POEM-F for Achalasia International Study

Last updated: November 29, 2024
Sponsor: Chinese University of Hong Kong
Overall Status: Active - Recruiting

Phase

N/A

Condition

Achalasia

Esophageal Disorders

Treatment

Conventional POEM

POEM-F

Clinical Study ID

NCT06042127
2023 145-T
  • Ages 18-65
  • All Genders

Study Summary

Per-oral endoscopic myotomy (POEM) has emerged as the endoscopic treatment of choice for achalasia, offering comparable symptom relief with laparoscopic Heller's cardiomyotomy. The main concern with POEM is the higher incidence of post-procedure gastroesophageal reflux disease (GERD), occurring in up to 50-60% of patients. In order to reduce the risk of GERD, endoscopic fundoplication has been developed as a novel procedure mimicking surgical anterior partial fundoplication that can be performed in the same session as POEM (POEM-F). Case series of POEM-F in patients with achalasia reported encouraging outcomes of low GERD rate of ~12% at 1 year. Prospective comparative data between POEM-F and conventional POEM on post-procedure GERD is current lacking. The investigators therefore designed an international multicenter prospective randomized study to investigate the efficacy of POEM-F. The investigators postulate that POEM-F could reduce the incidence of post-procedure GERD when compared with conventional POEM.

This is an international multicenter randomized controlled trial conducted between high volume expert centers from Hong Kong SAR, China, India and United States of America. Adult patients with manometry confirmed achalasia would be randomised to undergo POEM-F or POEM. The procedure would be performed by experts with vast experience in POEM. The primary outcome is the incidence of post-procedure GERD at 1 year, defined by the updated Lyon consensus. Secondary outcomes include technical and clinical success rates, adverse events, post-POEM endoscopic and manometry findings as well as patients' symptom scores.

Sample size calculation Based on existing pilot comparative data on POEM-F and POEM, it is estimated that 84 patients would be required to demonstrate a difference in post-procedure GERD of 47.6% to 18.2%, with 80% power and false positive rate of 0.05, accounting for 10% loss to follow-up.

Purpose and potential The current study proposal could demonstrate the superiority of POEM-F over POEM in reducing post-procedural GERD. It would also demonstrate the safety and reproducibility of the technique in expert centers across the globe. It could potentially replace conventional POEM as the preferred minimally invasive endoscopic treatment for achalasia.

Eligibility Criteria

Inclusion

Inclusion criteria:

  1. Adult patient (age ≥18 and ≤65 years-old) and with symptomatic achalasia type I orII.

  2. Capability of understanding and complying with the study requirements, includingfilling the Eckardt Score, GERD-HRQL and RSI questionnaire and signing the informedconsent form.

  3. Patients with achalasia type I, II or III who are one of the following:

  • Treatment naïve, or

  • Failed prior through-the-scope balloon dilation, Savary or pneumatic dilation

Exclusion

Exclusion criteria:

  1. Patients unable or unwilling to provide consent.

  2. Previous esophageal or gastric surgery.

  3. Prior achalasia treatment including Heller myotomy, POEM.

  4. Sigmoid achalasia, or significant esophageal dilatation >6cm in lower esophagus

  5. Disrupted mucosal integrity at the distal esophagus, eg. Ulcer, fibrotic scars etc

  6. Patients with large hiatal hernias (axial length > 2 cm and Hill grade >2).

  7. Patients with significant cardiorespiratory comorbidities which may limit theirability to undertake general anesthesia for the procedure, including ASA grade IIIor above.

  8. Patients with obesity (Body Mass Index (BMI) ≥ 30).

  9. Pregnant women or those planning pregnancy or breastfeeding women.

  10. Uncorrectable coagulopathy defined by international normalized ratio (INR) > 1.5 orplatelet count < 50000/µl.

Study Design

Total Participants: 81
Treatment Group(s): 2
Primary Treatment: Conventional POEM
Phase:
Study Start date:
December 01, 2023
Estimated Completion Date:
October 31, 2026

