Esophageal Dysmotility
Conditions
Brief summary
The purpose of the study is to prospectively evaluate the most effective treatment of esophageal dysmotility that alleviates symptoms of dysphagia and improves quality of life. Current practice uses either semi-rigid Savary dilators or balloon dilators for esophageal dilation to treat dysphagia due to esophageal dysmotility. The study aims to show which treatment method is more effective in alleviating symptoms, since there are no other treatments available. The null hypothesis is that there is no difference between the clinical benefits of each treatment.
Detailed description
Both the Savary and balloon dilators are used as routine procedure at University of Massachusetts (UMass) Medical School's academic medical center UMass Memorial Health Care (UMMHC). Gastroenterologists are required as part of their training to be familiar with both types of procedures. This study aims to definitively determine which treatment method is more effective in alleviating the symptoms of esophageal dysmotility. The primary clinical endpoint is to assess the therapeutic efficacy of different types of dilators in an esophageal dilation procedure in patients with esophageal dysmotility. This will be evaluated through the use of the dysphagia scale (Knyrim et al, 1993). The secondary aims are to assess time to relapse, diet improvement, as well as change in quality of life. Investigators will determine time to relapse by time between the dilation procedures. Participants are instructed to contact the GI office to schedule another dilation if they feel as though the therapeutic effect of the procedure has subsided and they are in need of additional relief. Diet will be assessed using the diet score (Cox et al, 1998) and quality of life will be assessed using the Short Form (SF) SF-12 Health Survey. If patients fail the first procedure, they can be offered the alternative procedure at a later date. Both the patient and physician must agree that the treatment has not worked, and then the patient will be given the opportunity to cross over to the other procedure, still blinded to the device that will be used. Investigators will conduct an interim evaluation to assess the progress of the study and degree of crossover. This will ensure that all subjects are given the opportunity to have both devices used in the event that one is significantly superior to the other.
Interventions
DEVICEDilation by Balloon
Esophageal dilation by balloon device.
DEVICEDilation by Semi-rigid Savary
Esophageal dilation by semi-rigid savary device
Sponsors
University of Massachusetts, Worcester
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
SINGLE (Subject)
Intervention model description
Participants are randomized to procedure, but if participant fails the first procedure they are given the opportunity to crossover to the other arm for a follow up procedure.
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* \> 18 years old * Dysphagia to liquids and/or solids * Diagnosis of esophageal dysmotility * Normal endoscopic exam
Exclusion criteria
* Diagnosis of achalasia * Defined strictures or webs * Vulnerable populations: * Adults unable to consent (Individuals who are not yet adults (infants, children, teenagers), Pregnant women, Prisoners)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Therapeutic Efficacy as Measured by Change in Dysphagia Scale Score | Baseline to 6 months post-procedure | Therapeutic efficacy of different types of dilators in an esophageal dilation procedure in patients with esophageal dysmotility. This will be evaluated through the use of the dysphagia scale (Knyrim 1993). Minimum score is 0. Maximum score is 4. Higher scores indicate a worse outcome. The data presented represent a change from baseline to 6 months calculated as the value at 6 months minus the value at baseline. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Participants With Relapse | Baseline to 6 months | Participants who required esophageal dilation within time frame |
| Time to Relapse | Initial intervention to second dilation | For the participants who relapsed, measure of time to relapse based on time from procedure to need for additional dilation due to subsidence of therapeutic effect of procedure. |
| Diet Dysphagia Score Change | baseline to 6 months post procedure | Diet dysphagia score change based on change to Cox diet score, taken from Table 1 of publication Gut, 1988, 29, 1741-1747. The scores are determined based on the foods that cause dysphagia. The minimum value on the score is 0 (no dysphagia), followed by 1 (dysphagia with meat), 2 (dysphagia with bread), 3 (dysphagia with semi-solids), 4 (dysphagia with liquids), and the maximum score is 5 (total dysphagia). A higher score indicates a worse clinical outcome. The data presented represent a change from baseline to 6 months calculated as the value at 6 months minus the value at baseline |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Dilation by Balloon Esophageal dilation by balloon device.
