BOOSTB4 (Boost Brittle Bones Before Birth) Trial
Research type
Research Study
Full title
An exploratory, open label, multiple dose, multicentre phase I/II trial evaluating safety and efficacy of postnatal or prenatal and postnatal intravenous 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.
IRAS ID
241431
Contact name
Anna David
Contact email
Sponsor organisation
Karolinska Institutet
Eudract number
2015-003699-60
Duration of Study in the UK
13 years, 0 months, 1 days
Research summary
The BOOSTB4 trial aims to investigate whether administration of allogeneic (un-matched) fetal mesenchymal stem cells (MSCs, "BOOST cells") are a safe and effective treatment for severe types of Osteogenesis Imperfecta (OI). BOOST cells will be given either after birth to babies with OI or before birth to babies in the womb (fetus) that have a confirmed molecular diagnosis of OI. OI patients suffer with repeated painful fractures, bony deformities and short stature. There are no treatments for this debilitating condition which target the underlying pathology. In preclinical (animal) studies, MSCs improve the growth and reduce the fracture rate and bone fragility in mice with OI. MSCs have been administered to a small number of fetuses and babies with OI who appear to have had better outcomes than would otherwise have been expected. Worldwide, MSCs have been used to treat many other conditions and appear to be safe, with no evidence of tumour formation and few side effects. We aim to give the BOOST MSCs to 15 babies aged up to 12 months of age (Part 1) and 15 fetuses at 16-35+6 weeks of gestational age (Part 2). The babies or fetuses will each receive a total of 4 doses of MSC, given four months apart. For fetal (prenatal) participants, the first dose will be given as an ultrasound-guided injection into the umbilical cord. Short term follow up will be for 1 year, with long term follow up until 10 years after 1st dose (or age 10 for prenatal participants). Participants will be matched with prospective or historical untreated OI control patients. The trial is sponsored by the Karolinska Institutet in Sweden. Within the UK, the study will be conducted at University College London Hospital and Great Ormond Street Hospital. There are 3 additional sites within Europe.
REC name
North East - York Research Ethics Committee
REC reference
19/NE/0030
Date of REC Opinion
22 Mar 2019
REC opinion
Further Information Favourable Opinion