Trial Outcomes & Findings for Endoscopic Peroral Myotomy for Treatment of Achalasia (NCT NCT01405469)

NCT ID: NCT01405469

Last Updated: 2019-10-15

Results Overview

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.

• Recruitment status: COMPLETED
• Study phase: NA
• Target enrollment: 16 participants
• Primary outcome timeframe: 3 months after treatment
• Results posted on: 2019-10-15

Participant Flow

Patients with symptomatic achalasia

16 patients were included as per protocol, no patient was lost during follow-up over a 2 year period

Participant milestones

Measure	POEM Patients pilot group of achalasia patients who received POEM Treatment: Endoscopic Myotomy of the Lower Esophageal Sphincter
Overall Study STARTED	16
Overall Study COMPLETED	16
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Endoscopic Peroral Myotomy for Treatment of Achalasia

Baseline characteristics by cohort

Measure	POEM Patients n=16 Participants pilot study, first 16 patients who received POEM for treatment of achalasia
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	13 Participants n=5 Participants
Age, Categorical >=65 years	3 Participants n=5 Participants
Age, Continuous	45 years STANDARD_DEVIATION 16.22 • n=5 Participants
Sex: Female, Male Female	4 Participants n=5 Participants
Sex: Female, Male Male	12 Participants n=5 Participants
Region of Enrollment Germany	16 participants n=5 Participants

PRIMARY outcome

Timeframe: 3 months after treatment

Population: 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.

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.

Outcome measures

Measure	POEM Patients n=16 Participants pilot group of achalasia patients who received POEM treatment
Treatment Success Defined as Symptom Relief 3 Months After Treatment Based on an Eckhardt Score ≤ 3 symptom relief 3 months after treatment	1.4 Eckardt Score Standard Deviation 2.217355783
Treatment Success Defined as Symptom Relief 3 Months After Treatment Based on an Eckhardt Score ≤ 3 eckhardt score baseline	8.8 Eckardt Score Standard Deviation 1.28

SECONDARY outcome

Timeframe: manometry at 3 month after therapy

Population: 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.

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

Outcome measures

Measure	POEM Patients n=16 Participants pilot group of achalasia patients who received POEM treatment
mmHg of the Lower Esophageal Sphincter 3 Months After POEM Procedure	11.8 mmHg Standard Deviation 9.016200316

SECONDARY outcome

Timeframe: 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

Outcome measures

Measure	POEM Patients n=16 Participants pilot group of achalasia patients who received POEM treatment
Number of Participants With Reflux Symptoms	1 participants

SECONDARY outcome

Timeframe: procedure to 3 months post procedure

procedure-related adverse events per protocol

Outcome measures

Measure	POEM Patients n=16 Participants pilot group of achalasia patients who received POEM treatment
Number of Participants With Procedure-related Adverse Events	2 Participants

SECONDARY outcome

Timeframe: 3 months

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

Outcome measures

Measure	POEM Patients n=16 Participants pilot group of achalasia patients who received POEM treatment
Medication 3 Months After POEM	1 participants

SECONDARY outcome

Timeframe: procedure

Population: 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.

duration time of POEM procedures in minutes

Outcome measures

Measure	POEM Patients n=16 Participants pilot group of achalasia patients who received POEM treatment
Duration Time Procedure	114 minutes Standard Deviation 37.48238475

SECONDARY outcome

Timeframe: days of hospitalization for POEM procedure, an average of 4 days

Population: 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.

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

Outcome measures

Measure	POEM Patients n=16 Participants pilot group of achalasia patients who received POEM treatment
Days Duration Hospitalization	4.5625 days Standard Deviation 0.892094913

SECONDARY outcome

Timeframe: POEM procedure

Population: 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.

myotomy length in cm

Outcome measures

Measure	POEM Patients n=16 Participants pilot group of achalasia patients who received POEM treatment
cm Myotomy Length	12 cm Standard Deviation 2.704933764

SECONDARY outcome

Timeframe: 3 months after treatment

Population: 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.

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.

Outcome measures

Measure	POEM Patients n=16 Participants pilot group of achalasia patients who received POEM treatment
Percentage of Participants Who Achieved Treatment Success 3 Months After Treatment	94 percentage of treated patients

Adverse Events

POEM Patients

Serious events: 2 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Measure	POEM Patients n=16 participants at risk pilot group of achalasia patients who received POEM treatment
Gastrointestinal disorders 1 cm superficial ulcer (Forrest III)	6.2% 1/16 • Number of events 1 • 2 days after POEM 1 cm superficial ulcer (Forrest III) at the cardia
Gastrointestinal disorders ulcer in the distal esophagus	6.2% 1/16 • Number of events 1 • 2 days after POEM 1 cm superficial ulcer (Forrest III) at the cardia

Other adverse events

Measure	POEM Patients n=16 participants at risk pilot group of achalasia patients who received POEM treatment
Gastrointestinal disorders treatment failure	6.2% 1/16 • Number of events 1 • 2 days after POEM 1 cm superficial ulcer (Forrest III) at the cardia

Additional Information

Prof. Dr. Thomas Rösch, Principal Investigator

University Hospital Hamburg-Eppendorf

Phone: +49 40 7410 50098

Email: t.roesch@uke.de

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place