Study Description

Achalasia is the most common esophageal motility disorder worldwide, with an annual incidence of 1.6 per 100'000 individual and prevalence of 10 per 100'000 population. The incidence of achalasia has been dramatically rising over the past decade. A study conducted in Chicago suggested that over the 10 years from 2004 to 2014, the incidence and prevalence of achalasia were two- to threefold greater than estimates would have predicted. The likely explanation to the rise in incidence is an increased awareness of this condition and widespread availability of accurate diagnostic test with high resolution manometry. The cardinal features of achalasia include failed relaxation of lower esophageal sphincter and absent esophageal peristalsis. Owing to the benign nature of the disease, patients suffering from it would experience longstanding debilitating symptoms of dysphagia, chest pain, regurgitation, resulting in poor quality of life. In the past, laparoscopic Heller's cardiomyotomy with partial fundoplication and endoscopic pneumatic dilatation were considered as treatment options for achalasia.

In the past 10 years, per-oral endoscopic myotomy (POEM) has emerged as the incisionless minimally invasive endoscopic treatment of choice for achalasia. This Natural Orifice Transluminal Endoscopic Surgical (NOTES) technique involves mucosal incision and creation of a submucosal tunnel in the distal esophagus down to gastric cardia, followed by esophageal and gastric myotomy. Since the procedure was first reported by Inoue H, et al in 2010, it has seen worldwide acceptance as first-line therapy. POEM has been associated with excellent relief of dysphagia and achalasia-related symptoms with a low and acceptable adverse event rate of 0.5%. In a previous randomised controlled trial, POEM had a higher treatment success rate than pneumatic dilatation. When compared with laparoscopic Heller's cardiomyotomy, POEM was associated with at least similar treatment efficacy and a trend towards reduced short term adverse event. POEM could perform even better than surgical myotomy in patients with type III achalasia.

The main limitation of POEM is the incidence of gastroesophageal reflux disease (GERD) post-POEM. Kumbhari et al. looked at rates of GERD after POEM on patients who underwent subsequent objective pH testing. A total of 282 patients were included in this analysis from multiple centers in America, Asia, and Europe. About 58% of patients had objective evidence of abnormal acid exposure, with 23% showing evidence of esophagitis. More concerning, however, was that 60% of the patients with GERD were completely asymptomatic. This highlights an important issue at hand that many patients, either related to the propensity of GERD to be silent or the nature of achalasia to develop and insensate esophagus, do not report symptoms while showing high rates of GERD. POEM has been found to be consistently associated with higher rate of GERD than conventional Heller's cardiomyotomy or pneumatic dilatation across multiple studies.

Supported by evidence demonstrating the efficacy of partial fundoplication during Heller's operation, there has been a significant interest in performing fundoplication after POEM during the same session to prevent the development of post-POEM GERD. Inoue et al. pioneered the technique of POEM-F that mimics the surgical Dor (anterior partial fundoplication) procedure. In the pilot study, technical success was achieved in all 21 patients, and all but one had an intact fundoplication wrap on upper endoscopy at a 1-month follow-up. Four more studies conducted in Japan and India have reported good early outcomes with POEM-F (Technical success of 85-100%) for achalasia. No severe adverse event has been reported in these studies which reiterates the safety of this procedure. In the longest follow-up study on POEM-F, GERD diagnosed by an abnormal esophageal acid exposure was seen in 11.1% of the 21 cases at one-year follow-up: comparable (8.8%) to a large meta-analysis of 4871 Heller's cardiomyotomy with fundoplication procedures.

To date, no prospective multicenter randomised study has been conducted to compare the efficacy of POEM-F with conventional POEM in reducing post-procedure GERD. Therefore, in this study, the investigators aim to evaluate the of POEM-F in patients with achalasia through an international multicenter randomized controlled trial.

Connect with a study center

  • Nanfang Hospital, Southern Medical University

    Guangzhou,
    China

    Site Not Available

  • The Chinese University of Hong Kong

    Hong Kong,
    Hong Kong

    Active - Recruiting

  • Post Graduate Institute of Medical Education and Research

    Chandigarh,
    India

    Site Not Available

  • Asian Institute of Gastroenterology

    Hyderabad,
    India

    Site Not Available

  • Baldota Institute of Digestive Sciences

    Mumbai,
    India

    Site Not Available

  • Deenanath Mangeshkar Hospital & Research Center

    Pune,
    India

    Site Not Available

  • Johns Hopkins Hospital

    Baltimore, Maryland 21287
    United States

    Site Not Available

Not the study for you?

Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.