NCT02663206

Brief Summary

To compare the efficacy of peroral endoscopic myotomy and Botulinum toxin injection in spastic esophageal disorders.

Trial Health

30
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Timeline
Completed

Started Sep 2016

Longer than P75 for not_applicable

Geographic Reach
1 country

1 active site

Status
withdrawn

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

January 5, 2016

Completed
21 days until next milestone

First Posted

Study publicly available on registry

January 26, 2016

Completed
7 months until next milestone

Study Start

First participant enrolled

September 1, 2016

Completed
3.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2020

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2020

Completed
Last Updated

January 12, 2018

Status Verified

January 1, 2018

Enrollment Period

3.8 years

First QC Date

January 5, 2016

Last Update Submit

January 10, 2018

Conditions

Esophageal AchalasiaEsophageal Spasm, DiffuseEsophageal Motility Disorders

Keywords

spastic esophageal disordersachalasiadiffuse esophageal spasmhypercontractile (jackhammer) esophagusBotulinum toxin injectionperoral endoscopic myotomy

Outcome Measures

Primary Outcomes (1)

  • Eckardt score

    symptoms scores

    3-month

Secondary Outcomes (5)

  • Eckardt score

    1-year

  • changes in individual symptom scores

    3 months and 12 months

  • changes in esophageal manometry

    3 months

  • rate of complications

    3 months and 12 months

  • quality of life scores

    3 months and 12 months

Study Arms (2)

Botulinum toxin injection

OTHER

Drug/Device: Botulinum toxin injection; Endoscopic Botulinum toxin (BTX) injection at lower esophagus; Upper endoscopy with Botulinum toxin injection. The procedure will be performed as an outpatient basis by an endoscopist. Sedation can be in form of conscious sedation, monitored anesthesia care or general anesthesia. An upper endoscope will be inserted into the patient's mouth and advanced into lower esophagus. Botulinum toxin (Botox@) 100 units 8-10 (25 units/mL) will be injected in 1-ml portion in each of four quadrants about 1 cm above the Z-line (the LES region). * At 1 month follow-up, patients who do not response to the first botox injection (Eckardt score \> 3) will receive the second botox injection. * At 3-month follow-up, POEM will be offered as a rescue therapy to both non-responders (Eckardt score \> 3 at 3-month follow-up after the procedure) and relapsers (Eckardt score ≤ 3 at 3-month follow-up but becomes \> 3 during the follow-up)

Procedure: Botulinum toxin injection

peroral endoscopic myotomy

OTHER

Procedure/Surgery: peroral endoscopic myotomy. The procedure will be performed by an endoscopist (gastroenterologist or surgeon). General anesthesia will be started and upper endoscope will be inserted into the patient's mouth and advanced into the stomach. Endoscopic myotomy will be performed. Mucosal entry will then be closed using endoscopic clips or endoscopic suturing. All patients will recover from their procedures according to standard practice. They will remain nothing per oral (NPO) the night after the procedure and started on intravenous proton pump inhibitors. A gastrografin esophagram will be obtained the next day and if no evidence of leak, the diet will be advanced to a soft diet for two weeks. The patients will be evaluated by study coordinator/PI on a daily basis during their hospitalization.

Procedure: peroral endoscopic myotomy

Interventions

peroral endoscopic myotomyPROCEDURE

peroral endoscopic myotomy

peroral endoscopic myotomy
Botulinum toxin injectionPROCEDURE

endoscopic Botulinum toxin (BTX) injection at lower esophagus

Botulinum toxin injection

Eligibility Criteria

Age18 Years - 80 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Adult patients age 18 - 80 years old.
  • Spastic disorders of the esophagus include spastic (type III) achalasia, distal esophageal spasm (DES), and hypercontractile (jackhammer) esophagus via high resolution esophageal manometry (HRM) 2.
  • DES is characterized by normal esophagogastric junction relaxation (integrated relaxation pressure \[IRP\] \<15 mm Hg) and ≥ 20% premature contractions.
  • Spastic achalasia is defined as impaired EGJ relaxation (IRP ≥15 mm Hg) associated with ≥ 20% premature contractions.
  • The diagnosis of jackhammer esophagus is defined as at least 1 swallow with a distal contractile integral (DCI) greater than 8000 mm Hg- s- cm.
  • At least 6 months of symptoms (chest pain, dysphagia, regurgitation and/or weight loss) with no adequate response or intolerance to medical therapy including nitrates and/or calcium channel blockers.
  • Overall symptoms score (Eckardt score) \> 3
  • Ability to understand and the willingness to sign a written informed consent document

You may not qualify if:

  • Diagnosis of spastic esophageal disorder was not confirmed by HRM testing.
  • Previous surgery of the esophagus or stomach
  • Previous BTX injection at the esophagogastric junction (EGJ) or LES.
  • Active severe esophagitis
  • Large lower esophageal diverticula
  • Large \> 3cm hiatal hernia
  • Megaesophagus (\> 6 cm)
  • Sigmoid esophagus
  • Known gastroesophageal malignancy
  • Inability to tolerate sedated upper endoscopy due to cardiopulmonary instability, severe pulmonary disease or other contraindication to endoscopy
  • Cirrhosis with portal hypertension, varices, and/or ascites
  • Uncorrectable coagulopathy defined by prothrombin time \< 50% of control; partial thromboplastin time (PTT) \> 50 sec, or international normalized ratio (INR) \> 1.5), on chronic anticoagulation, or platelet count \<75,000.
  • Pregnant or breastfeeding women (all women of child-bearing age will undergo urine pregnancy testing)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Johns Hopkins Hospital

Baltimore, Maryland, 21287, United States

Location

MeSH Terms

Conditions

Esophageal AchalasiaEsophageal Spasm, DiffuseEsophageal Motility Disorders

Interventions

Botulinum Toxins

Condition Hierarchy (Ancestors)

Deglutition DisordersEsophageal DiseasesGastrointestinal DiseasesDigestive System Diseases

Intervention Hierarchy (Ancestors)

MetalloendopeptidasesEndopeptidasesPeptide HydrolasesHydrolasesEnzymesEnzymes and CoenzymesMetalloproteasesBacterial ProteinsProteinsAmino Acids, Peptides, and ProteinsBacterial ToxinsToxins, BiologicalBiological Factors

Study Officials

  • Mouen A Khashab, MD

    Johns Hopkins University

    PRINCIPAL INVESTIGATOR
0

Study Design

Study Type
interventional
Phase
not applicable
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 5, 2016

First Posted

January 26, 2016

Study Start

September 1, 2016

Primary Completion

July 1, 2020

Study Completion

July 1, 2020

Last Updated

January 12, 2018

Record last verified: 2018-01

Locations

US(1)