Digestive Events in Duchenne Muscular Dystrophy Patients

Completed

• Conditions: Duchenne Muscular Dystrophy

• Registration Number: NCT04384354
• Lead Sponsor: Centre d'Investigation Clinique et Technologique 805

Brief Summary

Relation between clinical and genetic features and acute digestive events in Duchenne muscular dystrophy patients

Detailed Description

Clinical Data of the disease Digestive events (pseudo occlusion, gastrostomy, cholecystectomy) Nutritionnal parameters Genetic of dystrophin (approved genetic nationnal Data base, CHU Cochin, Paris France)

Study Timeline

• Study Start: 2007-01
• Primary Completion: 2018-12
• Study Completion: 2019-04
• Study First Submitted: 2019-07-19
• Status Verified: 2020-05
• Study First Submitted QC: 2020-05-11
• Last Update Submitted: 2020-05-11
• Study First Posted: 2020-05-12
• Last Update Posted: 2020-05-12

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 200

Inclusion Criteria

• all Duchenne muscular dystrophy patients in our center

Exclusion Criteria

• none

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Risks factors for digestive events (5)	11 years	genetic parameters (type of mutation, functional domain of altered dystrophin)
Incidence of digestive events	11 years	Clinical parameters (yes or no): gastrostomy, bowel occlusion, acute gastric paresia, biliary complication
Risk factors for digestive events (3)	11 years	echocardiography parameters (LVEF: left ventricular ejection fraction in %)
Risk factors for digestive events (1)	11 years	age (years)
Risk factors for digestive events (2)	11 years	pulmonary functional tests (vital capacity in ml/kg)
Prevalence of digestive events	11 years	Clinical parameters (yes or no) : gastrostomy, bowel occlusion, acute gastric paresia, biliary complication
Risk factors for digestive events (4)	11 years	residual dystrophin level (Western blot and/or immunohistochemistry in muscle)

Secondary Outcome Measures

Name	Time	Method
Nutritional status (1)	11 years	Weigh (kg) and height (in m) agregated in BMI (kg/m\^2)
Nutritional status (2)	11 years	albuminemia in g/L

Trial Locations

Locations (1)

CICIT805; 🇫🇷 Garches, France