BGC101 (EnEPC) Autologous Cell Therapy From Patient's Own Blood for Treatment of Critical Limb Ischemia (CLI)

NCT02805023 | Active Not Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: BioGenCell Ltd
• Study Type: interventional
• Target: 40
• Locations: 2 countries
• Sites: 5

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.

Conditions

Chronic Limb-Threatening Ischemia; Peripheral Arterial Disease; Peripheral Vascular Disease

Outcome Measures

Primary Outcomes (2)

• Safety (Incidence of adverse events)

  * Incidence and proportion of incidence between treatment arms of adverse events of specific interest (AESI) and injection-related AE * Incidence of serious adverse events (SAEs) including SAEs related or probably related to the treatment * Vital signs, physical examination, and electrocardiogram (ECG) * Safety laboratory values of hematology, blood chemistry, and urinalysis * Local tolerability (injection site reaction)

  12 Months

• Efficacy (Improvement of indication signs)

  * Major amputation (below or above the knee) rate at Month 12 * Major amputation-free survival (AFS) rate at Month 12

  12 Months

Study Arms (2)

Placebo

PLACEBO COMPARATOR

Intramuscular injection of control medium only

Biological: Control medium

BGC101

EXPERIMENTAL

Intramuscular injection of BGC101 (autologous EnEPC preparation)

Biological: BGC101 (autologous EnEPC preparation)

Interventions

BGC101 (autologous EnEPC preparation) BIOLOGICAL

Intramuscular injections - single treatment session

BGC101

Control medium BIOLOGICAL

Intramuscular injections - single treatment session

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Able to complete the study and comply with instructions.
• Capable of understanding the purpose of the study and the contents of the informed consent form.
• Aged at least 18 years.
• Non-pregnant and non-lactating female patients.
• Have the clinical indications diagnostic of CLI based on Rutherford category 4-5
• Have at least one of the hemodynamic indicators of severe peripheral arterial occlusive disease (WIfI ischemia grade 2):
• Toe pressure \< 40 mmHg
• Ankle pressure \< 70 mmHg
• TcPO2 \< 40mmHg
• Meeting one of the following conditions:
• Poor candidate for standard revascularization treatment for peripheral arterial disease due to unfavorable anatomy or high surgical/intervention risk based on the patient's underlying comorbidities.
• After undergoing clinically ineffective revascularization. Six weeks or more after undergoing a prior index limb revascularization the patient demonstrates:
• No improvement in clinical signs and symptoms of CLI as evidenced by lack of improvement in rest pain (when not under increased pain relief) and/or inadequate wound healing or progression of tissue loss despite adequate standard treatment.
• Four weeks or more after a revascularization failure.
• Technical Failure of the revascularization (inability to successfully cross or treat the intended target arterial path, thrombosis of the bypass graft or treated artery within 7 days of procedure)

You may not qualify if:

• Severe uncorrected aorto-iliac and/or common femoral artery disease, absent of femoral pulse or monophasic common femoral artery Doppler waveform.
• Concurrent therapy that, in the Investigator's opinion, would interfere with the evaluation of the feasibility 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 negatively impact the outcomes of the treatment under investigation.
• Prognosis of a major amputation (below or above the knee), within 4 weeks after screening.
• Severe wound (WIfI wound grade 2 or 3).
• Significant ongoing infection (WIfI infection grade 2 or 3).
• Relative or absolute contraindications for intramuscular injections at the intended treatment site, in cases such as severe skin lesions, severe edema or morbid obesity, based on clinician opinion.
• Patient suffering from active vasculitis
• Blood transfusions during the preceding 4 weeks (to exclude the potential of non-autologous cells in the harvested blood).
• Hemoglobin (Hb) less than 9 g/dL.
• Patient with HbA1C \> 8.5%
• Myocardial infarction, cerebral infarction , uncontrolled myocardial ischemia or persistent severe heart failure (ejection fraction \[EF\] \< 25%) during the preceding 3 months.
• Heart failure (New York Heart Association \[NYHA\] 3-4).
• Significant valvular disease or less than 4 weeks after valve replacement or repair

