Status:
UNKNOWN
Induced Pluripotent Stem Cells for the Development of Novel Drug Therapies for Inborn Errors of Metabolism (iPSC-IEM)
Lead Sponsor:
CENTOGENE GmbH Rostock
Conditions:
Inborn Errors of Metabolism
Eligibility:
All Genders
2-50 years
Brief Summary
Human induced pluripotent stem cells (iPSCs), are reprogrammed from somatic cells that can self-renew indefinitely and produce different types of cells. They provide human model cell lines for orphan ...
Eligibility Criteria
Inclusion
- Informed consent is obtained from the participant or from the parent/ legal guardian
- The participant is aged between 2 months and 50 years
- The diagnosis of an Inborn Error of Metabolism (IEM) is genetically confirmed by Centogene
- The participant is a first-degree or a second-degree relative of an individual with Inborn Error of Metabolism (IEM) genetically confirmed by Centogene
Exclusion
- Inability to provide informed consent
- The participant is younger than 2 months or older than 50 years
- The diagnosis of an Inborn Error of Metabolism (IEM) is not genetically confirmed by Centogene and
- The participant is not a first-degree or a seconddegree relative of an individual with Inborn Error of Metabolism (IEM) genetically confirmed by Centogene
- Previously enrolled in the study
Key Trial Info
Start Date :
August 1 2019
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
August 1 2024
Estimated Enrollment :
1000 Patients enrolled
Trial Details
Trial ID
NCT04097275
Start Date
August 1 2019
End Date
August 1 2024
Last Update
February 10 2023
Active Locations (1)
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1
Children Hospital and Institute of Child Health
Lahore, Pakistan