Esophageal Dysmotility - Prospective Study Evaluating Methods for Esophageal Dilation

• Sponsor: University of Massachusetts, Worcester (Other)
• Overall Status: Terminated
• CT.gov ID: NCT03604523
• Collaborator: (none)
• Enrollment: 10
• Location: 1
• Arms: 2
• Actual Duration (Months): 27.4
• Patients Per Site Per Month: 0.4

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Esophageal Dysmotility	Device: Dilation by Balloon; Device: Dilation by Semi-rigid Savary	N/A

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.

Study Design

• Study Type: Interventional
• Actual Enrollment: 10 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• 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.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.
• Masking: Single (Participant)
• Primary Purpose: Treatment
• Official Title: Effectiveness of Semi-rigid Savary Dilators vs. Balloon Dilators During Esophageal Dilation in the Treatment of Esophageal Dysmotility
• Actual Study Start Date: Aug 14, 2014
• Actual Primary Completion Date: Nov 26, 2016
• Actual Study Completion Date: Nov 26, 2016

Arms and Interventions

Arm	Intervention/Treatment
Active Comparator: Dilation by Balloon Esophageal dilation by balloon device.	Device: Dilation by Balloon; Esophageal dilation by balloon device.
Active Comparator: Dilation by Semi-rigid Savary Esophageal dilation by semi-rigid savary device.	Device: Dilation by Semi-rigid Savary; Esophageal dilation by semi-rigid savary device

Outcome Measures

Primary Outcome Measures

1. 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 Outcome Measures

1. Participants With Relapse [Baseline to 6 months]

   Participants who required esophageal dilation within time frame

2. 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.

3. 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

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts 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)

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	UMass Memorial Medical Center	Worcester	Massachusetts	United States	01655

Sponsors and Collaborators

• University of Massachusetts, Worcester

Investigators

• Principal Investigator: David Cave, MD, Professor of Medicine, UMass Medical School

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: David Cave, Professor, University of Massachusetts, Worcester
• ClinicalTrials.gov Identifier: NCT03604523
• Other Study ID Numbers: H00004174
• First Posted: Jul 27, 2018
• Last Update Posted: Apr 13, 2021
• Last Verified: Mar 1, 2021

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: Yes
• Product Manufactured in and Exported from the U.S.: Yes
• Additional relevant MeSH terms: Esophageal Motility Disorders; Esophageal Spasm, Diffuse

Study Results

Participant Flow

Arm/Group Title	Dilation by Balloon	Dilation by Semi-rigid Savary
Arm/Group Description	Esophageal dilation by balloon device. Dilation by Balloon: Esophageal dilation by balloon device.	Esophageal dilation by semi-rigid savary device. Dilation by Semi-rigid Savary: Esophageal dilation by semi-rigid savary device
Period Title: Overall Study
STARTED	5	5
COMPLETED	4	5
NOT COMPLETED	1	0

Baseline Characteristics

Arm/Group Title	Dilation by Balloon	Dilation by Semi-rigid Savary	Total
Arm/Group Description	Esophageal dilation by balloon device. Dilation by Balloon: Esophageal dilation by balloon device.	Esophageal dilation by semi-rigid savary device. Dilation by Semi-rigid Savary: Esophageal dilation by semi-rigid savary device	Total of all reporting groups
Overall Participants	5	5	10
Age (Count of Participants)
<=18 years	0 0%	0 0%	0 0%
Between 18 and 65 years	4 80%	2 40%	6 60%
>=65 years	1 20%	3 60%	4 40%
Age (years) [Mean (Full Range) ]
Mean (Full Range) [years]	66	56.8	61.4
Sex: Female, Male (Count of Participants)
Female	4 80%	4 80%	8 80%
Male	1 20%	1 20%	2 20%
Region of Enrollment (participants) [Number]
United States	5 100%	5 100%	10 100%

Outcome Measures

1. Primary Outcome

Title	Therapeutic Efficacy as Measured by Change in Dysphagia Scale Score
Description	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

