Who can participate

Clinical Personnel Pages

In order to take part in the trial we will need to carry out a DNA test to confirm that your patient, or their unborn baby has severe OI (type 3 or type 4). This can be carried out prenatally on a sample of amniotic fluid or placenta, or from a blood test postnatally.

 

We will need to make sure that your patient meets all the inclusion criteria for the trial, and none of the exclusion criteria.

 

There are three different groups in the trial. One group of babies will have the first dose of stem cells before birth and 3 doses after birth. The second group will have all 4 doses of stem cells after birth, but before 12 months of age. Finally, there will be a control group of babies (historical and prospective) who do not receive any stem cells.  

 

Your patient can choose which group they would like their child to be in. 

 

Taking part in the trial will mean your patient spends more time travelling to and attending hospital appointments than they would otherwise. The dose of stem cells before birth is given via an injection into the umbilical cord. The doses after birth are given via a drip into a vein. We aim to follow up the treated children frequently until they are two, and then once a year until ten years after the first dose. The follow-ups will be done during the routine OI clinic visits.

 

Your patient may be eligible to take part in the trial if they fit into one of the three groups below.

 

    1. Postnatal Group:

The child may have brittle bone disease (OI). If so the parents should be asked if they would like their child to take part in the BOOSTB4 (Boost Brittle Bones Before Birth) trial because their child may have OI type 3 or severe type 4.

 

     2. Prenatal Group:

The parents have recently been informed that their unborn child may have brittle bone disease (OI). If so they should be asked if they would like to take part in the BOOSTB4 trial because their unborn child may be suffering from OI type 3 or severe type 4.

 

    3. Historical and prospective Control Group:

With their agreement, we may like to collect information about the mother and her child as part of a historical and prospective control group within the trial. They may be asked if we can collect information about them as a part of the BOOSTB4 trial because the child has OI type 3 or severe type 4.

What are the risks and benefits to taking part in the trial?

No treatment is risk free. Potential risks related to the stem cell dose fall into two groups.

  • The way the cells are given:

The risks of the prenatal injection include miscarriage, premature labour, infection or a drop in the baby’s heart rate. 

The risks of the postnatal injection include bruising, infection and pain.

  • The stem cells themselves:

No adverse effects have been observed due to the stem cells themselves in the <10 babies treated to date. However, the following risks are possible: allergic or immune reaction, development of cancer, other diseases (we screen for bacteria, mycoplasma and viruses to make sure the cells are safe to use).

 

Our goal is that your patient, or their child will be helped by having the stem cell doses; by having fewer fractures, less chronic pain or that their height may be improved. None of these benefits can be guaranteed and it is important to remember that your patient, or their child will still be affected by Osteogenesis Imperfecta. Further information about risks and benefit will be provided if your patient is eligible to participate in the trial.

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This project has received funding from the European Union's Horizon 2020 research and innovation programme under grant agreement 681045

© 2019 BOOSTB4 Consortium