Mesenchymal Stem Cell Based Therapy for the Treatment of Osteogenesis Imperfecta

EudraCT Number:2012-002553-38

The purpose of this study is to determine the safety and effectiveness of five infusions of characterized HLA-identical MSC in non immunosuppressed children with Osteogenesis Imperfecta (OI).

2014-04-01

2018-12-01

2018-12-01

Completed

Early phase I

2

Inclusion Criteria: * Patient age: older than 6 months and younger than 12 years old. * Patients with molecular confirmation of mutation in either COL1A1 or COL1A2 genes associated with OI (type III). * Patients with HLA identical (that shared at least 5/6 antigens) siblings willing to donate bone marrow-MSCs. * All patients that fulfil the inclusion criteria regardless of whether or not they are undergoing biphosphonate treatment. * Patients whose parents or the legal guardians are willing to sign the consent forms to participate in this clinical trial. Exclusion Criteria: * Patient age: older than 12 years old * Patients lacking confirmation of mutation in either COL1A1 or COL1A2 genes associated with severe deforming OI (type III). * Other pathological subtypes of OI. * Patients lacking of HLA identical (that shared at least 5/6 antigens) siblings willing to donate bone marrow-MSCs. * Immunodeficiencies and any other malignancies. * Participation in other clinical trial. * Any medical or psychiatric condition that in the researcher´s opinion could affect the patient´s ability to complete the trial or hamper the participation in the trial. * Patients whose parents or the legal guardians do not sign the consent forms

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 2, 'type': 'ACTUAL'}}

2023-10-03