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NCT06892236
Preparation of IPSC for Cell Gene Editing for the Treatment of AATD
NA trial testing iPSC generation in Alpha1-antitrypsin Deficiency in 3 participants. Enrolling by invitation.
28 February 2026
Quick facts
| Lead sponsor | Fondazione IRCCS Policlinico San Matteo di Pavia |
|---|---|
| Phase | NA |
| Status | ENROLLING BY INVITATION |
| Study type | INTERVENTIONAL |
| Allocation | na |
| Design | single group |
| Masking | none |
| Primary purpose | other |
| Enrollment | 3 |
| Start date | 15 January 2025 |
| Primary completion | 28 February 2026 |
| Estimated completion | 28 February 2026 |
| Sites | 1 location across Italy |
Drugs / interventions tested
- iPSC generation
Conditions studied
- Alpha1-antitrypsin Deficiency — all drugs for Alpha1-antitrypsin Deficiency →
Sponsor
Fondazione IRCCS Policlinico San Matteo di Pavia
Who can join
18 and older, any sex, with Alpha1-antitrypsin Deficiency. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
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)
Publications & conference data
1 peer-reviewed publication reference this trial (live from Europe PMC):
-
Application of stem cells in the precise diagnosis and treatment of liver diseases.
Wang YX, Ren YN, Zhang SS, Sun S, et al · · 2025 · PMID 41479645 · DOI 10.3748/wjg.v31.i46.114415
Verify or expand the search:
- PubMed search for NCT06892236
- Europe PMC full search
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Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT06892236 (US National Library of Medicine, public domain)
- Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Fondazione IRCCS Policlinico San Matteo di Pavia
- Last refreshed: 24 March 2025
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT06892236.
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