Sponsors & Collaborators

• BioGenCell Ltd.: lead
• Laniado Hospital: collaborator
• Rabin Medical Center: collaborator

Study Sites (5)

University of San Francisco

San Francisco, California, 94143, United States

Location

Yale University School of Medicine

New Haven, Connecticut, 06520-8039, United States

Location

Johns Hopkins Hospital

Baltimore, Maryland, 21287, United States

Location

Rambam Health Care Campus

Haifa, 31096, Israel

Location

Laniado Hospital

Netanya, 42150, Israel

Location

Related Publications (1)

• Porat Y, Assa-Kunik E, Belkin M, Krakovsky M, Lamensdorf I, Duvdevani R, Sivak G, Niven MJ, Bulvik S. A novel potential therapy for vascular diseases: blood-derived stem/progenitor cells specifically activated by dendritic cells. Diabetes Metab Res Rev. 2014 Oct;30(7):623-34. doi: 10.1002/dmrr.2543.

  PMID: 24638886 BACKGROUND

MeSH Terms

Conditions

Chronic Limb-Threatening Ischemia; Peripheral Arterial Disease; Peripheral Vascular Diseases

Condition Hierarchy (Ancestors)

Atherosclerosis; Arteriosclerosis; Arterial Occlusive Diseases; Vascular Diseases; Cardiovascular Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Ischemia

Study Officials

• Shlomo J Baytner, MD

  Director of Vascular Surgery, Laniado Hospital, IL

  PRINCIPAL INVESTIGATOR

• Michael Conte, MD

  University of California, San Francisco - Division Vascular and Endovascular surgery

  PRINCIPAL INVESTIGATOR

• Edouard Aboian, MD

  Yale University School of Medicine- Division of Vascular Surgery, Department of Surgery

  PRINCIPAL INVESTIGATOR

• Caitlin Hicks, MD

  Division of Vascular Surgery and Endovascular Therapy, Johns Hopkins Hospital

  PRINCIPAL INVESTIGATOR

• Tony Karram, MD

  Director Department of Vascular Surgery & Transplantation Rambam Health Care Campus - IL

  PRINCIPAL INVESTIGATOR

• Nathalie Moreels, MD

  University Hospital Ghent-Thoracale en vasculaire heelkunde

  PRINCIPAL INVESTIGATOR

• Jeffrey J Siracuse, MD

  Boston Medical Center

  PRINCIPAL INVESTIGATOR

• Khanjan Nagarsheth, MD

  University of Maryland

  PRINCIPAL INVESTIGATOR

• Paata Meshveliani, MD

  West Georgia Medical Center (Kutaisi Hospital)

  PRINCIPAL INVESTIGATOR

• Moshe Halak, MD

  The Sheba Fund for Health Services and Research, Sheba Medical Center at Tel HaShomer

  PRINCIPAL INVESTIGATOR

• Igor Laskowski, MD

  New York Medical College ("NYMC") and Westchester County Health Care Corporation, operator of Westchester Medical Center.

  PRINCIPAL INVESTIGATOR

• Mark Wyers, MD

  Beth Israel Deaconess Medical Center (Harvard-Boston)

  PRINCIPAL INVESTIGATOR

• Alisha Oropallo, MD

  Northwell Health

  PRINCIPAL INVESTIGATOR

• Alexander Reyzelman, MD

  Center for Clinical Research Castro Valley- Main site Post Street -Satellite site

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, OUTCOMES ASSESSOR
• Masking Details: the study will be double-blind for treatment allocation. Both BGC101 and placebo are supplied fresh in 5 mL ready-to use-syringes. Both BGC101 and placebo will be prepared by an unblinded trained personnel (external to the study staff members) who will not take part in the treatment and / or assessment of the patient. The patient will also be blinded and the investigator will delegate the unblinded and blinded staff in order to perform study procedures in a double-blind manner
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 15, 2016
• First Posted: June 17, 2016
• Study Start: June 1, 2016
• Primary Completion: July 1, 2017
• Study Completion (Estimated): December 1, 2027
• Last Updated: June 6, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (3); IL (2)