EnEPC-CLI: Assessing the Feasibility of BGC101 in the Treatment of PAD & CLI
Study Details
Study Description
Brief Summary
Evaluate the feasibility of an autologous cell preparation composed of a mixture of cells enriched for endothelial progenitor cells (EnEPCs) and multipotent adult hematopoietic stem/progenitor cells (HSPC) (BGC101), in the treatment of patients suffering from peripheral arterial disease (PAD) with critical limb ischemia (CLI) who have not responded to optimal pharmacological treatment or control of risk factors and/or had a revascularization failure, and do not have the option of further revascularization treatment.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 1/Phase 2 |
Detailed Description
BGC101 is designed to treat peripheral vascular disease in patients suffering from Critical Leg Ischemia (CLI) also referred to as chronic limb threatening ischemia (CLTI).
This part of the study is designed as a placebo double-blind randomized controlled trial (CRT) assessing the safety and efficacy of BGC101 in 45 eligible subjects in 2 Arms: Arm A: BGC101 treatment and Arm B: Placebo treatment. The Arm A:Arm B ratio is 2:1 A single dose treatment of the personalized cells by intramuscular injections into the affected leg takes less than 10 minutes.
Cells from a standard blood draw (with no pre-treatment, bone marrow aspiration, mobilization or apheresis) are transformed, within a day, into the investigational medicinal product BGC101.
BGC101, intended for autologous use, is a 'ready-to-use' cell suspension in prefilled syringes.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Placebo Comparator: Placebo Intramuscular injection of control medium only |
Biological: Control medium
Intramuscular injections - single treatment session
|
| Experimental: BGC101 Intramuscular injection of BGC101 (autologous EnEPC preparation) |
Biological: BGC101 (autologous EnEPC preparation)
Intramuscular injections - single treatment session
|
Outcome Measures
Primary Outcome Measures
- Safety (Evaluation of adverse events - lack of severe adverse events) [Six months]
Evaluation of adverse events - lack of severe adverse events
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Have the time and ability to complete the study and comply with instructions.
-
Capable of understanding the purpose of the study and the contents of the informed consent form
-
Age > 18 Male or non-pregnant, non-lactating female
-
At least one of the clinical diagnostic indications of CLI:
-
Clinical assessment as Rutherford 4-5
-
Rest pain
-
Non-healing ischemic ulcers
-
Minor tissue loss
- At least one of the hemodynamic indicators of severe peripheral arterial occlusive disease:
-
Ankle brachial index (ABI) <0.45
-
Toe brachial index (TBI) <0.4
-
TcPO2 < 40mmHg
-
A poor candidate for standard revascularization treatment options for peripheral arterial disease due to (1) unfavorable anatomy (e.g. small vessel disease with no major vessel stenosis/obstruction) OR (2) continued presence of smaller vessel microvasculature) disease six weeks or more after revascularization (performed as part of standard care) based on patency of the treated vessel(s).
Exclusion Criteria:
Patients who meet any of the following criteria are not eligible for this study.
-
Concurrent therapy that, in the Investigator's opinion, would interfere with the evaluation of the safety or efficacy of the study medication.
-
Treatment with any investigational product within the last 6 months or enrollment in any active study involving the use of investigational devices or drugs.
-
Presence of any other condition or circumstance that, in the judgment of the investigator, might increase the risk to the patient or decrease the chance of obtaining satisfactory data to achieve the objectives of the study.
-
Based on clinician opinion - prognosis of a major amputation (below or above the knee) within 4 weeks from the assessment visit
-
Lack of femoral artery blood flow
-
Based on clinician opinion inability to perform intramuscular injections in cases such as sever skin lesions, severe edema or morbid obesity
-
Blood transfusions during preceding 4 weeks (to exclude the potential of non-autologous cells in the harvested blood)
-
Heart failure (NYHA 3-4)
-
Hgb Less than 9gm
-
Myocardial infarction, brain infarction, uncontrolled myocardial ischemia or persistent severe heart failure (EF< 25 %) during the preceding 3 months
-
Significant valvular disease or after valve replacement (based on medical record)
-
Renal failure (eGFR <30, Chronic Kidney Damage Stage 4-5)
-
Liver function tests are more than three times normal upper limit (AST, ALT, ALP, GGT, LDH).
-
Abnormal coagulation tests (PT(INR) >2)
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Pregnant or lactating women at entry to study
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People who are unwilling to agree to use acceptable methods of contraception such as condom from screening during the study to prevent pregnancy and chronic infectious diseases (such as HIV-1,HIV-2, HBV, HCV)
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Malignancy within the preceding 3 years, except for BCC
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Concurrent acute infectious disease with septicemia
-
Chronic infectious diseases (HIV-1,HIV-2, Hepatitis viruses B and C)
-
Immunodeficiency syndrome
-
Cytotoxic drugs treatment
-
Inability to communicate (that may interfere with the clinical evaluation of the patient)
-
Patient unlikely to be available for follow-up
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | University of San Francisco | San Francisco | California | United States | 94143 |
| 2 | Laniado Hospital | Netanya | Israel | 42150 |
Sponsors and Collaborators
- BioGenCell Ltd.
- Laniado Hospital
- Rabin Medical Center
Investigators
- Principal Investigator: Shlomo J Baytner, MD, Director of Vascular Surgery, Laniado Hospital, IL
- Principal Investigator: Michael Conte, MD, University of San Francisco - Division Vascular and Endovascular surgery
Study Documents (Full-Text)
None provided.More Information
Publications
- BioGenCell Ltd