusted for initial values.
	Type of Statistical Test	Superiority or Other (legacy)
	Comments
Statistical Test of Hypothesis	p-Value	<0.001
	Comments
	Method	t-test, 2 sided
	Comments
Method of Estimation	Estimation Parameter	Mean Difference (Net)
	Estimated Value	-7.44
	Confidence Interval	() 95%; to
	Parameter Dispersion	Type: Standard Deviation; Value: 2.66
	Estimation Comments

4. Secondary Outcome

Title	Number of Participants With Procedure-related Adverse Events
Description	procedure-related adverse events per protocol
Time Frame	procedure to 3 months post procedure

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	POEM Patients
Arm/Group Description	pilot group of achalasia patients who received POEM treatment
Measure Participants	16
Count of Participants [Participants]	2 12.5%

5. Secondary Outcome

Title	Medication 3 Months After POEM
Description	proton pump inhibitor (PPI) use at 3 months after POEM procedure
Time Frame	3 months

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	POEM Patients
Arm/Group Description	pilot group of achalasia patients who received POEM treatment
Measure Participants	16
Number [participants]	1 6.3%

6. Secondary Outcome

Title	Duration Time Procedure
Description	duration time of POEM procedures in minutes
Time Frame	procedure

Outcome Measure Data

Analysis Population Description

Pilot study group to evaluate feasibility and safety of POEM procedure before initiating studies evaluating long-term efficacy.Patients with primary achalasia, diagnosed by established methods(contrast fluoroscopy, manometry, esophagogastroduodenoscopy(EGD)) and age greater than 18 years were included.

Arm/Group Title	POEM Patients
Arm/Group Description	pilot group of achalasia patients who received POEM treatment
Measure Participants	16
Mean (Standard Deviation) [minutes]	114 (37.48238475)

7. Secondary Outcome

Title	Days Duration Hospitalization
Description	participants were followed for the duration of hospital stay, an average of 4 days
Time Frame	days of hospitalization for POEM procedure, an average of 4 days

Outcome Measure Data

Analysis Population Description

Pilot study group to evaluate feasibility and safety of POEM procedure before initiating studies evaluating long-term efficacy.Patients with primary achalasia, diagnosed by established methods(contrast fluoroscopy, manometry, esophagogastroduodenoscopy(EGD)) and age greater than 18 years were included.

Arm/Group Title	POEM Patients
Arm/Group Description	pilot group of achalasia patients who received POEM treatment
Measure Participants	16
Mean (Standard Deviation) [days]	4.5625 (0.892094913)

8. Secondary Outcome

Title	cm Myotomy Length
Description	myotomy length in cm
Time Frame	POEM procedure

Outcome Measure Data

Analysis Population Description

Pilot study group to evaluate feasibility and safety of POEM procedure before initiating studies evaluating long-term efficacy.Patients with primary achalasia, diagnosed by established methods(contrast fluoroscopy, manometry, esophagogastroduodenoscopy(EGD)) and age greater than 18 years were included.

Arm/Group Title	POEM Patients
Arm/Group Description	pilot group of achalasia patients who received POEM treatment
Measure Participants	16
Mean (Standard Deviation) [cm]	12 (2.704933764)

9. Secondary Outcome

Title	Percentage of Participants Who Achieved Treatment Success 3 Months After Treatment
Description	eckhardt score is a score to evaluate achalasia discomfort in patients. Patients are being interrogated for dysphagia, regurgitation, and retrosternal pain , correlated with the time frame of occurrence. "with every meal" giving 3 points," daily" (2 points), "sometimes" (1 Point) or "no" (0 Points), as well as weight loss,(>10 kg= 3 points, 5-10 kg=2 points, 0-5 kg=1 point, None=0 points. Scale range is from 0 Points (no achalasia) up to 12 points for the worst achalasia symptoms. Post-myotomy eckhardt score ≤ 3 n individuals has been reached in 15 individuals.
Time Frame	3 months after treatment

Outcome Measure Data

Analysis Population Description

Pilot study group to evaluate feasibility and safety of POEM procedure before initiating studies evaluating long-term efficacy.Patients with primary achalasia, diagnosed by established methods(contrast fluoroscopy, manometry, esophagogastroduodenoscopy(EGD)) and age greater than 18 years were included.

Arm/Group Title	POEM Patients
Arm/Group Description	pilot study, first 16 patients who received POEM for treatment of achalasia
Measure Participants	16
Number [percentage of treated patients]	94

Adverse Events

Time Frame	2 days after POEM
Adverse Event Reporting Description	1 cm superficial ulcer (Forrest III) at the cardia
Arm/Group Title	POEM Patients
Arm/Group Description	pilot group of achalasia patients who received POEM treatment
All Cause Mortality
	POEM Patients
	Affected / at Risk (%)	# Events
Total	/ (NaN)
Serious Adverse Events
	POEM Patients
	Affected / at Risk (%)	# Events
Total	2/16 (12.5%)
Gastrointestinal disorders
1 cm superficial ulcer (Forrest III)	1/16 (6.3%)	1
ulcer in the distal esophagus	1/16 (6.3%)	1
Other (Not Including Serious) Adverse Events
	POEM Patients
	Affected / at Risk (%)	# Events
Total	1/16 (6.3%)
Gastrointestinal disorders
treatment failure	1/16 (6.3%)	1

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

All Principal Investigators ARE employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.