Phase 2a Study on Intravenous Infusion of Autologous Osteoblastic Cells in Severe Osteoporosis
Osteoporosis is a systemic skeletal disease characterized by low bone mass and microarchitectural deterioration of bone tissue, with a subsequent increase in bone fragility and susceptibility to fracture.
Osteoporosis is one of the most common and debilitating chronic diseases, and a global health concern with a high prevalence not only in Western countries, but also in Asia and Latin America. Most efficacious anti-osteoporotic treatments either inhibit bone resorption like bisphosphonates or denosumab or increase bone formation like teripartide an anabolic agent. Anti-osteoporotic drugs have demonstrated safety and efficacy with an increase in bone mass and a decrease of fracture risk (at the hip) by 30 to 50% after 3 years of treatment (Black et al., 1996; Neer et al., 2001; Meunier et al., 2004). Despite the availability of pharmacological treatments, osteoporosis remains a significant health problem for patients who do not respond to the available treatments or fail to comply with their regimens.
The present phase 2a study aims to demonstrate the safety and efficacy of PREOB®, a proprietary population of autologous osteoblastic cells, in the treatment of osteoporotic patients who do not respond to pharmacological treatments.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Treatment of Severe Osteoporosis by Intravenous Infusion of Pre-osteoblastic Cells - An Open Pilot Study|
- Cell biodistribution after intravenous infusion as assessed by SPECT/CT scan [ Time Frame: 72 hours ] [ Designated as safety issue: No ]
- Potential occurrence of any AE or SAE, related to the product, using patient open non-directive questionnaire, physical examination and laboratory measurements [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
- Pain measured by the Visual Analogue Scale [ Time Frame: 12 months ] [ Designated as safety issue: No ]
- Well-being score using SF-36 questionnaire [ Time Frame: 12 months ] [ Designated as safety issue: No ]
- Occurrence of new vertebral fractures as assessed by X-ray [ Time Frame: 12 months ] [ Designated as safety issue: No ]
- Bone mineral density evolution as assessed by Dual-energy X-ray absorptiometry (DEXA) [ Time Frame: 12 months ] [ Designated as safety issue: No ]
- Measurement of bone formation and resorption markers [ Time Frame: 12 months ] [ Designated as safety issue: No ]
|Study Start Date:||December 2013|
|Estimated Study Completion Date:||October 2016|
|Estimated Primary Completion Date:||October 2016 (Final data collection date for primary outcome measure)|
|Experimental: PREOB® Intravenous Infusion||
Drug: PREOB® Intravenous Infusion
Each patient will undergo a single intravenous administration of PREOB®.
Please refer to this study by its ClinicalTrials.gov identifier: NCT02061995
|Contact: Valérie Gangji, MD, PhDemail@example.com|
|Investigating site BE01||Recruiting|