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Clinical Trials in Italy / NCT06892236
Enrolling by invitation Not applicable

Preparation of IPSC for Cell Gene Editing for the Treatment of AATD

NCT06892236 · tracked via the Priya Life Science Italy tracker
Sponsor
Fondazione IRCCS Policlinico San Matteo di Pavia
Phase
Not applicable
Started
2025-01-15
Last updated
2025-03-24

Condition(s) studied

Alpha1-antitrypsin Deficiency

Investigational drug(s) / intervention(s)

iPSC generation

iPSC generation: Base editing

Study 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 efficacy. Novel treatments based on gene editing can restore physiological levels of AAT and address lung and liver disease.

The aim is to generate induced pluripotent stem cells (iPSC) from blood and urine of patients with different severe Alpha1-antitrypsin deficiency (AATD) genotypes. Further, the iPSC will be differentiated into hepatocytes (iHep). Since hepatocytes are the main producers of AAT, the iHep will be used to test different approaches of gene editing to correct various mutations. Gene editing will be conducted at University of Bern (Switzerland)

Eligibility

Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria: * age ≥ 18 years at the time of signing the ICF * diagnosis of severe AATD (2 pathological variants) Exclusion Criteria: * incapability to give informed consent * subject under augmentation therapy

Primary outcome measure(s)

  • Number of severe AATD patients included in the study — 24 months

Trial sites (1)

FacilityCityRegionStatus
Fondazione IRCCS Policlinico San Matteo, Sc Pneumologia Pavia Pavia

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Other trials for the same condition

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Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06892236 on ClinicalTrials.gov ↗ ← All trials in Italy