Study to Assess Efficacy and Safety of Bone Marrow Derived Stem Cells in Patients With Critical Limb Ischemia (CLI)

February 20, 2019 updated by: Lifecells, LLC.

An Open-label Single Arm Phase 2 Proof of Concept Study to Assess the Efficacy and Safety of ASCT01 in Patients With Critical Limb Ischemia

This study will assess the safety and efficacy of intra-arterial infusion and intramuscular injection of an autologous, bone marrow-derived stem cell preparation (ASCT01) in patients with critical limb ischemia who have exhausted all medical and surgical therapeutic options. The safety and tolerability will be evaluated by regular monitoring of the general physical condition, vital signs, and the occurrence of AE and SAE, respectively. Furthermore, the standard biochemical and blood variables (red and white blood cell counts, Hb, Ht, platelets, sodium, potassium, chloride, calcium, phosphor, ASAT, ALAT, bilirubin, total protein, albumin, AP, cholesterol (LDL,HDL), triglycerides, urea and creatinine, immunoglobulins, HBA1c, C-peptide) will be checked before the treatment as well as 30 and 90 days after the treatment.

Study Overview

Status

Completed

Detailed Description

Efficacy will be determined by number of collateral arteries as assessed magnetic resonance angiography at baseline and again at 3 months, rate of major amputation (above the ankle) of the affected limb within 3 months or an unchanged critical limb ischemia of the affected limb after 3 months defined as less than 15% change in tcPO2 or ABI or absolute ankle pressure.Efficacy endpoint is at 3 months. Clinical endpoints and safety will be measured through 12 months.

Study Type

Interventional

Enrollment (Actual)

24

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

    • Missouri
      • North Kansas City, Missouri, United States, 64116
        • Kansas City Vascular Foundation (KCV)

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 80 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  1. Male and Female patients in the age group of 18-80yrs.
  2. Established CLI (confirmed by Rutherford 4 to 5) with angiographic evidence of significant infra-inguinal arterial occlusive disease
  3. Ankle Brachial Pressure Index (ABI) ≤ 0.6 or the absolute ankle blood pressure < 60 mm Hg or TcPO2<20 mmHg without tissue loss or TcPO2<40 mmHg if there is tissue loss or alternatively toe Brachial Pressure Index (TBI) less 0.5 or the absolute toe blood pressure less than 50 mm Hg
  4. No surgical or interventional option for revascularization and no response to best standard care delivered as confirmed by a vascular surgeon and/or physician.
  5. No immediate life-threatening complication from CLI which would demand immediate amputation.
  6. Patients who are able to understand the requirements of the study, and willing to provide voluntary written informed consent, abide by the study requirements, and agree to return for required follow-up visits.
  7. On optimal medical therapy
  8. If diabetic, HgbA1c <10%

Exclusion Criteria:

  1. Acute life threatening complication of limb ischemia with the need for immediate limb amputation to avoid death or clinical deterioration
  2. Patients with confirmed Rutherford 6 condition with extensive tissue damage
  3. Patients with documented terminal illness or cancer or any concomitant disease process with a life expectancy of less than 6 months.
  4. Patients with a history of severe alcohol or drug abuse within 3 months of screening.
  5. Known bone marrow diseases which preclude transplantation.
  6. End-stage renal failure on regular dialysis treatment. Creatinine ≥2.0 mg/dl
  7. Patients already enrolled in another investigational drug trial or completed within 1month.
  8. Pregnancy.
  9. Patients tested positive for HIV screen1or2, Hepatitis C antibody Hepatitis B surface-antigen, HepatitisBcore Antibody, Syphilis screen
  10. Myocardial infarction / CVA / TIA within the past three months prior to enrollment
  11. Revascularization procedure in target limb within 6 weeks prior to enrollment
  12. Laboratory values as show below*
  13. Currently taking immunosuppressive agents
  14. If diabetic, diagnosis of proliferative retinopathy
  15. Patients with infected ulcers or systemic infections *Laboratory Values: Hemoglobin <10 g/dL Platelet count <100,000/microL ALT >60 U/L AST >60 U/L Bilirubin >1.0 mg/dL INR >1.3 unless on Coumadin and at Investigator discretion APTT >40 second unless on Lovenox or Heparin and at Investigator's discretion

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Other: ASCT01
ASCT01 (Autologous Stem Cell Transplantation)
Single Administration of the entire ASCT01 preparation at rate if 10million CD45+ cells per milliliter at 5 ml per minute via the intra- via intra-arterial infusion route and 6 or more injections of approximately 0.2 ml each intramuscular route

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
To assess the efficacy and safety of intra-arterial infusion and intramuscular injection of ASCT01 on the combined primary endpoint of major amputation (above the ankle) or persisting critical limb ischemia (no clinical or perfusion improvement).
Time Frame: primary outcome measured at 3 months
The primary outcome variable is "treatment failure" defined as major amputation (above the ankle) of the affected limb within 3 months or an unchanged critical limb ischemia of the affected limb after 3 months defined as less than 15% change in tcPO2 or ABI or absolute ankle pressure.
primary outcome measured at 3 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Investigators

  • Study Director: Paul T Sudhakar, MS, Pharm, Lifecells, LLC.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

May 1, 2013

Primary Completion (Actual)

March 1, 2016

Study Completion (Actual)

March 1, 2016

Study Registration Dates

First Submitted

May 29, 2013

First Submitted That Met QC Criteria

May 31, 2013

First Posted (Estimate)

June 3, 2013

Study Record Updates

Last Update Posted (Actual)

February 22, 2019

Last Update Submitted That Met QC Criteria

February 20, 2019

Last Verified

February 1, 2019

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • IND 15069

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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