A randomized, double-blind, placebo-controlled, multicenter trial to evaluate the clinical efficacy of a single intrapyloric injection of botulinum toxin type A (Botox®) in patients with idiopathic gastroparesis. The BIG study.

ISRCTN ISRCTN32422510
DOI https://doi.org/10.1186/ISRCTN32422510
Secondary identifying numbers P05.170
Submission date
09/06/2006
Registration date
09/06/2006
Last edited
09/06/2006
Recruitment status
No longer recruiting
Overall study status
Completed
Condition category
Nutritional, Metabolic, Endocrine
Prospectively registered
Protocol
Statistical analysis plan
Results
Individual participant data
Record updated in last year

Plain English summary of protocol

Not provided at time of registration

Contact information

Dr J.J.L. Haans
Scientific

Leiden University Medical Center (LUMC)
Department of Gastroenterology and Hepatology C-04-P
Room 192
P.O. Box 9600
Leiden
2300 RC
Netherlands

Phone +31 (0)71 5261104
Email J.J.L.Haans@lumc.nl

Study information

Study designA randomized, double-blind, placebo-controlled, multicenter trial
Primary study designInterventional
Secondary study designRandomised controlled trial
Study setting(s)Not specified
Study typeTreatment
Scientific title
Study acronymBIG study
Study objectivesSeveral 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(s)Not provided at time of registration
Health condition(s) or problem(s) studiedIdiopathic gastroparesis
InterventionIntrapyloric 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 typeDrug
Pharmaceutical study type(s)
PhaseNot Specified
Drug / device / biological / vaccine name(s)Botulinum toxin
Primary outcome measureGastric 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 measures1. 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 study start date01/06/2006
Completion date01/06/2007

Eligibility

Participant type(s)Patient
Age groupAdult
Lower age limit18 Years
SexBoth
Target number of participants48
Key inclusion criteriaPatients 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
Key exclusion criteriaPatients 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
Date of first enrolment01/06/2006
Date of final enrolment01/06/2007

Locations

Countries of recruitment

  • Netherlands

Study participating centre

Leiden University Medical Center (LUMC)
Leiden
2300 RC
Netherlands

Sponsor information

Leiden University Medical Center (LUMC), Department of Gastroenterology and Hepatology (The Netherlands)
University/education

P.O. Box 9600
Leiden
2300 RC
Netherlands

ROR logo "ROR" https://ror.org/05xvt9f17

Funders

Funder type

University/education

Leiden University Medical Center (LUMC) Department of Gastroenterology and Hepatology

No information available

Results and Publications

Intention to publish date
Individual participant data (IPD) Intention to shareNo
IPD sharing plan summaryNot provided at time of registration
Publication and dissemination planNot provided at time of registration
IPD sharing plan