Ignite Creation Date:
2025-12-24 @ 5:42 PM
Ignite Modification Date:
2026-02-06 @ 10:14 AM
Study NCT ID:
NCT04120168
Status:
COMPLETED
Last Update Posted:
2022-10-24
First Post:
2019-10-07
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Study Determining the Frequency of Duchenne Muscular Dystrophy and Late-onset Pompe Disease
Sponsor:
Turkish Society of Pediatric Gastroenterology, Hepatology and Nutrition
Organization:
Study Overview
Official Title:
Multicenter Non-Drug Screening Study to Determine the Frequency of Duchenne Muscular Dystrophy and Late-onset Pompe Disease in Children With Unexplained Transaminase Elevation
Status:
COMPLETED
Status Verified Date:
2022-05
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
VICTORIA
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 histories of the patients included in the study will be collected at the first admission. The following laboratory values of the patients will be collected:
* Alanine Transaminase (ALT)
* Aspartate Transaminase (AST)
* Gamma Glutamyl Transferase (GGT)
* Creatine Phosphokinase (CPK)
* In addition, physical examination information and Abdominal USG and Liver Biopsy Results, if any, will be collected. Following the above scans, enzyme analysis for late-onset Pompe disease in boys and girls and adolescents with high CPK levels and molecular genetic tests for Duchenne muscular dystrophy in boys and adolescents with high CPK levels will be performed.
Demographic data, medical and family histories of the patients included in the study will be collected at the first admission. The following laboratory values of the patients will be collected:
* Alanine Transaminase (ALT)
* Aspartate Transaminase (AST)
* Gamma Glutamyl Transferase (GGT)
* Creatine Phosphokinase (CPK)
* In addition, physical examination information and Abdominal USG and Liver Biopsy Results, if any, will be collected. Following the above scans, enzyme analysis for late-onset Pompe disease in boys and girls and adolescents with high CPK levels and molecular genetic tests for Duchenne muscular dystrophy in boys and adolescents with high CPK levels will be performed.
Detailed Description:
None
Study Oversight
Has Oversight DMC:
False
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: