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Induced Pluripotent Stem Cells for the Development of Novel Drug Therapies for Inborn Errors of Metabolism (iPSC-IEM)

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CENTOGENE

Status

Enrolling

Conditions

Inborn Errors of Metabolism

Treatments

Other: Skin biopsy
Other: Withdraw of blood

Study type

Observational

Funder types

Industry

Identifiers

NCT04097275
iPSC-IEM-2019

Details and patient eligibility

About

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 drug development. It is the goal of this study to define new cellular disease models for Inborn Erors of Metabolism, as enabling tools for both drug discovery and development.

Enrollment

1,000 estimated patients

Sex

All

Ages

2 months to 50 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

  • 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 criteria

  • 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

Trial design

1,000 participants in 1 patient group

Participants with an Inborn Error of Metabolism
Treatment:
Other: Skin biopsy
Other: Withdraw of blood

Trial contacts and locations

1

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Central trial contact

Peter Bauer, Prof.

Data sourced from clinicaltrials.gov

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