Botulinum Toxin Injection in Hypercontractile Esophagus

NCT ID: NCT01955174

Last Updated: 2025-12-19

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

24 participants

Study Classification

INTERVENTIONAL

Study Start Date

2013-08-31

Study Completion Date

2018-02-28

Brief Summary

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This study aims to evaluate the efficacy and the safety of endoscopic injection of 100 IU of botulinum toxin (BTX) in the distal esophagus in patients with symptoms related to hypercontractile esophageal motility disorders.

Detailed Description

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Eligible patients will present chest pain and/or dysphagia related to the following hypercontractile esophageal motility disorders: distal esophageal spasm, jackhammer esophagus, nutcracker esophagus or type III achalasia with normalization of the integrated relaxation pressure after treatment, based on the Chicago classification of esophageal motility disorders for high resolution manometry (HRM). Upper gastrointestinal endoscopy and barium swallow will be performed before BTX injection to eliminate secondary disorders.

This is a prospective, randomized, double blind, controlled trial comparing BTX injection to sham procedure (absence of injection, the clinical team performing the follow-up will not be aware of the result of the randomization).

Drugs which could affect esophageal motility (nitrates and calcium channel blockers) will be stopped during the study.

Included patients will undergo esophageal endoscopic ultrasound examination (EEUS) and upper gastrointestinal endoscopy under general anesthesia. In absence of contraindications, patients will be randomized in two arms: BTX injection or no injection. The active treatment group will receive 100 units of type A BTX (Botox®, Allergan) diluted in 10 mL of saline serum; BTX will be injected into the lower third of the esophageal wall in 10 sites between 2 and 10 cm above the squamo-columnar junction. The control arm will receive no injection after the EEUS and upper GI endoscopy (sham procedure).

Clinical response will be assessed based on the evolution of the Eckardt score, a quality of life score (Gastrointestinal Quality of Life Index (GIQLI), and weight gain. A significant clinical response will be defined as an Eckardt score \< 3 (together with individual scores \< 2). Manometry patterns will be compared before and after the procedure. Safety will be monitored based on the occurrence of chest pain scored on a Likert scale and occurrence of adverse events.

Follow-up clinical evaluation will be performed 1 and 2 months after the procedure. A second esophageal HRM will be realized 3 months after the procedure, followed by endoscopic BTX injection in case of non-response and two monthly visits.

Follow-up visits will be done 6 and 12 months after the first procedure. An additional HRM will be performed at 12 months.

Conditions

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Nutcracker Oesophagus

Keywords

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Botulinum toxin Esophageal motility disorder Distal esophageal spasm

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Outcome Assessors

Study Groups

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Botulinum toxin injection

Esophageal endoscopic injection of botulinum toxin

Group Type EXPERIMENTAL

Esophageal endoscopic injection of botulinum toxin

Intervention Type DRUG

Endoscopic injection of botulinum toxin in the esophageal muscle : one hundred units of type A botulinum toxin (Botox®, Allergan) diluted in 10 mL of normal saline and injected at the lower third of the esophageal wall in 10 sites between 2 and 10 cm above the Z-line.

No injection

No injection of botulinum toxin

Group Type SHAM_COMPARATOR

No injection of botulinum toxin

Intervention Type OTHER

Upper gastro-intestinal endoscopy without any injection

Interventions

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Esophageal endoscopic injection of botulinum toxin

Endoscopic injection of botulinum toxin in the esophageal muscle : one hundred units of type A botulinum toxin (Botox®, Allergan) diluted in 10 mL of normal saline and injected at the lower third of the esophageal wall in 10 sites between 2 and 10 cm above the Z-line.

Intervention Type DRUG

No injection of botulinum toxin

Upper gastro-intestinal endoscopy without any injection

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

* Patient with non-cardiac chest pain and/or dysphagia with global Eckardt score strictly above 3 points or equal to 3 only for " chest pain " or " dysphagia " - Hypercontractile esophagus with normal esogastric junction relaxation based on the Chicago classification in high resolution manometry
* Man or woman 18-year old or older
* Normal upper gastrointestinal endoscopy within one year before inclusion
* Barium swallow without argument for external compression
* Effective form of birth control (if applicable)
* Signed written informed consent form voluntarily
* Patient with health insurance

Exclusion Criteria

* Man or woman under 18-year old
* Past history of eso-gastric surgery
* Evolutive cancer or coagulation disorders
* Absence of effective form of birth control (if applicable)
* Pregnant woman or woman who plans to become pregnant during the expected length of the study
* Breastfeeding woman
* Allergy to botulinum toxin or excipients
* Myasthenia
* Aminoglycoside treatment
* Endoscopic contraindication prior to the study
* Endoscopic contraindication during the study
* Psychiatric or addictive disease which could affect compliance to the constraints of the study
* Patient refusing to participate to the study
* Language barrier limiting the understanding of the study
* Incapability to give consent
* Concomitant participation to another research study
* No written consent form
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Hospices Civils de Lyon

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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François MION, MD

Role: PRINCIPAL_INVESTIGATOR

Hospices Civils de Lyon

Locations

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Service d'Hépato-Gastro-Entérologie et Oncologie Digestive- Hôpital Haut Lévêque - CHU de Bordeaux

Bordeaux, , France

Site Status

Service d'Exploration Fonctionnelle Digestive - Hôpital Edouard Herriot Pavillon H 5 - Hospices Civils de Lyon

Lyon, , France

Site Status

Countries

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France

References

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Mion F, Marjoux S, Subtil F, Pioche M, Rivory J, Roman S, Zerbib F. Botulinum toxin for the treatment of hypercontractile esophagus: Results of a double-blind randomized sham-controlled study. Neurogastroenterol Motil. 2019 May;31(5):e13587. doi: 10.1111/nmo.13587. Epub 2019 Apr 11.

Reference Type RESULT
PMID: 30974039 (View on PubMed)

Other Identifiers

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2012.779

Identifier Type: -

Identifier Source: org_study_id