Botulinum toxin type A for Retrograde cricopharyngeus dysfunction|Belching

Inclusion criteria

• {"criterion_text":"- Voluntary written informed consent of the participant or their legally authorized representative has been obtained prior to any screening procedures"}
• {"criterion_text":"- 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."}
• {"criterion_text":"- Between 18 and 65 years old"}
• {"criterion_text":"- 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:, a. presence of associated gastrointestinal symptoms such as abdominal bloating and discomfort/nausea, flatulence, gurgling noises from the chest or lower neck. b. Absence of other pharyngeal and esophageal pathologies, determined on the basis of history taking and diagnostic tests. * Bothersome = severe enough to impact on usual activities, according to the Rome IV diagnostic criteria"}
• {"criterion_text":"- 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."}

Exclusion criteria

• {"criterion_text":"- Participant has a history of surgery in the upper GI tract, that might interfere with the belch reflex (such as anti-reflux surgery)"}
• {"criterion_text":"- Any disorder, which in the Investigator’s opinion might jeopardise the participant’s safety or compliance with the protocol"}
• {"criterion_text":"- 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."}
• {"criterion_text":"- If applicable: Female who is pregnant, breast-feeding or intends to become pregnant or is of child-bearing potential and not using an adequate, highly effective contraceptive"}
• {"criterion_text":"- Participation in another interventional Trial with an investigational medicinal product (IMP) or device"}
• {"criterion_text":"- >65 years old; < 18 years of age"}
• {"criterion_text":"- Known hypersensitivity to botulinum toxin type A or to human albumin or sodium chloride."}
• {"criterion_text":"- Presence of infection at the proposed injection site(s)."}

Primary endpoints

• {"endpoint_text":"- Response to Botox versus placebo, 12 weeks after treatment. We will use the questionnaire of ‘Difficulties with belching’, and response is defined as at least a 50% improvement on question 6A ‘To what extent have the symptoms of 'difficulty burping air' been reduced after treatment?'.","definition_or_measurement_approach":"Measured using the ‘Difficulties with belching’ questionnaire; response defined as at least 50% improvement on question 6A at 12 weeks."}

Secondary endpoints

• {"endpoint_text":"- Response to Botox versus placebo, 48 weeks after treatment. We will use the questionnaire of ‘Difficulties with belching’, and response is defined as at least a 50% improvement on question 6A ‘To what extent have the symptoms of 'difficulty burping air' been reduced after treatment?'.","definition_or_measurement_approach":"Measured using the ‘Difficulties with belching’ questionnaire; response defined as at least 50% improvement on question 6A at 48 weeks."}
• {"endpoint_text":"- Safety: assessed by the number and type of (serious) adverse events that occurred in each group by week 48, and by the SSQ 1 week and 4 weeks after the procedure.","definition_or_measurement_approach":"Safety assessed by counting and categorising (serious) adverse events up to week 48 and SSQ assessments at 1 and 4 weeks post-procedure."}
• {"endpoint_text":"- Exploratory: HRQOL using EQ-5D VAS score short-term (1, 4, 12 weeks) and long-term (30 - 48 weeks after the procedure).","definition_or_measurement_approach":"Health-related quality of life measured by EQ-5D VAS at specified timepoints (1, 4, 12 weeks short-term; 30–48 weeks long-term)."}
• {"endpoint_text":"- Exploratory: Patients experience and satisfaction of the treatment based on OTE and OSS short-term (1, 4, 12 weeks after the procedure), and long-term (20 - 48 weeks after the procedure)","definition_or_measurement_approach":"Patient experience and satisfaction measured using OTE and OSS at specified short- and long-term timepoints."}
• {"endpoint_text":"- Exploratory: Evolution of associated gastro-intestinal symptoms short-term (1, 4, 12 weeks) and long-term (20 - 48 weeks after the procedure).","definition_or_measurement_approach":"Monitoring change in associated gastrointestinal symptoms at specified short- and long-term timepoints."}
• {"endpoint_text":"- Exploratory: Change in objective measurements after, versus before BT injeciton using high resolution impedance manometry at week 20, and comparing with baseline.","definition_or_measurement_approach":"Objective change measured by high resolution impedance manometry at week 20 compared with baseline."}
• {"endpoint_text":"- Exploratory: Improvement in symptoms after Botox versus placebo, 12 and 48 weeks after treatment. We will use the questionnaire of ‘Difficulties with belching’, using a cut-off for improvement of 30% on question 6A ‘To what extent have the symptoms of 'difficulty burping air' been reduced after treatment?'.","definition_or_measurement_approach":"Measured using ‘Difficulties with belching’ questionnaire; improvement defined as ≥30% improvement on question 6A at 12 and 48 weeks."}
• {"endpoint_text":"- Exploratory: Safety at week 12, 20 and 48 using SSQ","definition_or_measurement_approach":"Safety assessments using SSQ at weeks 12, 20 and 48."}

Belgium

Earliest CTIS Part Ii Submission Date

25-06-2024

Latest Decision Or Authorization Date

13-06-2025

Processing Time Days

353

Number Of Sites

1

Number Of Participants

44

Primary sponsor

Full Name

Algemeen Ziekenhuis Delta

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

Belgium