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

Primary Purpose

Critical Limb Ischemia

Status

Completed

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

ASCT01 (Autologous Stem Cell Transplantation)

About this trial

This is an interventional treatment trial for Critical Limb Ischemia focused on measuring CLI

Eligibility Criteria

18 Years - 80 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Male and Female patients in the age group of 18-80yrs.
Established CLI (confirmed by Rutherford 4 to 5) with angiographic evidence of significant infra-inguinal arterial occlusive disease
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
No surgical or interventional option for revascularization and no response to best standard care delivered as confirmed by a vascular surgeon and/or physician.
No immediate life-threatening complication from CLI which would demand immediate amputation.
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.
On optimal medical therapy
If diabetic, HgbA1c <10%

Exclusion Criteria:

Acute life threatening complication of limb ischemia with the need for immediate limb amputation to avoid death or clinical deterioration
Patients with confirmed Rutherford 6 condition with extensive tissue damage
Patients with documented terminal illness or cancer or any concomitant disease process with a life expectancy of less than 6 months.
Patients with a history of severe alcohol or drug abuse within 3 months of screening.
Known bone marrow diseases which preclude transplantation.
End-stage renal failure on regular dialysis treatment. Creatinine ≥2.0 mg/dl
Patients already enrolled in another investigational drug trial or completed within 1month.
Pregnancy.
Patients tested positive for HIV screen1or2, Hepatitis C antibody Hepatitis B surface-antigen, HepatitisBcore Antibody, Syphilis screen
Myocardial infarction / CVA / TIA within the past three months prior to enrollment
Revascularization procedure in target limb within 6 weeks prior to enrollment
Laboratory values as show below*
Currently taking immunosuppressive agents
If diabetic, diagnosis of proliferative retinopathy
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

Sites / Locations

Kansas City Vascular Foundation (KCV)

Arms of the Study

Arm 1

Arm Type

Other

Arm Label

ASCT01

Arm Description

ASCT01 (Autologous Stem Cell Transplantation)

Outcomes

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).

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.

Secondary Outcome Measures

Full Information

NCT ID

NCT01867190

First Posted

May 29, 2013

Last Updated

February 20, 2019

Sponsor

Lifecells, LLC.

1. Study Identification

Unique Protocol Identification Number

NCT01867190

Brief Title

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

Acronym

CLI

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

Study Type

Interventional

2. Study Status

Record Verification Date

February 2019

Overall Recruitment Status

Completed

Study Start Date

May 2013 (undefined)

Primary Completion Date

March 2016 (Actual)

Study Completion Date

March 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Lifecells, LLC.

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

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.

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Critical Limb Ischemia

Keywords

CLI

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Model Description

Autologous treatment for CLI open label

Masking

None (Open Label)

Allocation

N/A

Enrollment

24 (Actual)

8. Arms, Groups, and Interventions

Arm Title

ASCT01

Arm Type

Other

Arm Description

ASCT01 (Autologous Stem Cell Transplantation)

Intervention Type

Biological

Intervention Name(s)

ASCT01 (Autologous Stem Cell Transplantation)

Intervention Description

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

Primary Outcome Measure Information:

Title

Description

Time Frame

primary outcome measured at 3 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

80 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Male and Female patients in the age group of 18-80yrs. Established CLI (confirmed by Rutherford 4 to 5) with angiographic evidence of significant infra-inguinal arterial occlusive disease 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 No surgical or interventional option for revascularization and no response to best standard care delivered as confirmed by a vascular surgeon and/or physician. No immediate life-threatening complication from CLI which would demand immediate amputation. 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. On optimal medical therapy If diabetic, HgbA1c <10% Exclusion Criteria: Acute life threatening complication of limb ischemia with the need for immediate limb amputation to avoid death or clinical deterioration Patients with confirmed Rutherford 6 condition with extensive tissue damage Patients with documented terminal illness or cancer or any concomitant disease process with a life expectancy of less than 6 months. Patients with a history of severe alcohol or drug abuse within 3 months of screening. Known bone marrow diseases which preclude transplantation. End-stage renal failure on regular dialysis treatment. Creatinine ≥2.0 mg/dl Patients already enrolled in another investigational drug trial or completed within 1month. Pregnancy. Patients tested positive for HIV screen1or2, Hepatitis C antibody Hepatitis B surface-antigen, HepatitisBcore Antibody, Syphilis screen Myocardial infarction / CVA / TIA within the past three months prior to enrollment Revascularization procedure in target limb within 6 weeks prior to enrollment Laboratory values as show below* Currently taking immunosuppressive agents If diabetic, diagnosis of proliferative retinopathy 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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Paul T Sudhakar, MS, Pharm

Organizational Affiliation

Lifecells, LLC.

Official's Role

Study Director

Facility Information:

Facility Name

Kansas City Vascular Foundation (KCV)

City

North Kansas City

State/Province

Missouri

ZIP/Postal Code

64116

Country

United States

12. IPD Sharing Statement

Learn more about this trial

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

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