Dysphagia in Oculopharyngeal Muscular Dystrophy (OPMD)- Evaluation, Endoscopic Examination of Swallowing, Treatment and Long Term Follow up

Sponsor

Hillel Yaffe Medical Center (Other)

Overall Status

Unknown status

CT.gov ID

NCT01167439

Collaborator

(none)

Enrollment

Location

Arms

Study Details

Study Description

Brief Summary

The investigators aimed to review the natural history of dysphagia and dysphonia in OPMD in order to identify the best candidates and the proper timing to perform dysphagia alleviating procedures in both heterozygote and homozygote patients from the large pool of cases with this disease in Israel.

Condition or Disease	Intervention/Treatment	Phase
Dysphagia	Procedure: Upper esophageal sphincter myotomy	Phase 1

Detailed Description

Clinical evaluation of patients suspected of having OPMD. Genetic confirmatory tests (diagnostic DNA test) at the genetic unit in Afula Hospital. Clinical follow-up with endoscopic fiber optic evaluation of swallowing. Pre-operative assessment. Crycopharyngeal myotomy intervention in selected patients. Nutrition follow-up.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

40 participants

Allocation:

Non-Randomized

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Supportive Care

Official Title:

Dysphagia in Oculopharyngeal Muscular Dystrophy. Evaluation, Endoscopic Examination of Swallowing, Treatment and Long Term Follow up.

Study Start Date :

Jul 1, 2009

Anticipated Primary Completion Date :

Jul 1, 2011

Arms and Interventions

Arm	Intervention/Treatment
No Intervention: Mild dysphagia
Active Comparator: Severe dysphagia	Procedure: Upper esophageal sphincter myotomy Upper esophageal sphincter release operation to allow better swallowing. The intervention is done under GA and by extra-mucosal approach.

Arm

Intervention/Treatment

No Intervention: Mild dysphagia

Active Comparator: Severe dysphagia

Procedure: Upper esophageal sphincter myotomy

Upper esophageal sphincter release operation to allow better swallowing. The intervention is done under GA and by extra-mucosal approach.

Outcome Measures

Primary Outcome Measures

Dysphagia, quality of life. Proper intervention timing in OPMD based on knowledge about the natural history of the feeding difficulty. [at least 3 years]
Dysphagia, quality of life. Proper intervention timing in OPMD patients is very important and must be based on deep knowledge about the natural history of the feeding difficulty.