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

This study is currently recruiting participants. (see Contacts and Locations)
Verified September 2014 by Lifecells, LLC.
Sponsor:
Information provided by (Responsible Party):
Lifecells, LLC.
ClinicalTrials.gov Identifier:
NCT01867190
First received: May 29, 2013
Last updated: September 12, 2014
Last verified: September 2014
  Purpose

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) versus placebo 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.


Condition Intervention Phase
Critical Limb Ischemia
Biological: ASCT01 (Autologous Stem Cell Transplantation)
Phase 1
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: 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

Further study details as provided by Lifecells, LLC.:

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: Yes ]
    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.


Estimated Enrollment: 24
Study Start Date: May 2013
Estimated Study Completion Date: June 2015
Estimated Primary Completion Date: December 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: ASCT01
CD34+ and CD45+ cells in ASCT01 preparation
Biological: 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

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.

  Eligibility

Ages Eligible for Study:   18 Years to 80 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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 Bsurface-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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01867190

Contacts
Contact: Paul T Sudhakar, M.S. Pharm 816-507-8249 paulsmrtc@aol.com

Locations
United States, Missouri
Kansas City Vascular Foundation (KCV) Recruiting
North Kansas City, Missouri, United States, 64116
Contact: Rebecca Thomas, CVRN - BC    816-842-5555 ext 1605    rthomas@kcvascular.com   
Contact: Kim Campbell, ANP-BC    816-842-5555    kcampbell@kcvascular.com   
Principal Investigator: Karl R Stark, MD         
Sponsors and Collaborators
Lifecells, LLC.
Investigators
Study Director: Paul T Sudhakar, MS, Pharm Lifecells, LLC.
  More Information

No publications provided

Responsible Party: Lifecells, LLC.
ClinicalTrials.gov Identifier: NCT01867190     History of Changes
Other Study ID Numbers: IND 15069
Study First Received: May 29, 2013
Last Updated: September 12, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Lifecells, LLC.:
CLI

Additional relevant MeSH terms:
Ischemia
Pathologic Processes

ClinicalTrials.gov processed this record on November 20, 2014