Status:
COMPLETED
Study Determining the Frequency of Duchenne Muscular Dystrophy and Late-onset Pompe Disease
Lead Sponsor:
Turkish Society of Pediatric Gastroenterology, Hepatology and Nutrition
Conditions:
Duchenne Muscular Dystrophy
Pompe Disease (Late-onset)
Eligibility:
All Genders
3-18 years
Brief Summary
This is a multicenter prospective non-drug screening study. The working period is 12 months. There is no research product to be followed or used in the study. Demographic data, medical and family his...
Eligibility Criteria
Inclusion
- 3 months -18 years old boys and girls
- Serum transaminase levels (serum ALT and / or AST levels\> 1.52 upper limit of normal (ULN)) for at least 3 months
- The willingness of the patient and / or legal representative to sign the written consent form
Exclusion
- Patients less than 3 months
- Patients with a known history of liver disease
- Patients with a known history of muscle disease
- Patients with a known history of rheumatologic disease
- Patients with clinical history or physical examination findings that support the possibility of liver disease (Jaundice, variceal bleeding, hepatomegaly, splenomegaly, ascites)
- ICU patients
- Patients with known congenital anomalies
- Patients with organ failure
- Patients with elevated serum GGT, Total Bliribun or Direct Bilirubin levels
Key Trial Info
Start Date :
April 1 2019
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
October 21 2022
Estimated Enrollment :
590 Patients enrolled
Trial Details
Trial ID
NCT04120168
Start Date
April 1 2019
End Date
October 21 2022
Last Update
October 24 2022
Active Locations (51)
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1
Adana City Hospital
Adana, Turkey (Türkiye)
2
Baskent University Adana Hospital
Adana, Turkey (Türkiye)
3
Çukurova University Faculty of Medicine, Pediatric Gastroenterology
Adana, Turkey (Türkiye)
4
Afyonkarahisar Health Sciences University
Afyonkarahisar, Turkey (Türkiye)