Study to Assess the Efficacy and Safety of CLBS12 in Patients with Critical Limb Ischemia (CLI)

Lisata Therapeutics

Status and phase: Completed; Phase 2

Conditions: Atherosclerosis Obliterans; Buerger Disease; Critical Limb Ischemia; Thromboangiitis Obliterans

Treatments: Drug: SOC; Biological: CLBS12

Study type: Interventional

Funder types: Industry

Identifiers

NCT02501018

CLBS12-P01

Details and patient eligibility

About

A prospective, open label, controlled, randomized, double arm, multi-center study to assess the efficacy and safety of CLBS12 in patients with critical limb ischemia (CLI) due to arteriosclerosis obliterans (ASO) with a single arm sub-study to assess the safety and potential efficacy of CLBS12 in patients with CLI due to Buerger's Disease (BD).

Full description

Main ASO Study: This study will compare safety and efficacy of intramuscular transplantation of autologous CD34+ cells (CLBS12) plus standard of care (SOC) pharmacotherapy (cell treatment arm) versus SOC pharmacotherapy alone (e.g., antiplatelets, anticoagulants, and vasodilators including prostanoids) (control arm) in subjects with CLI categorized as Rutherford score 4 or 5 due to ASO aged 20 to 85 years and with no endovascular or surgical revascularization options. Subjects assigned to the cell treatment arm will continue SOC pharmacotherapy and will also receive subcutaneous injections of GRAN® 5 ug/kg/day for 5 days and undergo apheresis on the last day of GRAN® administration. Then each subject in the cell treatment arm will receive intramuscular injections of autologous CD34+ cells. Subjects assigned to the control arm will continue to receive SOC pharmacotherapy alone with the possibility of receiving cell treatment via the rescue option.

BD Substudy: A single arm substudy is included to assess the safety and efficacy of intramuscular transplantation of CLBS12 in patients (N=~5) with CLI categorized as 4 or 5 Rutherford score due to BD aged 20 to 85 years. Subjects who give informed consent will be screened for eligibility within 28 days before registration. Subjects will continue SOC pharmacotherapy and will also receive subcutaneous injections of GRAN® 5 µg/kg/day for 5 days (Pretreatment Days 1 5) to mobilize CD34+ cells into the peripheral blood and undergo apheresis on pretreatment Day 5 to collect CD34+ cells. The choice of pharmacotherapy will be made by the investigators.

Each subject in a cell treatment arm will receive intramuscular injections of up to 1 × 10^6 autologous CD34+ cells/kg/limb. All subjects will be evaluated for efficacy and safety assessments during approximately 12 months.

Efficacy assessments include CLI free status, AFS, PFS, ABI, TBI, SPP, TcPO2, ICD, VAS, and AQA.

Enrollment: 33 patients

Sex: All

Ages: 20 to 85 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• subject has CLI caused by ASO or BD

Exclusion criteria

• < 20 years old

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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33 participants in 3 patient groups

CLI Due to ASO with CLBS12 + SOC

Experimental group

Description:

This group of subjects with CLI due to ASO will be administered with CLBS12 + SOC for collecting efficacy and safety data.

Treatment:

Biological: CLBS12

Drug: SOC

CLI Due to ASO with SOC

Active Comparator group

Description:

This group of subjects with CLI due to ASO will be administered with SOC only.

Treatment:

Drug: SOC

CLI Due to BD with CLBS12

Experimental group

Description:

CLBS12 will be administered to patients with CLI due to BD for collecting safety and efficacy data.

Treatment:

Biological: CLBS12

Drug: SOC