MedPath

An Efficacy and Safety Study of Ixmyelocel-T in Patients With Critical Limb Ischemia (CLI)

Phase 3
Completed
Conditions
Critical Limb Ischemia
Interventions
Biological: Ixmyelocel-T
Other: Placebo
Registration Number
NCT01483898
Lead Sponsor
Vericel Corporation
Brief Summary

This study is designed to evaluate the efficacy and safety of ixmyelocel-T, a patient-specific expanded multicellular therapy, for the treatment of patients with critical limb ischemia (CLI). The study is a randomized, vehicle controlled (placebo)study in CLI patients who have no option for revascularization procedures. All patients randomized will undergo a small volume bone marrow aspiration in a 15-minute outpatient or in-office procedure. All patients will receive injections of either ixmyelocel-T or vehicle-control into their pre-identified index leg. Patients will be followed for 18 months.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
41
Inclusion Criteria
  • Males and nonpregnant, nonlactating females

  • Ages 35 to 90 years of age

  • Diagnosis of CLI with tissue loss (corresponding to Rutherford Category 5; see Appendix B) having an ulcer size of at least 0.5 cm2, a smaller sized ulcer penetrating into the subcutaneous tissue, and/or gangrene (dry). In addition, the subject must have ONE of the following documented at screening:

    • Ankle systolic pressure < 70 mm Hg
    • Toe systolic pressure < 50 mm Hg
    • TcPO2 < 30 mm Hg (in a supine position)
  • Subjects must have no reasonable standard-of-care options for surgical or endovascular revascularization interventions

  • Subjects must have the following:

    • A narrative documenting the reasons why the site vascular specialist considers the subject "no option". A vascular specialist will be the principal investigator (PI) or subinvestigator and is defined as: vascular surgeon, interventional cardiologist, certified vascular medicine specialist, or interventional radiologist; AND
    • Secondary confirmation by an independent Eligibility Review Committee (ERC; see Section 8.1) after review of appropriate documents including, but not limited to: imaging results, medical records, surgical history, site vascular specialist narrative documenting reasons for "no option," and/or lab reports.
  • Major amputation in the index leg or death is not anticipated within 3 months of screening in the opinion of the vascular specialist (who must be the PI or subinvestigator)

  • In the opinion of the investigator, the subject is controlled on medical therapy indicated for CLI (unless there is a documented contraindication or intolerance)

  • Subject is current with all age-appropriate American Cancer Society (ACS) or similar (e.g., United States Preventative Service Task Force) screening guidelines

  • Given medical history and concurrent medication, the subject is an acceptable candidate for bone marrow aspiration and intramuscular injection procedures in the opinion of the Investigator

  • Subject is willing and able to comply with the scheduled visits, aspiration/injection procedure, wound care instructions treatment plan, and other study procedures for the duration of the study

  • Provide a personally-signed and dated informed consent document indicating that the subject (or a legally-acceptable representative, if permitted by the site's Investigational Review Board [IRB]) has been informed of all pertinent aspects of the study

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Exclusion Criteria

Patients presenting with any of the following will not be randomized:

Disease-specific:

  • Failed open surgical revascularization (on index leg) within 4 weeks of screening Visit 1
  • Acute limb-threatening ischemia, trauma, known non-atherosclerotic vascular disease (e.g., temporal/giant cell arteritis, Takayasu's arteritis, Raynaud's occlusive disease, Buerger's disease), embolic disease, aortoiliac disease with > 50% stenosis, or history of hypercoagulable states
  • Advanced CLI (i.e., nonsalvageable) defined as Rutherford Category 6
  • Clinical evidence of invasive infection in index leg (e.g., cellulitis, osteomyelitis, wet gangrene)
  • At screening, non-heel wound size of > 20 cm2 (excluding toe gangrene); or wounds on the heel > 10 cm2 on the index leg as measured by the Wound Core Lab (WCL) from photographs (and/or acetates) provided by the site
  • Previous amputation at or above the talus in the index leg Medical History
  • Hemoglobin A1c (HbA1c) ≥ 10% at screening
  • Diabetic subjects with uncontrolled or untreated proliferative retinopathy as determined by dilated eye exam (by qualified eye care professional as per American Diabetes Association guidelines)
  • Blood clotting disorder not caused by medication (e.g., thrombophilia)
  • Active non-basal cell cutaneous malignancy requiring surgery, chemotherapy, and/or radiation in the past 12 months
  • Current documented drug or alcohol abuse that would interfere with the subject's compliance with study procedures
  • Known allergies to any equine, porcine, or bovine products
  • Body mass index (BMI) ≥ 50 kg/m2 at screening
  • Established chronic kidney disease (CKD) requiring dialysis (Stage 5); estimated creatinine clearance < 15 mg/mL/min at screening
  • Systolic blood pressure (SBP) > 200 mm Hg or diastolic blood pressure (DBP) > 120 mm Hg or papilledema noted via ophthalmoscope at screening physical exam
  • Within 3 months prior to screening, a clinically significant history of cardiac disease

Laboratory Parameters:

