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Clinical Trials/DRKS00032765
DRKS00032765
Recruiting
N/A

Health-related quality of life in children and adolescents with Medium-chain-acyl-CoA dehydrogenase deficiency (MCADD) - Health-related quality of life in MACDD children

niversität Leipzig0 sites25 target enrollmentOctober 24, 2023

Overview

Phase
N/A
Intervention
Not specified
Conditions
E71.3
Sponsor
niversität Leipzig
Enrollment
25
Status
Recruiting
Last Updated
last year

Overview

Brief Summary

No summary available.

Registry
who.int
Start Date
October 24, 2023
End Date
TBD
Last Updated
last year
Study Type
Observational
Sex
All

Investigators

Sponsor
niversität Leipzig

Eligibility Criteria

Inclusion Criteria

  • MCADD diagnosis by newborn screening and enzymatic and/or genetic confirmation of diagnosis
  • \-From birth until 18 years of age
  • \-Signed consent (patients and caregivers)
  • \-No life\-threatening metabolic crisis until study enrollment

Exclusion Criteria

  • \-MCADD diagnosis in the context of a metabolic crisis and not through newborn screening
  • \-Patients proved to be heterozygote for MCADD after positive new born screening
  • \-Additional severe chronical diseases with regular use of medication or dietary treatment
  • \-participation in another interventional trial

Outcomes

Primary Outcomes

Not specified

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