Health Condition 1: M859- Disorder of bone density and structure, unspecified Health Condition 2: null- Osteogenesis Imperfecta
Conditions
Interventions
Intervention1: Injection of fetal liver derived mesenchymal stem cells: Intravenous dose - Approximately 3 million viable cells per kg body weight
Intraosseous dose- Approximately 0.1 million viable MSC per kg per long bone (3.2 million viable MSC for 4 lower limbs)
Duration- Four infusions in total administered every 4 months during a 12-month
Sponsors
Department of Biotechnology
Eligibility
Inclusion criteria
Inclusion criteria: Children diagnosed with OI type III or severe OI type IV 1. Clinical and molecular diagnosis of OI in the child (Glycine substitution in the collagen triple-helix encoding region of either the COL1A1 or COL1A2 gene) 1. Patient between 1-4 year of age 2. Bisphosphonate (BP) treatment initiated before inclusion 3. Parent/authorized representative over 18 years of age 4. Ability of the parent/authorized representative to give informed consent
Exclusion criteria
Exclusion criteria: 1. Signs of fever or infections 24 hours before infusion 2. Existence of other disorder that might interfere with the treatment (such as severe malformations, hypoxic encephalopathy (l-lll), neurological problems, immune deficiencies muscle diseases, syndromes 3. Abnormal karyotype 4. Any psychological, familial, sociological and/or geographical condition potentially hampering compliance with the study protocol and follow-up schedule
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary endpoints are seriousness, severity and frequency of treatment related adverse events (AEs), with specific focus on the following: Vital signs in conjunction with the MSC infusion Transfusion reactions (infusion toxicity, embolism, allergy, infections) Immunogenicity (reaction towards the cells, donor specific antibodies, graft rejection, Graft versus Host Disease, autoimmunity) Tumourigenicity Mortality/morbidity Timepoint: 4 Months up to 16 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Fracture frequency Time (days) to first fracture after each MSC infusion Change in BMD during 16 months after the first MSC infusion Change in growth (cm and kg) during 16 months after the first MSC infusion Change in clinical status of OI based on parameters defined under efficacy assessments described below during 16 months Change in biochemical bone turnover during 16 monthsTimepoint: 16 months | — |
Countries
India
Contacts
Public ContactVrisha Madhuri
Christian Medical College
Outcome results
None listed