frozen autologous cd34+ hematopoietic stem and progenitor cells genetically modified with the lentiviral vector idua lv, encoding for the α-l-iduronidase cdna
Cell therapyexperimental
Clinical trial activity
1 trials · 2 clinical orgs · 0 marketing orgs
Phase 1
1
Phase 2
1
Phase 3
0
Phase 4
0
Earliest trial started May 11, 2018 (NCT03488394)
Timeline
2010s
- Jan 1, 2018
Earliest Phase 1 Sponsor(trial)
Indications
Studied for
Mechanism of action
- Stem CellSupplementation
Stem Cell supplementation
Chemistry & pharmacology
Oral
No
Parenteral
No
Topical
No
Clinical trials
| NCT ID | Phase | Start date | Sponsor(s) |
|---|---|---|---|
| NCT03488394 | Phase 1/Phase 2 (Phase 2) | May 11, 2018 | Fondazione Telethon, Vita-Salute University of Milano. Italy |
Showing 1 of 1 trials
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Organizations
Research & Development (2)
| Organization | Org type | Trials | As lead sponsor | Phases | Earliest year |
|---|---|---|---|---|---|
| Fondazione Telethon | Academic/Hospital | 1 | 0 | 1 | 2018 |
| Vita-Salute University of Milano. Italy | Academic/Hospital | 1 | 1 | 1 | 2018 |
2 organizations
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