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| Name | Class |
|---|---|
| Ministry of Science and Technology, India | OTHER_GOV |
| Vinnova | OTHER_GOV |
| Karolinska Institutet | OTHER |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Prospective Control (Untreated) and historical controls | No Intervention | Subjects eligible for the trial but not willing/able to participate in any of the experimental arms Matched historical controls. Subjects will be identified in historical registries and data will be retrieved from OI database | |
| Treatment | Experimental | Administration of four doses of BOOST cells with the first dose between 1-4 years of age and the three additional doses at +4, +8 and +12 months after the first dose. Each dose is 3x10^6 MSC/kg body weight. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| BOOST cells | Biological | Four doses of expanded human 1st trimester fetal liver-derived mesenchymal stem cells. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Safety and tolerability measured as seriousness, severity and frequency of treatment related adverse events (AEs) | The primary endpoint is safety and tolerability measured as seriousness, severity and frequency of treatment related adverse events (AEs)/Serious AE (SAE)/Suspected Unexpected Serious Adverse Reaction (SUSAR)with specific focus on the following:
| From baseline to 16 months follow up |
| Measure | Description | Time Frame |
|---|---|---|
| Number of fractures [ Time Frame: From baseline to 16 months follow-up ] | Number of fractures. | From baseline to 16 months follow up |
| Time (days) to first fracture after each stem cell administration. [ Time Frame: From each dose of stem cells to the time point of the first fracture. |
| Measure | Description | Time Frame |
|---|---|---|
| Impact on the subjects Quality of Life: Pediatric Quality of Life Questionnaire™ (PedsQOL) [ Time Frame: From baseline to the 16 month follow-up | Quality of life assessed using the Infant Pediatric Quality of Life Questionnaire™ (PedsQOL). | From baseline to 16 months follow up |
| Incidence of donor cells engrafted into patient tissue samples assessed by histology. [ Time Frame: From baseline to the 16 month follow up |
(i)Inclusion Criteria Treatment group
(ii)Inclusion Criteria Prospective Untreated Control Group and Historical Control Group:
(iii)Exclusion Criteria Treatment group Prospective and historical control group:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Vrisha Madhuri, MS Orth | Contact | 91-416-2282172 | madhuriwalter@cmcvellore.ac.in | |
| Suhasini Ganesh, M.Pharm | Contact | 91-416-2285117 | brittlebonekids@cmcvellore.ac.in |
| Name | Affiliation | Role |
|---|---|---|
| Vrisha Madhuri, MS Orth | Christian Medical College, Vellore, India | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Christian Medical College | Recruiting | Vellore | Tamil Nadu | 632004 | India |
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| ID | Term |
|---|---|
| D010013 | Osteogenesis Imperfecta |
| ID | Term |
|---|---|
| D010009 | Osteochondrodysplasias |
| D001848 | Bone Diseases, Developmental |
| D001847 | Bone Diseases |
| D009140 | Musculoskeletal Diseases |
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Historical and Untreated prospective control and Treatment group
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Time (days) to first fracture after each stem cell administration. Number of fractures |
| From baseline to 16 months follow up |
| Change in bone-marrow density (g/cm2). [ Time Frame: From baseline to the primary follow-up (From baseline to 16 months follow up) | Change in bone-marrow density (g/cm2). | From baseline to 16 months follow up |
| Growth (cm). [ Time Frame: From baseline to16 months follow up] | Growth (cm) as assessed by clinician | From baseline to 16 months follow up |
| Weight (kg). [ Time Frame: From baseline to 16 months follow up] | Growth (kg) as assessed by clinician. | From baseline to 16 months follow up |
| Change in clinical status of OI. [ Time Frame: From baseline to 16 months follow up] | Change in clinical status of OI based on parameters defined under efficacy assessments described in protocol, as assessed by OI clinician. | From baseline to 16 months follow up |
| Assessment of biochemical bone turnover by analysis of the markers P-Calcium (mg %) in blood samples. | Assessment of biochemical bone turnover marker P-Calcium (mg %) | From at baseline to 16 months follow up |
| Assessment of biochemical bone turnover by analysis of the markers P-Phosphate (mg %) in blood samples. | Assessment of biochemical bone turnover marker P-Phosphate (mg %) | From baseline to 16 months follow up |
| Assessment of biochemical bone turnover by analysis of the markers P-Albumin (g/dL) | Assessment of biochemical bone turnover marker P-Albumin (g/dL) | From baseline to 16 months follow up |
| Assessment of biochemical bone turnover by analysis of the markers S-ALP (IU/L) in blood samples. | Assessment of biochemical bone turnover marker S-ALP (IU/L) | From baseline to 16 months follow up |
| Assessment of biochemical bone turnover by analysis of the markers S-CTx (mg %) in blood samples. | Assessment of biochemical bone turnover marker S-CTx (mg %) | From baseline to 16 months follow up |
| Assessment of biochemical bone turnover by analysis of the markers fP-PTH (pg/mL)in blood samples. | Assessment of biochemical bone turnover marker fP-PTH (pg/mL) | From baseline to 16 months follow up |
| Assessment of biochemical bone turnover by analysis of the markers Vitamin D (nmol/L) in blood samples. | Assessment of biochemical bone turnover marker Vitamin D (nmol/L) | From baseline to 16 months follow up |
| Assessment of biochemical bone turnover by analysis of the markers Bone specific S-ALP (μg/L) in blood samples. | Assessment of biochemical bone turnover marker Bone specific S-ALP (μg/L) | From baseline to 16 months follow up |
| Assessment of biochemical bone turnover by analysis of the markers S-Osteocalcin (ng/mL) in blood samples. | Assessment of biochemical bone turnover marker S-Osteocalcin (ng/mL) | From baseline to 16 months follow up |
| Assessment of biochemical bone turnover by analysis of the markers U-DPD/Krea and U-NTx/Krea (mg %) in blood samples. | Assessment of biochemical bone turnover marker U-DPD/Krea and U-NTx/Krea (mg %) | From baseline to 16 months follow up |
Donor cell engraftment. |
| From baseline to 16 months follow up |
| Analysis of an array of cytokines and micro vesicles to evaluate paracrine effects. [ Time Frame: From baseline to the 16 month follow up | Paracrine effects will be analysed from plasma isolated from peripheral blood. | From baseline to 16 months follow up |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D003095 | Collagen Diseases |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |