Botulinum toxin injection for treating swallowing difficulties

Botulinum Toxin Injection in the Upper Esophageal Sphincter for Retrograde-cricopharyngeus Dysfunction: a Prospective, Double Blind, Placebo-controlled Trial

This study is testing if botulinum toxin injections can help people with swallowing difficulties feel better over time.

Quick facts

• Phase: Phase 4
• Study type: Interventional
• Enrollment: 30 (estimated)
• Ages: 18 Years to 65 Years
• Sex: All
• Sponsor: AZ Delta (Academic / other)
• Locations: 1 site (Roeselare)
• Trial ID: NCT06356025 on ClinicalTrials.gov

What this trial studies

This study evaluates the effectiveness of botulinum toxin injections into the upper esophageal sphincter for patients suffering from retrograde cricopharyngeus dysfunction. It is a double-blind, placebo-controlled trial that aims to assess symptom improvement both short-term (1-20 weeks) and long-term (up to 48 weeks) after the injection. Participants will complete questionnaires to report their symptoms at various intervals, and those who do not experience significant improvement may receive a second treatment with active botulinum toxin in an open-label manner.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 to 65 who have been unable to belch for at least six months and find this condition bothersome.

Not a fit: Patients who do not experience bothersome symptoms related to their inability to belch may not benefit from this treatment.

Why it matters

Potential benefit: If successful, this treatment could significantly alleviate swallowing difficulties and improve quality of life for affected patients.

How similar studies have performed: Other studies have shown promising results with botulinum toxin for similar conditions, indicating potential for success in this approach.

Eligibility criteria

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).

Where this trial is running

Roeselare

• AZ Delta — Roeselare, Belgium (Recruiting)

Study contacts

• Principal investigator: Kathelijne G Delsupehe, MD — AZ Delta
• Study coordinator: Kathelijne G Delsupehe, MD

Conditions Retrograde Cricopharyngeus Dysfunction