Boost Brittle Bones Before Birth

Phase 1/2 Active not recruiting 18 enrolled Karolinska Institutet Great Ormond Street Hospital for Children NHS Foundation Trust · collab Karolinska University Hospital · collab Leiden University Medical Center · collab Lund University · collab UMC Utrecht · collab University College, London · collab Universitätsklinikum Köln · collab

Non-randomizedParallel-groupOpen-labelTreatment

Summary

An exploratory, open label, multiple dose, multicentre phase I/II trial evaluating safety and efficacy of postnatal or prenatal and postnatal administration of allogeneic expanded fetal mesenchymal stem cells for the treatment of severe Osteogenesis Imperfecta compared with a combination of historical and untreated prospective controls.

Timeline

Start: 2019-08-12
Primary completion: 2030-04
Completion: 2030-04

Publications

Sagar RL, Astrom E, Chitty LS, Crowe B, David AL, DeVile C, Forsmark A, Franzen V, Hermeren G, Hill M, Johansson M, Lindemans C, Lindgren P, Nijhuis W, Oepkes D, Rehberg M, Sahlin NE, Sakkers R, Semler O, Sundin M, Walther-Jallow L, Verweij EJTJ, Westgren M, Gotherstrom C. An exploratory open-label multicentre phase I/II trial evaluating the safety and efficacy of postnatal or prenatal and postnatal administration of allogeneic expanded fetal mesenchymal stem cells for the treatment of severe osteogenesis imperfecta in infants and fetuses: the BOOSTB4 trial protocol. BMJ Open. 2024 Jun 4;14(6):e079767. doi: 10.1136/bmjopen-2023-079767.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	BOOST cells	Cell therapy	3 cells/kg	Intravenous

Boost Brittle Bones Before Birth

Summary

Timeline

Publications

Drugs

Indications