Trial Outcomes & Findings for Esophageal Dysmotility - Prospective Study Evaluating Methods for Esophageal Dilation (NCT NCT03604523)

Results Overview

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.

Recruitment status

TERMINATED

Study phase

NA

Target enrollment

10 participants

Primary outcome timeframe

Baseline to 6 months post-procedure

Results posted on

2021-04-13

Participant Flow

Participant milestones

Participant milestones
Measure	Dilation by Balloon Esophageal dilation by balloon device. Dilation by Balloon: Esophageal dilation by balloon device.	Dilation by Semi-rigid Savary Esophageal dilation by semi-rigid savary device. Dilation by Semi-rigid Savary: Esophageal dilation by semi-rigid savary device
Overall Study STARTED	5	5
Overall Study COMPLETED	4	5
Overall Study NOT COMPLETED	1	0

Reasons for withdrawal

Reasons for withdrawal
Measure	Dilation by Balloon Esophageal dilation by balloon device. Dilation by Balloon: Esophageal dilation by balloon device.	Dilation by Semi-rigid Savary Esophageal dilation by semi-rigid savary device. Dilation by Semi-rigid Savary: Esophageal dilation by semi-rigid savary device
Overall Study Physician Decision	1	0

Baseline Characteristics

Esophageal Dysmotility - Prospective Study Evaluating Methods for Esophageal Dilation

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Dilation by Balloon n=5 Participants Esophageal dilation by balloon device. Dilation by Balloon: Esophageal dilation by balloon device.	Dilation by Semi-rigid Savary n=5 Participants Esophageal dilation by semi-rigid savary device. Dilation by Semi-rigid Savary: Esophageal dilation by semi-rigid savary device	Total n=10 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Age, Categorical Between 18 and 65 years	4 Participants n=93 Participants	2 Participants n=4 Participants	6 Participants n=27 Participants
Age, Categorical >=65 years	1 Participants n=93 Participants	3 Participants n=4 Participants	4 Participants n=27 Participants
Age, Continuous	66 years n=93 Participants	56.8 years n=4 Participants	61.4 years n=27 Participants
Sex: Female, Male Female	4 Participants n=93 Participants	4 Participants n=4 Participants	8 Participants n=27 Participants
Sex: Female, Male Male	1 Participants n=93 Participants	1 Participants n=4 Participants	2 Participants n=27 Participants
Region of Enrollment United States	5 participants n=93 Participants	5 participants n=4 Participants	10 participants n=27 Participants

PRIMARY outcome

Timeframe: 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.

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.

Outcome measures

Outcome measures
Measure	Dilation by Balloon n=3 Participants Esophageal dilation by balloon device. Dilation by Balloon: Esophageal dilation by balloon device.	Dilation by Semi-rigid Savary n=4 Participants Esophageal dilation by semi-rigid savary device. Dilation by Semi-rigid Savary: Esophageal dilation by semi-rigid savary device
Therapeutic Efficacy as Measured by Change in Dysphagia Scale Score	-1 units on a scale Interval -2.0 to 0.0	-0.25 units on a scale Interval -1.0 to 0.0

SECONDARY outcome

Timeframe: Baseline to 6 months

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

Participants who required esophageal dilation within time frame

Outcome measures

Outcome measures
Measure	Dilation by Balloon n=4 Participants Esophageal dilation by balloon device. Dilation by Balloon: Esophageal dilation by balloon device.	Dilation by Semi-rigid Savary n=5 Participants Esophageal dilation by semi-rigid savary device. Dilation by Semi-rigid Savary: Esophageal dilation by semi-rigid savary device
Participants With Relapse	2 Participants	0 Participants

SECONDARY outcome

Timeframe: 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.

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.

Outcome measures

Outcome measures
Measure	Dilation by Balloon n=2 Participants Esophageal dilation by balloon device. Dilation by Balloon: Esophageal dilation by balloon device.	Dilation by Semi-rigid Savary Esophageal dilation by semi-rigid savary device. Dilation by Semi-rigid Savary: Esophageal dilation by semi-rigid savary device
Time to Relapse	72 days Interval 27.0 to 117.0	—

SECONDARY outcome

Timeframe: baseline to 6 months post procedure

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

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

Outcome measures

Outcome measures
Measure	Dilation by Balloon n=1 Participants Esophageal dilation by balloon device. Dilation by Balloon: Esophageal dilation by balloon device.	Dilation by Semi-rigid Savary Esophageal dilation by semi-rigid savary device. Dilation by Semi-rigid Savary: Esophageal dilation by semi-rigid savary device
Diet Dysphagia Score Change	-2 units on a scale	—

Adverse Events

Dilation by Balloon

Serious events: 1 serious events

Other events: 4 other events

Deaths: 0 deaths

Dilation by Semi-rigid Savary

Serious events: 1 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Dilation by Balloon n=4 participants at risk Esophageal dilation by balloon device. Dilation by Balloon: Esophageal dilation by balloon device.	Dilation by Semi-rigid Savary n=5 participants at risk Esophageal dilation by semi-rigid savary device. Dilation by Semi-rigid Savary: Esophageal dilation by semi-rigid savary device
Nervous system disorders Migraine	25.0% 1/4 • Number of events 1 • Baseline to 6 months after procedure	0.00% 0/5 • Baseline to 6 months after procedure
Respiratory, thoracic and mediastinal disorders Shortness of breath	0.00% 0/4 • Baseline to 6 months after procedure	20.0% 1/5 • Number of events 1 • Baseline to 6 months after procedure

Other adverse events

Other adverse events
Measure	Dilation by Balloon n=4 participants at risk Esophageal dilation by balloon device. Dilation by Balloon: Esophageal dilation by balloon device.	Dilation by Semi-rigid Savary n=5 participants at risk Esophageal dilation by semi-rigid savary device. Dilation by Semi-rigid Savary: Esophageal dilation by semi-rigid savary device
Gastrointestinal disorders Dysphagia	75.0% 3/4 • Number of events 3 • Baseline to 6 months after procedure	0.00% 0/5 • Baseline to 6 months after procedure
Musculoskeletal and connective tissue disorders Joint pain	50.0% 2/4 • Number of events 2 • Baseline to 6 months after procedure	20.0% 1/5 • Number of events 1 • Baseline to 6 months after procedure
General disorders Chest pain	25.0% 1/4 • Number of events 1 • Baseline to 6 months after procedure	20.0% 1/5 • Number of events 1 • Baseline to 6 months after procedure
Musculoskeletal and connective tissue disorders Leg cramps	0.00% 0/4 • Baseline to 6 months after procedure	20.0% 1/5 • Number of events 1 • Baseline to 6 months after procedure
Musculoskeletal and connective tissue disorders Leg pain	0.00% 0/4 • Baseline to 6 months after procedure	20.0% 1/5 • Number of events 1 • Baseline to 6 months after procedure
Gastrointestinal disorders Dyspepsia	0.00% 0/4 • Baseline to 6 months after procedure	20.0% 1/5 • Number of events 1 • Baseline to 6 months after procedure

Additional Information

Dr. David Cave

UMass Medical School

Phone: 508-856-8399

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place