Contact information
Type
Scientific
Primary contact
Dr J.J.L. Haans
ORCID ID
Contact details
Leiden University Medical Center (LUMC)
Department of Gastroenterology and Hepatology C-04-P
Room 192
P.O. Box 9600
Leiden
2300 RC
Netherlands
+31 (0)71 5261104
J.J.L.Haans@lumc.nl
Additional identifiers
EudraCT number
ClinicalTrials.gov number
Protocol/serial number
P05.170
Study information
Scientific title
Acronym
BIG study
Study hypothesis
Several open-label pilot studies have shown a promising effect of intrapyloric botulinum toxin injection on symptoms in severe gastroparesis. However, data from randomized, double-blind, placebo-controlled studies are not yet available. We intend to evaluate the clinical efficacy of a single intrapyloric injection of botulinum toxin type A in patients with idiopathic gastroparesis.
Ethics approval
Not provided at time of registration
Study design
A randomized, double-blind, placebo-controlled, multicenter trial
Primary study design
Interventional
Secondary study design
Randomised controlled trial
Trial setting
Not specified
Trial type
Treatment
Patient information sheet
Condition
Idiopathic gastroparesis
Intervention
Intrapyloric injection of botulinum toxin type A 200 IU single injection or placebo single injection.
Scintigraphy for solid/liquid gastric emptying (before and after treatment).
Gastroparesis cardinal symptom index questionnaire
Quality of life (QOL) questionnaires
Psychometric assessment and patient perception questionnaires
Intervention type
Drug
Phase
Not Specified
Drug names
Botulinum toxin
Primary outcome measures
Gastric emptying for solids.
1. Half-emptying time at visit 4 (follow-up: week 6) compared to visit 1b (eligibility: week 1)
2. Emptying rate per hour at visit 4 (follow-up: week 6) compared to visit 1b (eligibility: week 1)
3. One, two and four hour meal retention at visit 4 (follow-up: week 6) compared to visit 1b (eligibility week 1).
Gastroparesis cardinal symptom index (GCSI) outcome
4. Total GCSI score at visit 3, 4, 5 and 6 (follow-up: week 3, 6, 9 and 12) compared to visit 2 (randomization: week 0)
5. Mean GCSI score during follow-up (week 0-12) compared to mean GCSI score during eligibility and randomization (week 10)
Secondary outcome measures
1. Gastric emptying for liquids; retention of liquid phase at 15, 30, 45, 60, 75, 90, 105 and 120 min
2. Quality of life as measured with the patient assessment of upper gastrointestinal disorders - quality of life (PAGI-QoL), the RAND-36 and EQ-5D questionnaires
3. Psychometric assessment and patient perception of gastrointestinal endoscopy
Overall trial start date
01/06/2006
Overall trial end date
01/06/2007
Reason abandoned
Eligibility
Participant inclusion criteria
Patients are eligible to participate in this study if all of the following criteria are met:
1. Presence of clinical symptoms associated with idiopathic gastroparesis: nausea, vomiting, early satiety, postprandial fullness and a Gastroparesis Cardinal Symptom Index (GCSI) score ≥1 at visit 1b (eligibility; week 1)
2. Delayed gastric emptying for solids at visit 1b (eligibility; week 1) as measured with scintigraphy:
a. 2h retention ≥60%; and/or
b. 4h retention ≥10%
3. Absence of an obstructing structural lesion in the stomach or small intestine at prior diagnostic gastrointestinal endoscopy (performed within the previous 5 years) or judged absent by the responsible physician
4. Patients, both male and female, must use adequate means of birth control during the study
5. Patients must provide written informed consent prior to any study procedures being performed
6. Patients aged between 18 and 70 years inclusive
7. Patients, in the opinion of the investigator, must be able to understand the study and comply with the study requirements
Participant type
Patient
Age group
Adult
Gender
Both
Target number of participants
48
Participant exclusion criteria
Patients are excluded from the study if any of the following criteria are met:
1. Patients with predominant abdominal pain/discomfort in the opinion of the investigator
2. Presence of an obstructing structural lesion in the stomach or small intestine if observed during treatment gastrointestinal endoscopy (visit 2, randomization; week 0)
3. Patients taking drugs that interfere with the effect of botulinum toxin type A
4. Patients previously treated with intrapyloric injection of botulinum toxin type A;
5. Females who are pregnant or lactating
6. Patients with diabetes mellitus
7. Patients with delayed gastric emptying due to systemic disorders, e.g. systemic lupus erythematosus (SLE), sclerodermia, hypothyroidism
8. Patients with disorders of neuromuscular transmission, e.g. myasthenia gravis and Eaton-Lambert syndrome
9. Patients with abdominal surgery in their medical history, except (laparoscopic) appendectomy, (laparoscopic) cholecystectomy and/or hysterectomy
10. Patients with any condition which, in the opinion of the investigator, makes the patient unsuitable for entry into the study
Recruitment start date
01/06/2006
Recruitment end date
01/06/2007
Locations
Countries of recruitment
Netherlands
Trial participating centre
Leiden University Medical Center (LUMC)
Leiden
2300 RC
Netherlands
Funders
Funder type
University/education
Funder name
Leiden University Medical Center (LUMC) Department of Gastroenterology and Hepatology
Alternative name(s)
Funding Body Type
Funding Body Subtype
Location
Results and Publications
Publication and dissemination plan
Not provided at time of registration
Intention to publish date
Participant level data
Not provided at time of registration
Results - basic reporting
Publication summary