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 range
18 Months
Sex
ALL
See this in plain English?
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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
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).
. 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)
. Positive Donor Specific Antibody-test
. Known allergy/hypersensitivity to Fungizone and/or Gensumycin
. Abnormal karyotype or other confirmed genetic syndromes
. Oncologic disease (previous or current malignancy)
. Inability to comply with the trial protocol and follow-up schedule