| Autor: |
Anna L David, Mats Johansson, Magnus Westgren, Belinda Crowe, Dick Oepkes, Melissa Hill, Lyn S Chitty, Catherine DeVile, Peter Lindgren, Eva Åström, Cecilia Götherström, Nils-Eric Sahlin, Rachel L Sagar, Annabelle Forsmark, Vera Franzen, Göran Hermeren, Caroline Lindemans, Wouter Nijhuis, Mirko Rehberg, Ralph Sakkers, O Semler, Mikael Sundin, Lilian Walther-Jallow, E J T Joanne Verweij |
| Jazyk: |
angličtina |
| Rok vydání: |
2024 |
| Předmět: |
|
| Zdroj: |
BMJ Open, Vol 14, Iss 6 (2024) |
| Druh dokumentu: |
article |
| ISSN: |
2044-6055 |
| DOI: |
10.1136/bmjopen-2023-079767 |
| Popis: |
Introduction Severe osteogenesis imperfecta (OI) is a debilitating disease with no cure or sufficiently effective treatment. Mesenchymal stem cells (MSCs) have good safety profile, show promising effects and can form bone. The Boost Brittle Bones Before Birth (BOOSTB4) trial evaluates administration of allogeneic expanded human first trimester fetal liver MSCs (BOOST cells) for OI type 3 or severe type 4.Methods and analysis BOOSTB4 is an exploratory, open-label, multiple dose, phase I/II clinical trial evaluating safety and efficacy of postnatal (n=15) or prenatal and postnatal (n=3, originally n=15) administration of BOOST cells for the treatment of severe OI compared with a combination of historical (1–5/subject) and untreated prospective controls (≤30). Infants |
| Databáze: |
Directory of Open Access Journals |
| Externí odkaz: |
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