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

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
24

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • 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

  • United States
    • 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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Other: ASCT01
ASCT01 (Autologous Stem Cell Transplantation)
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

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Lifecells, LLC.

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • 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

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
May 1, 2013

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
March 1, 2016

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
March 1, 2016

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
May 29, 2013

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 31, 2013

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
June 3, 2013

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
February 22, 2019

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 20, 2019

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
February 1, 2019

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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