Study Plan

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The BOOST2B project is designed to facilitate the conduct of a clinical trial of human fetal MSC for the treatment of OI that will provide data to support the future clinical development and commercialisation of this therapy. The overall ambition of the proposal is to develop and analyse a new treatment of severe OI, a disorder where no effective therapies are available.

Inclusion criteria

A.        Children diagnosed with OI type III or severe OI type IV

1.         Clinical and molecular diagnosis of OI in the child (Glycine substitution in the collagen triple-helix encoding region of either the COL1A1 or COL1A2 gene)

1.         Patient between 1-4 year of age

2.         Parent/authorized representative over 18 years of age

3.         Ability of the parent/authorized representative to give informed consent

B.        Historical controls with OI type III or severe OI type IV

1.         Clinical and molecular diagnosis of OI in the child (Glycine substitution in the collagen triple-helix encoding region of either the COL1A1 or COL1A2 gene)

1.         Current age of the patient less than 10 years

2.         Parent/authorized representative over 18 years of age

3.         Ability of the parent/authorized representative to give informed consent

Exclusion criteria

A.        Children diagnosed with OI type III or severe OI type IV

1.         Signs of fever or infections 24 hours before infusion

2.         Existence of other disorder that might interfere with the treatment (such as severe malformations, hypoxic encephalopathy (l-lll), neurological problems, immune deficiencies muscle diseases, syndromes

3.         Abnormal karyotype

4.         Any psychological, familial, sociological and/or geographical condition potentially hampering compliance with the study protocol and follow-up schedule

B.        Historical/concurrent controls with OI type III or severe OI type IV

1.         Existence of other disorder that might interfere with the study

2.         Abnormal karyotype

Recruited patients will receive the first treatment of fetal liver MSC between 1 year to 4 years of age, and then be treated and evaluated at 4, 8, 12 (treatments) and 16 months (follow-up) after inclusion and first treatment. A 10 years long-term follow-up will be performed in an extension to the study. Controls with OI type III or severe type IV will not undergo any clinical procedures in the study.

The clinical study will be carried out with an Advanced Therapy Medicinal Product (ATMP) product manufactured according to Good Manufacturing Practices (GMP)-standards at the accredited GMP-facility Vecura at Karolinska University Hospital in Sweden. The cryopreserved drug product (DP) will be certified and released after fulfilment of a defined set of criteria. Ready-to-be-used DPs will be shipped to CMC in Vellore, India for administration