An interventional study of Botulinum toxin A and Placebo in Gastroparesis, sponsored by Temple University. Completed at 1 site in United States. Open to participants aged 18 Years to 70 Years. Per ClinicalTrials.gov, last updated 2015-06-09.
Sponsored by Temple University · Not applicable, Interventional, and Treatment
It is hypothesized that in some patients with gastroparesis increased pyloric tone may be a contributing feature. Botox relaxes the pylorus so that food can empty the stomach more rapidly. Lesser quality studies have shown that this treatment works in about 40% of patients, and relieves symptoms for up to 3 months. This study compares this treatment to placebo (saline) injection. After a 1 month period patients may elect to receive open label botox who have not received relief from their first injection. Patients symptoms and gastric emptying are followed for 1 year.
Patients with gastroparesis, or delayed gastric emptying, present with early satiety, postprandial bloating, nausea, vomiting, and abdominal pain or discomfort.1 Gastric emptying is a highly regulated process reflecting the integration of propulsive forces generated by proximal fundic tone and distal antral contractions with the resistance of the pyloric sphincter. Antral hypomotility as well as increased gastric outlet resistance due to pyloric dysfunction or pylorospasm appear to be important physiologic disturbances in gastroparesis.2-4 Current treatment for gastroparesis employs prokinetic agents that increase antral contractility and accelerate gastric emptying.5 Unfortunately, prokinetic agents have limited efficacy and trials of these agents have suffered from significant methodological flaws.6 Troublesome side effects such as cardiac arrhythmias in the case of cisapride (Propulsid, Janssen Pharmaceutica) and extrapyramidal symptoms and sedation in the case of metoclopramide (Reglan, A.H. Robins) have limited the usefulness of these agents.7 Domperidone may be effective for treating symptoms of gastroparesis, however it is unavailable in the U.S.8
Botulinum toxin (Botox, Allergan) is an inhibitor of cholinergic neuromuscular transmission and has been used to treat spastic disorders of both striated and smooth muscles by local injection into affected muscles.9 Previous published work from our institution demonstrated that injection of botulinum toxin into the pylorus improved gastric emptying and reduces symptoms in idiopathic gastroparesis.10 In our open label study, patients had a 38% reduction in gastroparesis symptoms when interviewed 4 weeks after injection. Seventy percent of patients had improved gastric emptying. No immediate or short term (within 6 months) untoward events occurred in our study. The beneficial effect of botulinum toxin injection was suggested to be through decreasing pyloric resistance; however, manometric analysis of this region was not performed. Our results are similar to those seen in other studies that have demonstrated accelerated gastric emptying in response to pyloric botulinum toxin injection.11-13 These studies have included groups of patients with both idiopathic and diabetic gastroparesis. Unfortunately, in all studies, the patient groups have been very small and the study design has been open label that might bias results in favor of a positive response. In addition, follow-up has been for only 6 months.
Despite limited data, many gastroenterologists are now using botulinum toxin injection for the treatment of gastroparesis outside the context of clinical research studies. We are concerned that this practice may be increasing nationwide without definitive proof of efficacy. A randomized, placebo-controlled trial is necessary to establish the usefulness of pyloric botulinum toxin injection for gastroparesis. Botulinum toxin therapy is expensive and may not be efficacious. In addition, if efficacious, the mechanism by which botulinum toxin improves gastric emptying needs to be studied. This research protocol will answer several questions concerning this potentially useful therapy for gastroparesis.
307 studies on the registry are indexed under Gastroparesis; 68 are open to participants now.
This study's enrollment of 32 is below the median of 44 across 202 interventional studies indexed under Gastroparesis.
Browse Gastroparesis studies →Temple University is the lead sponsor of 219 studies on the registry; 30 are open to participants now.
Of its 12 completed or terminated interventional studies of FDA-regulated products, 7 (58%) have results posted.
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Exclusion Criteria:
200 U of Botox injected endoscopically into pylorus
Drug: Botulinum toxin A
saline into pylorus.
Drug: Placebo
200 U given by injection into the pylorus.
Also known as: Botox into pylorus
saline injection into pylorus.
Also known as: saline injection into pylorus
Symptom Response as Assessed by the Gastroparesis Cardinal Symptom Index.
Scale for GI symptoms related to gastroparesis. For this we will use the gastroparesis cardinal symptom index (GCSI). The GCSI is based on three subscales: post-prandial fullness/early satiety (4 items); nausea/vomiting (3 items), and bloating (2 items). Scores range from 0-5 for the nine items and an asymptomatic patient would have a score of 0 with a highly symptomatic patient having a score of 45. For this study the score had to be \>=27. In a previous study internal consistency reliability was 0.84 for the GCSI total score and ranged from 0.83 to 0.85 for the subscale scores. Two week test retest reliability was 0.76 for the total score and ranged from 0.68 to 0.81 for subscale scores.
Time frame: 1 month
Patients all recruited at Temple University Hospital from 7/1/2005 to 9/1/2007
| Milestone | Botulinum Toxin | Placebo |
|---|---|---|
| Started | 16 | 16 |
| Completed | 16 | 16 |
| Not completed | 0 | 0 |
Scale for GI symptoms related to gastroparesis. For this we will use the gastroparesis cardinal symptom index (GCSI). The GCSI is based on three subscales: post-prandial fullness/early satiety (4 items); nausea/vomiting (3 items), and bloating (2 items). Scores range from 0-5 for the nine items and an asymptomatic patient would have a score of 0 with a highly symptomatic patient having a score of 45. For this study the score had to be \>=27. In a previous study internal consistency reliability was 0.84 for the GCSI total score and ranged from 0.83 to 0.85 for the subscale scores. Two week test retest reliability was 0.76 for the total score and ranged from 0.68 to 0.81 for subscale scores.
| units on a scale | Botox | Placebo |
|---|---|---|
| Symptom Response as Assessed by the Gastroparesis Cardinal Symptom Index. | 6.8 ± 9.2 | 10.1 ± 12.7 |
Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Botox | — | 0/16 (0%) | 6/16 (37.5%) |
| Placebo | — | 0/16 (0%) | 3/16 (18.8%) |
| Event | Botox | Placebo |
|---|---|---|
| HeadacheNervous system disorders | 6/16 | 3/16 |
| FatigueNervous system disorders | 3/16 | 3/16 |
| OtherGeneral disorders | 0/16 | 0/16 |
| otherGeneral disorders | 0/16 | 0/16 |
All patients with documented gastroparesis
| Age, Categorical(Participants) | Botulinum Toxin | Placebo | Total |
|---|---|---|---|
| <=18 years | 0 | 0 | 0 |
| Between 18 and 65 years | 16 | 16 | 32 |
| >=65 years | 0 | 0 | 0 |
| Age, Continuous(years) | Botulinum Toxin | Placebo | Total |
|---|---|---|---|
| Mean | 41.6 ± 11.6 | 40.4 ± 13.0 | 41.1 ± 12.2 |
| Sex: Female, Male(Participants) | Botulinum Toxin | Placebo | Total |
|---|---|---|---|
| Female | 13 | 13 | 26 |
| Male | 3 | 3 | 6 |
| Region of Enrollment(participants) | Botulinum Toxin | Placebo | Total |
|---|---|---|---|
| United States | 16 | 16 | 32 |
This study is completed, as verified in May 2015. You cannot join it, but the record below documents what was studied.
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