Stem cell transplantation for treatment of weak bones
- Conditions
- Health Condition 1: M859- Disorder of bone density and structure, unspecifiedHealth Condition 2: null- Osteogenesis Imperfecta
- Registration Number
- CTRI/2019/07/020217
- Lead Sponsor
- Department of Biotechnology
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Open to Recruitment
- Sex
- Not specified
- Target Recruitment
- 0
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
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
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method The primary endpoints are seriousness, severity and frequency of treatment related adverse events (AEs), with specific focus on the following: <br/ ><br>Vital signs in conjunction with the MSC infusion <br/ ><br>Transfusion reactions (infusion toxicity, embolism, allergy, infections) <br/ ><br>Immunogenicity (reaction towards the cells, donor specific antibodies, graft rejection, Graft versus Host Disease, autoimmunity) <br/ ><br>Tumourigenicity <br/ ><br>Mortality/morbidity <br/ ><br>Timepoint: 4 Months up to 16 months
- Secondary Outcome Measures
Name Time Method Fracture frequency <br/ ><br>Time (days) to first fracture after each MSC infusion <br/ ><br>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 <br/ ><br>Change in clinical status of OI based on parameters defined under efficacy assessments described below during 16 months <br/ ><br>Change in biochemical bone turnover during 16 monthsTimepoint: 16 months