Outcome Measure Data

Analysis Population Description

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/Group Title	Dilation by Balloon	Dilation by Semi-rigid Savary
Arm/Group Description	Esophageal dilation by balloon device. Dilation by Balloon: Esophageal dilation by balloon device.	Esophageal dilation by semi-rigid savary device. Dilation by Semi-rigid Savary: Esophageal dilation by semi-rigid savary device
Measure Participants	3	4
Mean (Full Range) [units on a scale]	-1	-0.25

2. Secondary Outcome

Title	Participants With Relapse
Description	Participants who required esophageal dilation within time frame
Time Frame	Baseline to 6 months

Outcome Measure Data

Analysis Population Description

The population of those who were enrolled in the arms and received the assigned intervention.

Arm/Group Title	Dilation by Balloon	Dilation by Semi-rigid Savary
Arm/Group Description	Esophageal dilation by balloon device. Dilation by Balloon: Esophageal dilation by balloon device.	Esophageal dilation by semi-rigid savary device. Dilation by Semi-rigid Savary: Esophageal dilation by semi-rigid savary device
Measure Participants	4	5
Count of Participants [Participants]	2 40%	0 0%

3. Secondary Outcome

Title	Time to Relapse
Description	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

Outcome Measure Data

Analysis Population Description

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/Group Title	Dilation by Balloon	Dilation by Semi-rigid Savary
Arm/Group Description	Esophageal dilation by balloon device. Dilation by Balloon: Esophageal dilation by balloon device.	Esophageal dilation by semi-rigid savary device. Dilation by Semi-rigid Savary: Esophageal dilation by semi-rigid savary device
Measure Participants	2	0
Mean (Full Range) [days]	72

4. Secondary Outcome

Title	Diet Dysphagia Score Change
Description	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

Outcome Measure Data

Analysis Population Description

Data is incomplete. Only 1 participant provided full diet score survey at 6 months.

Arm/Group Title	Dilation by Balloon	Dilation by Semi-rigid Savary
Arm/Group Description	Esophageal dilation by balloon device. Dilation by Balloon: Esophageal dilation by balloon device.	Esophageal dilation by semi-rigid savary device. Dilation by Semi-rigid Savary: Esophageal dilation by semi-rigid savary device
Measure Participants	1	0
Number [units on a scale]	-2

Adverse Events

Time Frame	Baseline to 6 months after procedure
Arm/Group Title	Dilation by Balloon		Dilation by Semi-rigid Savary
Arm/Group Description	Esophageal dilation by balloon device. Dilation by Balloon: Esophageal dilation by balloon device.		Esophageal dilation by semi-rigid savary device. Dilation by Semi-rigid Savary: Esophageal dilation by semi-rigid savary device
All Cause Mortality
	Dilation by Balloon		Dilation by Semi-rigid Savary
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/4 (0%)		0/5 (0%)
Serious Adverse Events
	Dilation by Balloon		Dilation by Semi-rigid Savary
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	1/4 (25%)		1/5 (20%)
Nervous system disorders
Migraine	1/4 (25%)	1	0/5 (0%)	0
Respiratory, thoracic and mediastinal disorders
Shortness of breath	0/4 (0%)	0	1/5 (20%)	1
Other (Not Including Serious) Adverse Events
	Dilation by Balloon		Dilation by Semi-rigid Savary
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	4/4 (100%)		5/5 (100%)
Gastrointestinal disorders
Dysphagia	3/4 (75%)	3	0/5 (0%)	0
Dyspepsia	0/4 (0%)	0	1/5 (20%)	1
General disorders
Chest pain	1/4 (25%)	1	1/5 (20%)	1
Musculoskeletal and connective tissue disorders
Joint pain	2/4 (50%)	2	1/5 (20%)	1
Leg cramps	0/4 (0%)	0	1/5 (20%)	1
Leg pain	0/4 (0%)	0	1/5 (20%)	1

Limitations/Caveats

Recruitment was much slower than planned and the study was terminated early with only 15.6% of overall study goal enrolled. Early termination led to small numbers of subjects analyzed. Subjects unable to be reached for followup led to missing data for outcome measures.

More Information

Certain Agreements

All Principal Investigators ARE employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.