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

Primary Purpose

Severe Osteoporosis

Status

Completed

Phase

Phase 2

Locations

Belgium

Study Type

Interventional

Intervention

PREOB® Intravenous Infusion

About this trial

This is an interventional treatment trial for Severe Osteoporosis focused on measuring Bone Diseases, Metabolic Bone Diseases, Osteoporosis, Bone Fractures, Osteoporotic Fractures, Musculoskeletal Diseases

Eligibility Criteria

40 Years - 85 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Investigating site BE01

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

PREOB® Intravenous Infusion

Arm Description

Outcomes

Primary Outcome Measures

Cell biodistribution after intravenous infusion as assessed by SPECT/CT scan

Potential occurrence of any AE or SAE, related to the product, using patient open non-directive questionnaire, physical examination and laboratory measurements

Secondary Outcome Measures

Pain measured by the Visual Analogue Scale

Well-being score using SF-36 questionnaire

Occurrence of new vertebral fractures as assessed by X-ray

Bone mineral density evolution as assessed by Dual-energy X-ray absorptiometry (DEXA)

Measurement of bone formation and resorption markers

Full Information

NCT ID

NCT02061995

First Posted

February 11, 2014

Last Updated

August 22, 2019

Sponsor

Erasme University Hospital

1. Study Identification

Unique Protocol Identification Number

NCT02061995

Brief Title

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

2. Study Status

Record Verification Date

February 2014

Overall Recruitment Status

Completed

Study Start Date

December 2013 (Actual)

Primary Completion Date

October 2016 (Actual)

Study Completion Date

October 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Erasme University Hospital

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Severe Osteoporosis

Keywords

Bone Diseases, Metabolic Bone Diseases, Osteoporosis, Bone Fractures, Osteoporotic Fractures, Musculoskeletal Diseases

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

20 (Actual)

8. Arms, Groups, and Interventions

Arm Title

PREOB® Intravenous Infusion

Arm Type

Experimental

Intervention Type

Drug

Intervention Name(s)

PREOB® Intravenous Infusion

Intervention Description

Each patient will undergo a single intravenous administration of PREOB®.

Primary Outcome Measure Information:

Title

Cell biodistribution after intravenous infusion as assessed by SPECT/CT scan

Time Frame

72 hours

Title

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

Secondary Outcome Measure Information:

Title

Pain measured by the Visual Analogue Scale

Time Frame

12 months

Title

Well-being score using SF-36 questionnaire

Time Frame

12 months

Title

Occurrence of new vertebral fractures as assessed by X-ray

Time Frame

12 months

Title

Bone mineral density evolution as assessed by Dual-energy X-ray absorptiometry (DEXA)

Time Frame

12 months

Title

Measurement of bone formation and resorption markers

Time Frame

12 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

40 Years

Maximum Age & Unit of Time

85 Years

Accepts Healthy Volunteers

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

Facility Information:

Facility Name

Investigating site BE01

City

Anderlecht

Country

Belgium

12. IPD Sharing Statement

Learn more about this trial

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

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