  • Abnormal laboratory values (performed at central lab) at screening:

    • Platelets < 50,000 μL
    • Aspartate aminotransferase/alanine aminotransferase (AST/ALT) > 3 times the upper limit of normal (ULN)
    • Human immunodeficiency virus 1 (HIV 1), HIV 2, or syphilis positive (rapid plasma reagin [RPR])
    • Active hepatitis B surface antigen (HBsAg) or hepatitis C virus (HCV); Exclusionary Procedures, Devices, or Medication
  • Exposure to immunosuppressive therapy for oncologic or chronic non-oncologic reasons in the prior 12 months or expected requirement over the course of the study (e.g., chemotherapy, radiation therapy, methotrexate)

  • Concurrent participation in another clinical trial or receiving experimental medication within 30 days of screening or having previously been exposed to Aastrom's ixmyelocel T product [previously known as tissue repair cells (TRC), cardiac repair cells (CRC), vascular repair cells (VRC)]

  • On the index leg, use of concomitant wound treatments not currently approved for ischemic wound-healing within 30 days prior to screening or plans to initiate new, nonstandard-of-care treatments to the index leg during the study

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Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
ixmyelocel-TIxmyelocel-T-
PlaceboPlacebo-
Primary Outcome Measures
NameTimeMethod
Amputation free survival (AFS) at 12 months post-injection12 months

The primary objective will be to assess the efficacy of ixmyelocel-T compared to placebo (vehicle control) on AFS at 12 months post-injection in CLI patients with no options for revascularization. Amputation free survival is defined as time to the first occurrence of either major amputation (above the talus) in the index leg or all-cause mortality (death).

Secondary Outcome Measures
NameTimeMethod
Percent of patients with adverse events18 months

A secondary objective will be to evaluate the overall safety and tolerability of ixmyelocel-T versus placebo in patients with CLI from time of aspiration through 18 months post-treatment/follow-up by % of patients with adverse events.

Percent of patients with complete wound closure by Month 1212 months

A secondary objective is to assess the percent of patients with at least 1 ischemic wound on the index leg that is present at Visit 3 (preinjection) having complete closure by Month 12.

Percent of patients experiencing a major cardiac event (MACE) by Months 6, 12, and 186, 12 and 18 months

% of patients experiencing a MACE event defined as cardiovascular mortality, myocardial infarction, chest pain requiring hospitalization, or stroke.

Trial Locations

Locations (86)

Union Memorial Hospital

🇺🇸

Baltimore, Maryland, United States

UCLA Gonda Venous Center & Ambulatory Procedure Unit

🇺🇸

Los Angeles, California, United States

Rush University Medical Center

🇺🇸

Chicago, Illinois, United States

Brigham and Women's Hospital

🇺🇸

Boston, Massachusetts, United States

Boston Medical Center

🇺🇸

Boston, Massachusetts, United States

VA Pittsburgh Healthcare System

🇺🇸

Pittsburgh, Pennsylvania, United States

Texas Heart Institute at St. Luke's Episcopal Hospital

🇺🇸

Houston, Texas, United States

Kaiser Permanente Medical Center

🇺🇸

San Diego, California, United States

University of Miami Hospital

🇺🇸

Miami, Florida, United States

St. Vincent Medical Group, Inc.

🇺🇸

Indianapolis, Indiana, United States

Abbott Northwestern Hospital / Minneapolis Heart Institute-Vascular Clinic

🇺🇸

Minneapolis, Minnesota, United States

Minneapolis VA Health Care System

🇺🇸

Minneapolis, Minnesota, United States

Duke University Medical Center

🇺🇸

Durham, North Carolina, United States

Audie L Murphy VA Hospital - Pathology Laboratory

🇺🇸

San Antonio, Texas, United States

Vanderbilt University Medical Center

🇺🇸

Nashville, Tennessee, United States

Arkansas Primary Care Clinic, P.A.

🇺🇸

Little Rock, Arkansas, United States

Cardiology and Vascular Associates, P.C.

🇺🇸

Berkley, Michigan, United States

Cardiology Consultants

🇺🇸

Pensacola, Florida, United States

University of Wisconsin

🇺🇸

Madison, Wisconsin, United States

Charleston Area Medical Center - Memorial Hospital

🇺🇸

Charleston, West Virginia, United States

Swedish Medical Center

🇺🇸

Seattle, Washington, United States

University of Washington

🇺🇸

Seattle, Washington, United States

University of Arizona

🇺🇸

Tucson, Arizona, United States

VA Loma Linda Healthcare

🇺🇸

Loma Linda, California, United States

Holy Cross Hospital

🇺🇸

Fort Lauderdale, Florida, United States

First Coast Cardiovascular Institute, P.A.

