Title

Assessing the Feasibility of BGC101 in the Treatment of PAD & CLI
Phase 1/2, Open Label & Double Blind Randomized Placebo Controlled Study to Assess the Safety and Efficacy of BGC101 (EnEPC) in the Treatment of PAD With CLI
  • Phase

    Phase 1/Phase 2
  • Study Type

    Interventional
  • Status

    Recruiting
  • Study Participants

    50
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.
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 Started
Jun 30
2016
Primary Completion
Dec 31
2023
Anticipated
Study Completion
Dec 31
2023
Anticipated
Last Update
Nov 08
2022
Estimate

Biological BGC101 (autologous EnEPC preparation)

Intramuscular injections - single treatment session

Biological Control medium

Intramuscular injections - single treatment session

Placebo Placebo Comparator

Intramuscular injection of control medium only

BGC101 Experimental

Intramuscular injection of BGC101 (autologous EnEPC preparation)

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)
Pregnant or lactating women at entry to study
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)
Malignancy within the preceding 3 years, except for BCC
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
No Results Posted