Phase 2a Study on Intravenous Infusion of Autologous Osteoblastic Cells in Severe Osteoporosis

Study Overview
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Brief Summary
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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.

Official Title
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Treatment of Severe Osteoporosis by Intravenous Infusion of Pre-osteoblastic Cells - An Open Pilot Study

Conditions
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Severe Osteoporosis

Intervention / Treatment
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* Drug: PREOB® Intravenous Infusion


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosis of severe osteoporosis Non-response to anti-osteoporotic treatment for over 2 years Normal hematology function Ability to provide a written, dated, and signed informed consent prior to any study related procedure, and to understand and comply with study requirements Exclusion Criteria: Positive serology for hepatitis B, hepatitis C, HIV, syphilis, HTLV-1 Current or past medical disease that could interfere with the evaluation of the efficacy, as judged by the investigator Severe renal or hepatic impairment

Ages Eligible for Study
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Minimum Age: 40 Years
Maximum Age: 85 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: PREOB® Intravenous Infusion<br> | Drug: PREOB® Intravenous Infusion<br>* Each patient will undergo a single intravenous administration of PREOB®.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Cell biodistribution after intravenous infusion as assessed by SPECT/CT scan |  | 72 hours | 
| Potential occurrence of any AE or SAE, related to the product, using patient open non-directive questionnaire, physical examination and laboratory measurements |  | 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Pain measured by the Visual Analogue Scale |  | 12 months | 
| Well-being score using SF-36 questionnaire |  | 12 months | 
| Occurrence of new vertebral fractures as assessed by X-ray |  | 12 months | 
| Bone mineral density evolution as assessed by Dual-energy X-ray absorptiometry (DEXA) |  | 12 months | 
| Measurement of bone formation and resorption markers |  | 12 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Bone Diseases, Metabolic Bone Diseases, Osteoporosis, Bone Fractures, Osteoporotic Fractures, Musculoskeletal Diseases