🇺🇸

Jacksonville, Florida, United States

John Muir Medical Center - Concord Campus

🇺🇸

Concord, California, United States

Yale School of Medicine

🇺🇸

New Haven, Connecticut, United States

Bethesda Memorial Hospital - Clinical Research Center

🇺🇸

Boynton Beach, Florida, United States

Florida Research Network, LLC

🇺🇸

Gainesville, Florida, United States

University of Illinois at Chicago - Clinical Sciences North

🇺🇸

Chicago, Illinois, United States

Baptist Hospital

🇺🇸

Pensacola, Florida, United States

Grove Place Surgery Center

🇺🇸

Vero Beach, Florida, United States

University of Maryland Medical Center

🇺🇸

Baltimore, Maryland, United States

Vascular Surgical Associates, P.C.

🇺🇸

Marietta, Georgia, United States

Memorial Medical Center

🇺🇸

Springfield, Illinois, United States

Heartland Vascular Medicine and Surgery

🇺🇸

Windsor Heights, Iowa, United States

UMASS Memorial Health Care

🇺🇸

Worcester, Massachusetts, United States

Ochsner Clinic foundation

🇺🇸

New Orleans, Louisiana, United States

Michigan Vascular Research Center

🇺🇸

Flint, Michigan, United States

Integrated Vascular Vein Center of Michigan

🇺🇸

Grand Blanc, Michigan, United States

Oakwood Hospital and Medical Center

🇺🇸

Dearborn, Michigan, United States

Saint Joseph Mercy Hospital

🇺🇸

Ypsilanti, Michigan, United States

Michigan CardioVascular Institutue at Covenant Medical Center

🇺🇸

Saginaw, Michigan, United States

Hattiesburg Clinic, P.A.

🇺🇸

Hattiesburg, Mississippi, United States

University of Minnesota Medical School

🇺🇸

Minneapolis, Minnesota, United States

University of North Carolina Hospital

🇺🇸

Chapel Hill, North Carolina, United States

Temple University Hospital

🇺🇸

Philadelphia, Pennsylvania, United States

Veterans' Administration Medical Center

🇺🇸

Pittsburgh, Pennsylvania, United States

The Methodist Hospital

🇺🇸

Houston, Texas, United States

Allegheny General Hospital

🇺🇸

Pittsburgh, Pennsylvania, United States

DFW Vascular Group, LLP

🇺🇸

Dallas, Texas, United States

University of Utah

🇺🇸

Salt Lake City, Utah, United States

Cascade Vascular Associates, P.S.

🇺🇸

Tacoma, Washington, United States

University of California San Diego Medical Center

🇺🇸

San Diego, California, United States

University of California San Francisco - Division of Vascular and Endovascular Surgery

🇺🇸

San Francisco, California, United States

Cardiology PC

🇺🇸

Birmingham, Alabama, United States

Cardio-Thoracic Surgeons, P.C.

🇺🇸

Birmingham, Alabama, United States

University Of Alabama At Birmingham

🇺🇸

Birmingham, Alabama, United States

Arizona Heart Institute

🇺🇸

Phoenix, Arizona, United States

Carl T. Hayden VA Medical Center

🇺🇸

Phoenix, Arizona, United States

Sutter Medical Group - Cardiology

🇺🇸

Sacramento, California, United States

University of Oklahoma Health Science Center

🇺🇸

Oklahoma City, Oklahoma, United States

University of Colorado Denver - Anschutz Medical Campus

🇺🇸

Aurora, Colorado, United States

Orlando Health

🇺🇸

Orlando, Florida, United States

Wheaton Franciscan Medical Group, Inc.

🇺🇸

Milwaukee, Wisconsin, United States

VA Ann Arbor Healthcare System

🇺🇸

Ann Arbor, Michigan, United States

John D. Dingell VA Medical Center

🇺🇸

Detroit, Michigan, United States

Henry Ford Hospital

🇺🇸

Detroit, Michigan, United States

Omaha VAMC

🇺🇸

Omaha, Nebraska, United States

Wake Forest Baptist Health

🇺🇸

Winston-Salem, North Carolina, United States

Medical College of Wisconsin

🇺🇸

Milwaukee, Wisconsin, United States

Columbia Saint Mary's

🇺🇸

Milwaukee, Wisconsin, United States

Indiana/Ohio Heart

🇺🇸

Fort Wayne, Indiana, United States

Dartmouth-Hitchcock Medical Center

🇺🇸

Lebanon, New Hampshire, United States

Saint Luke's Hospital

🇺🇸

Kansas City, Missouri, United States

Vascular Access Center of West Orange

🇺🇸

West Orange, New Jersey, United States

Mount Sinai Medical Center

🇺🇸

New York, New York, United States

University of Buffalo Surgeons Inc.

🇺🇸

Buffalo, New York, United States

Stony Brook University Medical Center

🇺🇸

Stony Brook, New York, United States

Jobst Vascular Institute

🇺🇸

Toledo, Ohio, United States

University Hospitals Case Medical Center

🇺🇸

Cleveland, Ohio, United States

University of Toledo Medical Center

🇺🇸

Toledo, Ohio, United States

Penn State Milton S. Hershey Medical Center

🇺🇸

Hershey, Pennsylvania, United States

Central Arkansas Veteran's Healthcare System

🇺🇸

Little Rock, Arkansas, United States

Malcom Randall Veterans Affairs Medical Center

🇺🇸

Gainesville, Florida, United States

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