Allogeneic fetal mesenchymal stem cells for Osteogenesis imperfecta (type III) | Osteogenesis imperfecta (type IV, severe)

Inclusion criteria

• {"criterion_text":"- Parent's/legal guardian's signed informed-consent form.\n- Clinical diagnosis of OI type III or IV AND\n- Molecular diagnosis of OI (Glycine substitution in the collagen triplehelix encoding region of either the COL1A1 or COL1A2 gene)\n- Age less than 18 months (calculated from gestational week 40+0, i.e. the corrected age)\n- Parent/legal guardian over 18 years of age"}

Exclusion criteria

• {"criterion_text":"- 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\n- Any contraindication for invasive procedures such as a moderate/severe bleeding tendency\n- 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)\n- Positive Donor Specific Antibody-test\n- Known allergy/hypersensitivity to Fungizone and/or Gensumycin\n- Abnormal karyotype or other confirmed genetic syndromes\n- Oncologic disease (previous or current malignancy)\n- Inability to comply with the trial protocol and follow-up schedule\n- Inability to understand the information and to provide informed consent"}

Primary endpoints

• {"endpoint_text":"- The primary endpoints are seriousness, severity and frequency of treatment-related AEs.)","definition_or_measurement_approach":"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."}

Secondary endpoints

• {"endpoint_text":"- Number of fractures from baseline to primary and long-time follow-up (key secondary endpoint)","definition_or_measurement_approach":"Key secondary endpoint; measured as number of fractures from baseline to primary and long-term follow-up."}
• {"endpoint_text":"- Time (days) to first fracture after last dose","definition_or_measurement_approach":"Measured in days from last dose to first fracture."}
• {"endpoint_text":"- Number of fractures at birth (prenatal treatment group, and postnatal treatment group when available)","definition_or_measurement_approach":"Count of fractures at birth for prenatal group (and postnatal group when available)."}
• {"endpoint_text":"- Change in Bone mineral density (g/cm2) (supportive for the endpoint fracture frequency)","definition_or_measurement_approach":"Change in BMD (g/cm2); supportive endpoint for fracture frequency."}
• {"endpoint_text":"- Growth (cm and kg)","definition_or_measurement_approach":"Measured change in length/height (cm) and weight (kg)."}
• {"endpoint_text":"- Change in clinical status of OI based on parameters defined under efficacy assessments","definition_or_measurement_approach":"Change in clinical status based on predefined efficacy assessment parameters (not further specified in available data)."}
• {"endpoint_text":"- Change in biochemical bone turnover","definition_or_measurement_approach":"Change in biochemical markers of bone turnover (specific markers not detailed in available data)."}

Sweden

Earliest CTIS Part Ii Submission Date

18-08-2024

Latest Decision Or Authorization Date

29-08-2024

Processing Time Days

11

Number Of Sites

1

Number Of Participants

130

Netherlands

Earliest CTIS Part Ii Submission Date

18-08-2024

Latest Decision Or Authorization Date

02-09-2024

Processing Time Days

15

Number Of Sites

2

Number Of Participants

8

Primary sponsor

Full Name

Karolinska Institutet

Organisation Type

Educational Institution

Country Of Registered Address

Sweden