Boost to Brittle Bones - Stem Cell Transplantation for Treatment of Brittle Bones (NCT04623606) | Clinical Trial Compass
UnknownPhase 1/2
Boost to Brittle Bones - Stem Cell Transplantation for Treatment of Brittle Bones
India15 participantsStarted 2019-05-20
Plain-language summary
An exploratory, open label, multiple dose, phase I/II trial (n=15) evaluating safety and efficacy of intravenous and intraosseous infusion of allogeneic expanded fetal mesenchymal stem cells (MSC) for the treatment of severe Osteogenesis Imperfecta (OI) compared with historical and untreated prospective controls.
Who can participate
Age range1 Year – 8 Years
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 IV AND
✓. Molecular diagnosis of OI (Glycine substitution in the collagen triple-helix encoding region of either the COL1A1 or COL1A2 gene)
✓. Age between 1 to 4 years
✓. BP treatment initiated before inclusion
✓. Parent/legal guardian over 18 years of age
✓. Parent's/legal guardian's signed informed-consent form
✓. Clinical and molecular diagnosis of OI (Glycine substitution in the collagen triple-helix encoding region of either the COL1A1 or COL1A2 gene)
Exclusion criteria
✕. Existence of other known disorder that might interfere with the treatment (such as severe malformations, congenital heart defect, hypoxic encephalopathy (l-lll), neurological problems, immune deficiencies, muscle diseases, syndromes) diagnosed by clinical examination
✕. Any contraindication for invasive procedures such as a moderate/severe bleeding tendency or contagious infections
✕. Abnormal karyotype or other confirmed genetic syndromes
✕. Oncologic disease
✕
What they're measuring
1
Safety and tolerability measured as seriousness, severity and frequency of treatment related adverse events (AEs)