Boost Brittle Bones Before Birth (NCT03706482) | Clinical Trial Compass
Active — Not RecruitingPhase 1/2
Boost Brittle Bones Before Birth
Sweden18 participantsStarted 2019-08-12
Plain-language 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.
Who can participate
Age range18 Months
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
✓. Parent's/legal guardian's signed informed-consent form
✓. Clinical diagnosis of OI type III or severe type IV AND
✓. Molecular diagnosis of OI (Glycine substitution in the collagen triple-helix encoding region of either the COL1A1 or COL1A2 gene)
✓. Age less than 18 months (calculated from gestational week 40+0, i.e. the corrected age)
✓. Parent/legal guardian over 18 years of age
✓. Woman has signed the informed-consent form
✓. Only women where termination of the pregnancy is no longer possible or where the women are committed to continue the pregnancy may be included in the trial
✓. Suspicion of OI type III or severe type IV in the fetus on ultrasound findings AND
Exclusion criteria
✕. Existence of other known disorder that might interfere with the treatment, such as, but not limited to organ dysfunction (for example liver or renal failure or bronchopulmonary dysplasia), congenital heart defect, hypoxic encephalopathy l-lll, severe neurological problems, immune deficiencies, muscle diseases, severe malformations or syndromes diagnosed by clinical examination.
✕. Any contraindication for invasive procedures such as a moderate/severe bleeding tendency
✕. Known risk factors for clotting, such as, but not limited to previous blood clot, family history of clots, clotting disorder (inherited or acquired), heart failure, inflammatory disorders (for example lupus, rheumatoid arthritis, inflammatory bowel disease)
What they're measuring
1
Safety and tolerability measured as seriousness, severity and frequency of treatment related adverse events.
Timeframe: From baseline to the long-time follow-up (10 years after the first dose).