Dilation by Balloon: Esophageal dilation by balloon device. | 5 |
| Dilation by Semi-rigid Savary Esophageal dilation by semi-rigid savary device.
Dilation by Semi-rigid Savary: Esophageal dilation by semi-rigid savary device | 5 |
| Total | 10 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Physician Decision | 1 | 0 |
Baseline characteristics
| Characteristic | Dilation by Semi-rigid Savary | Dilation by Balloon | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 3 Participants | 1 Participants | 4 Participants |
| Age, Categorical Between 18 and 65 years | 2 Participants | 4 Participants | 6 Participants |
| Age, Continuous | 56.8 years | 66 years | 61.4 years |
| Region of Enrollment United States | 5 participants | 5 participants | 10 participants |
| Sex: Female, Male Female | 4 Participants | 4 Participants | 8 Participants |
| Sex: Female, Male Male | 1 Participants | 1 Participants | 2 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 4 | 0 / 5 |
| other Total, other adverse events | 4 / 4 | 5 / 5 |
| serious Total, serious adverse events | 1 / 4 | 1 / 5 |
Outcome results
Primary
Therapeutic Efficacy as Measured by Change in Dysphagia Scale Score
Therapeutic efficacy of different types of dilators in an esophageal dilation procedure in patients with esophageal dysmotility. This will be evaluated through the use of the dysphagia scale (Knyrim 1993). Minimum score is 0. Maximum score is 4. Higher scores indicate a worse outcome. The data presented represent a change from baseline to 6 months calculated as the value at 6 months minus the value at baseline.
Time frame: Baseline to 6 months post-procedure
Population: Data missing from participants who were lost to follow up at 6 months. Data provided for participants who completed 6 month follow up dysphagia score assessment.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Dilation by Balloon | Therapeutic Efficacy as Measured by Change in Dysphagia Scale Score | -1 units on a scale |
| Dilation by Semi-rigid Savary | Therapeutic Efficacy as Measured by Change in Dysphagia Scale Score | -0.25 units on a scale |
Secondary
Diet Dysphagia Score Change
Diet dysphagia score change based on change to Cox diet score, taken from Table 1 of publication Gut, 1988, 29, 1741-1747. The scores are determined based on the foods that cause dysphagia. The minimum value on the score is 0 (no dysphagia), followed by 1 (dysphagia with meat), 2 (dysphagia with bread), 3 (dysphagia with semi-solids), 4 (dysphagia with liquids), and the maximum score is 5 (total dysphagia). A higher score indicates a worse clinical outcome. The data presented represent a change from baseline to 6 months calculated as the value at 6 months minus the value at baseline
Time frame: baseline to 6 months post procedure
Population: Data is incomplete. Only 1 participant provided full diet score survey at 6 months.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Dilation by Balloon | Diet Dysphagia Score Change | -2 units on a scale |
Secondary
Participants With Relapse
Participants who required esophageal dilation within time frame
Time frame: Baseline to 6 months
Population: The population of those who were enrolled in the arms and received the assigned intervention.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Dilation by Balloon | Participants With Relapse | 2 Participants |
| Dilation by Semi-rigid Savary | Participants With Relapse | 0 Participants |
Secondary
Time to Relapse
For the participants who relapsed, measure of time to relapse based on time from procedure to need for additional dilation due to subsidence of therapeutic effect of procedure.
Time frame: Initial intervention to second dilation
Population: Participants who experienced a relapse in time period from baseline to 6 months. Only 2 participants in the dilation by balloon group experienced a relapse. This population was used to calculate the time to relapse. No participants in the dilation by semi-rigid Savary experienced a relapse during this time.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Dilation by Balloon | Time to Relapse | 72 days |