Clinical trial NCT02061995: Phase 2a Study on Intravenous Infusion of Autologous Osteoblastic Cells in Severe Osteoporosis
Official title: Treatment of Severe Osteoporosis by Intravenous Infusion of Pre-osteoblastic Cells - An Open Pilot Study
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Severe Osteoporosis
Keywords: Bone Diseases
Metabolic Bone Diseases
Osteoporosis
Bone Fractures
Osteoporotic Fractures
Musculoskeletal Diseases
Lead sponsor: Erasme University Hospital
Sponsor class: OTHER
First posted: 2014-02-13
Enrollment: 20
Sex: ALL
Minimum age (years): 40
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: 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

Primary outcomes:
measure: Cell biodistribution after intravenous infusion as assessed by SPECT/CT scan
timeFrame: 72 hours
measure: Potential occurrence of any AE or SAE, related to the product, using patient open non-directive questionnaire, physical examination and laboratory measurements
timeFrame: 12 months
Secondary outcomes:
measure: Pain measured by the Visual Analogue Scale
timeFrame: 12 months
measure: Well-being score using SF-36 questionnaire
timeFrame: 12 months
measure: Occurrence of new vertebral fractures as assessed by X-ray
timeFrame: 12 months
measure: Bone mineral density evolution as assessed by Dual-energy X-ray absorptiometry (DEXA)
timeFrame: 12 months
measure: Measurement of bone formation and resorption markers
timeFrame: 12 months
Other outcomes:
Locations:
city: Anderlecht
country: Belgium
facility: Investigating site BE01
Study officials:
References: