Creation of disease model systems to understand and correct genetic diseases through gene or other therapy using iPS cells derived from somatic cells: the iPS study Addendum A: "Informatie voor proefpersonen en bijbehorend toestemmingsformulier voor de vervaardiging en het gebruik van geïnduceerde Pluripotente Stamcellen (iPS cellen) voor patiënten met een cardiovasculaire aandoening."

Creation of disease model systems to understand and correct genetic diseases through gene or other therapy using iPS cells derived from somatic cells: *the iPS study* Addendum A: "Informatie voor proefpersonen en bijbehorend toestemmingsformulier voor de vervaardiging en het gebruik van geïnduceerde Pluripotente Stamcellen (iPS cellen) voor patiënten met een cardiovasculaire aandoening." - The iPS study

Status

Active, not recruiting

Phases

Unknown

Study type

Observational

Source

NL-OMON

Registry ID

NL-OMON45114

Enrollment

600

Registered

2014-07-02

Start date

2016-01-29

Completion date

Unknown

Last updated

2024-04-23

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

genetic disease heritable disorder

Interventions

None listed

Eligibility

Age

2 Years to 99 Years

Inclusion criteria

Inclusion criteria: Patients suffering from diseases of genetic or non-genetic origin, including but not limited to cardiovascular, neural and blood disorders. Related or unrelated healthy individuals will serve as controls. In principle patients of all ages are eligible, as some forms of hereditary disease already affect patients at young age and may potentially even be fatal at that age.

Exclusion criteria

Exclusion criteria: Patients tested as HIV or hepatitis positive.

Design outcomes

Primary

Measure	Time frame
Main study parameters/endpoints: - Ability to generate iPSCs and differentiated derivative cells from patients* somatic cells - Ability to investigate the phenotype associated with the disease-specific cells - Ability to (genetically) repair the underlying cause of the disease - Ability to generate lineage reporter hiPSC lines - Ability to ameliorate disease phenotype using small molecules, drugs or si/shRNA	—

Measure

Time frame

Main study parameters/endpoints: - Ability to generate iPSCs and differentiated derivative cells from patients* somatic cells - Ability to investigate the phenotype associated with the disease-specific cells - Ability to (genetically) repair the underlying cause of the disease - Ability to generate lineage reporter hiPSC lines - Ability to ameliorate disease phenotype using small molecules, drugs or si/shRNA

—

Secondary

Measure	Time frame
- Ability to screen for new compounds/ development of new drugs.	—

Countries

Netherlands

Outcome results