Botulinum Toxin Injection in the UES for R-CPD

NCT06356025 | Active Not Recruiting Phase 4

• 2 other identifiers: 520822024-512546-42
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 1

Brief Summary

The aim of this study is to assess the effect of botulinum toxin injection into the upper esophageal sphincter in a double blind, placebo controlled study. Investigators want to assess the effect on symptoms short term (1-20 weeks after BT injection), and long term (48 weeks after BT injection). This is a prospective double-blind randomized placebo-controlled study. Questionnaires assessing symptoms will be filled out on several occasions. At 20 weeks, a reassessment of symptoms will be done, without unblinding patients or investigator. Failures (to BT or placebo, defined as no clinical improvement or improvement less than 50%) will get the chance to receive a second procedure with active treatment (BT) in open label.

Conditions

Retrograde Cricopharyngeus Dysfunction

Outcome Measures

Primary Outcomes (1)

• Response to Botox versus placebo, 12 weeks after treatment.

  This questionnaire is a treatment effect measure specific for inability to belch, where the change in symptoms from baseline is assessed, on a scale from 0% to 100%. 100% means complete resolution of symptoms, 0% means no improvement.

  week 12

Secondary Outcomes (3)

• Response to botox versus placebo 48 weeks after treatment.

  48 weeks

• Safety: count of each type of (serious) adverse events in each group

  4 weeks

• Safety on swallowing: with SSQ

  4 weeks

Other Outcomes (11)

• HRQOL

  1 to 48 weeks

• Patients experience and satisfaction of the treatment using overall treatment effect

  1-48 weeks

• Patients experience and satisfaction of the treatment using overall symptom severity

  1-48 weeks

Study Arms (2)

Botulinum toxin injection

EXPERIMENTAL

Drug: botulinum toxin type A

Placebo injection

PLACEBO COMPARATOR

Drug: Sodium Chloride 0.9% Inj

Interventions

botulinum toxin type A DRUG

Botulinum toxin type A 75U dissolved in 3mL physiological serum injection in the upper esophageal sphincter

Botulinum toxin injection

Sodium Chloride 0.9% Inj DRUG

Placebo: physiological serum 3mL injection in the upper esophageal sphincter

Placebo injection

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

Participants eligible for inclusion in this Trial must meet all of the following criteria: 1. Voluntary written informed consent of the participant or their legally authorized representative has been obtained prior to any screening procedures 2. Use of highly effective methods of birth control; defined as those that, alone or in combination, result in low failure rate (i.e., less than 1% per year) when used consistently and correctly; such as implants, injectables, combined oral contraceptives, some IUDs, true sexual abstinence (i.e. refraining from heterosexual intercourse during the entire period of risk associated with the Trial treatment(s)) or commitment to a vasectomised partner. 3. Between 18 and 65 years old 4. Inability to belch, which is bothersome enough to consider treatment\*, present for at least 6 months prior to inclusion, and in fulfillment of both categories a and b below:, 1. presence of associated gastrointestinal symptoms such as abdominal bloating and discomfort/nausea, flatulence, gurgling noises from the chest or lower neck. 2. Absence of other pharyngeal and esophageal pathologies, determined on the basis of history taking and diagnostic tests. 5. Typical findings on high resolution impedance manometry with belch provocation test (10). These typical findings include: No relaxation upon gastroesophageal gas reflux, no air clearance via the UES but instead an increased air presence time include air entrapment and oscillations. * Bothersome = severe enough to impact on usual activities, according to the Rome IV diagnostic criteria (11, 12). Participants eligible for this Trial must not meet any of the following criteria: 1. Participant has a history of surgery in the upper GI tract, that might interfere with the belch reflex (such as anti-reflux surgery) 2. Any disorder, which in the Investigator's opinion might jeopardise the participant's safety or compliance with the protocol 3. Any prior or concomitant treatment(s) that might jeopardise the participant's safety or that would compromise the integrity of the Trial, to be decided at the discretion of the investigator. 4. Female who is pregnant, breast-feeding or intends to become pregnant or is of child-bearing potential (WOCBP) and not using an adequate, highly effective contraceptive 5. Participation in another interventional Trial with an investigational medicinal product (IMP) or device 6. \>65 years old; \< 18 years of age 7. Known hypersensitivity to botulinum toxin type A or to human albumin or sodium chloride 8. Presence of infection at the proposed injection site(s).

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• AZ Delta: lead
• KU Leuven: collaborator

Study Sites (1)

AZ Delta

Roeselare, Belgium

Location

MeSH Terms

Interventions

Botulinum Toxins, Type A; Sodium Chloride

Intervention Hierarchy (Ancestors)

Botulinum Toxins; Metalloendopeptidases; Endopeptidases; Peptide Hydrolases; Hydrolases; Enzymes; Enzymes and Coenzymes; Metalloproteases; Bacterial Proteins; Proteins; Amino Acids, Peptides, and Proteins; Bacterial Toxins; Toxins, Biological; Biological Factors; Chlorides; Hydrochloric Acid; Chlorine Compounds; Inorganic Chemicals; Sodium Compounds

Study Officials

• Kathelijne G Delsupehe, MD

  AZ Delta

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 20, 2024
• First Posted: April 10, 2024
• Study Start: September 2, 2024
• Primary Completion: December 1, 2025
• Study Completion (Estimated): March 1, 2027
• Last Updated: December 8, 2025

Record last verified: 2025-12

Locations

BE (1)