Status:
ENROLLING_BY_INVITATION
Preparation of IPSC for Cell Gene Editing for the Treatment of AATD
Lead Sponsor:
Fondazione IRCCS Policlinico San Matteo di Pavia
Collaborating Sponsors:
University of Bern
Conditions:
Alpha1-antitrypsin Deficiency
Eligibility:
All Genders
18+ years
Phase:
NA
Brief Summary
Alpha 1-antitrypsin (AAT) deficiency is a genetic condition that leads to lung and/or liver diseases; current treatment of weekly augmentation of AAT addresses only lung diseases with moderate efficac...
Detailed Description
AAT is majorly produced in the hepatocytes therefore correction in the hepatocyte is a most promising approach. iHep cells as well as pluripotent stem cell or mesenchymal stem cell-derived hepatocyte-...
Eligibility Criteria
Inclusion
- age ≥ 18 years at the time of signing the ICF
- diagnosis of severe AATD (2 pathological variants)
Exclusion
- incapability to give informed consent
- subject under augmentation therapy
Key Trial Info
Start Date :
January 15 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
February 28 2026
Estimated Enrollment :
3 Patients enrolled
Trial Details
Trial ID
NCT06892236
Start Date
January 15 2025
End Date
February 28 2026
Last Update
March 24 2025
Active Locations (1)
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1
Fondazione IRCCS Policlinico San Matteo, Sc Pneumologia
Pavia, Pavia, Italy, 27100