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Treatment of severe congenital Brittle bone disease after or before and after birth.
EU CTIS ID: 2023-504593-38-00
What this study is testing
The primary objective is to assess safety and tolerability in the child, fetus and woman after postnatal or prenatal and postnatal intravenous administration of four doses of BOOST cells in individuals with OI type III or severe type IV.
- Phase I and Phase II (Integrated)- Other
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Parent's/legal guardian's signed informed-consent form.
- Clinical diagnosis of OI type III or IV AND
- Molecular diagnosis of OI (Glycine substitution in the collagen triplehelix 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
You likely can't join if
- Existence of other known disorder that might interfere with the treatment, such as, but not limited to organ dysfunction (for e.g. 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 e.g. 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
See the full eligibility criteria
- Parent's/legal guardian's signed informed-consent form.
- Clinical diagnosis of OI type III or IV AND
- Molecular diagnosis of OI (Glycine substitution in the collagen triplehelix 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
- Existence of other known disorder that might interfere with the treatment, such as, but not limited to organ dysfunction (for e.g. 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 e.g. 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
- Inability to understand the information and to provide informed consent
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 0-17 years, in utero. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.