Boost to Brittle Bones - Stem Cell Transplantation for Treatment of Brittle Bones
- Conditions
- Osteogenesis Imperfecta
- Interventions
- Biological: BOOST cells
- Registration Number
- NCT04623606
- Lead Sponsor
- Christian Medical College, Vellore, India
- Brief 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- UNKNOWN
- Sex
- All
- Target Recruitment
- 15
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Treatment BOOST cells 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.
- Primary Outcome Measures
Name Time Method Safety and tolerability measured as seriousness, severity and frequency of treatment related adverse events (AEs) From baseline to 16 months follow up 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:
1. Vital signs in conjunction with the MSC infusion
2. Transfusion reactions (infusion toxicity, embolism, allergy, infections)
3. Immune reaction towards the cells, donor-specific antibodies, graft rejection, Graft versus Host Disease, autoimmunity)
4. Tumourigenicity
5. Mortality/morbidity
- Secondary Outcome Measures
Name Time Method Assessment of biochemical bone turnover by analysis of the markers fP-PTH (pg/mL)in blood samples. From baseline to 16 months follow up Assessment of biochemical bone turnover marker fP-PTH (pg/mL)
Weight (kg). [ Time Frame: From baseline to 16 months follow up] From baseline to 16 months follow up Growth (kg) as assessed by clinician.
Number of fractures [ Time Frame: From baseline to 16 months follow-up ] From baseline to 16 months follow up Number of fractures.
Growth (cm). [ Time Frame: From baseline to16 months follow up] From baseline to 16 months follow up Growth (cm) as assessed by clinician
Change in clinical status of OI. [ Time Frame: From baseline to 16 months follow up] 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.
Assessment of biochemical bone turnover by analysis of the markers P-Calcium (mg %) in blood samples. From at baseline to 16 months follow up Assessment of biochemical bone turnover marker P-Calcium (mg %)
Assessment of biochemical bone turnover by analysis of the markers S-ALP (IU/L) in blood samples. From baseline to 16 months follow up Assessment of biochemical bone turnover marker S-ALP (IU/L)
Assessment of biochemical bone turnover by analysis of the markers S-CTx (mg %) in blood samples. From baseline to 16 months follow up Assessment of biochemical bone turnover marker S-CTx (mg %)
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. From baseline to 16 months follow up Time (days) to first fracture after each stem cell administration. Number of fractures
Assessment of biochemical bone turnover by analysis of the markers P-Phosphate (mg %) in blood samples. From baseline to 16 months follow up Assessment of biochemical bone turnover marker P-Phosphate (mg %)
Assessment of biochemical bone turnover by analysis of the markers Bone specific S-ALP (μg/L) in blood samples. From baseline to 16 months follow up Assessment of biochemical bone turnover marker Bone specific S-ALP (μg/L)
Assessment of biochemical bone turnover by analysis of the markers S-Osteocalcin (ng/mL) in blood samples. From baseline to 16 months follow up Assessment of biochemical bone turnover marker S-Osteocalcin (ng/mL)
Assessment of biochemical bone turnover by analysis of the markers U-DPD/Krea and U-NTx/Krea (mg %) in blood samples. From baseline to 16 months follow up Assessment of biochemical bone turnover marker U-DPD/Krea and U-NTx/Krea (mg %)
Change in bone-marrow density (g/cm2). [ Time Frame: From baseline to the primary follow-up (From baseline to 16 months follow up) From baseline to 16 months follow up Change in bone-marrow density (g/cm2).
Assessment of biochemical bone turnover by analysis of the markers P-Albumin (g/dL) From baseline to 16 months follow up Assessment of biochemical bone turnover marker P-Albumin (g/dL)
Assessment of biochemical bone turnover by analysis of the markers Vitamin D (nmol/L) in blood samples. From baseline to 16 months follow up Assessment of biochemical bone turnover marker Vitamin D (nmol/L)
Trial Locations
- Locations (1)
Christian Medical College
🇮🇳Vellore, Tamil Nadu, India