Digestive Events in Duchenne Muscular Dystrophy Patients

Centre d'Investigation Clinique et Technologique 805200 enrolled

Overview

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

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 Type

OBSERVATIONAL

Enrollment

200

Conditions

Duchenne Muscular Dystrophy

Eligibility

Sex: MALEMin age: 15 Years

Medical Language ↔ Plain English

Inclusion Criteria: * all Duchenne muscular dystrophy patients in our center Exclusion Criteria: * none

Locations (1)

CICIT805

Garches, France

Outcomes

Primary Outcomes

Incidence of digestive events

Clinical parameters (yes or no): gastrostomy, bowel occlusion, acute gastric paresia, biliary complication

Time frame: 11 years

Prevalence of digestive events

Clinical parameters (yes or no) : gastrostomy, bowel occlusion, acute gastric paresia, biliary complication

Time frame: 11 years

Risk factors for digestive events (1)

age (years)

Time frame: 11 years

Risk factors for digestive events (2)

pulmonary functional tests (vital capacity in ml/kg)

Time frame: 11 years

Risk factors for digestive events (3)

echocardiography parameters (LVEF: left ventricular ejection fraction in %)

Time frame: 11 years

Risk factors for digestive events (4)

residual dystrophin level (Western blot and/or immunohistochemistry in muscle)

Time frame: 11 years

Risks factors for digestive events (5)

genetic parameters (type of mutation, functional domain of altered dystrophin)

Time frame: 11 years

Secondary Outcomes

Nutritional status (1)

Weigh (kg) and height (in m) agregated in BMI (kg/m\^2)

Time frame: 11 years

Data from ClinicalTrials.gov

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