Clinical trial NCT02501018: Study to Assess the Efficacy and Safety of CLBS12 in Patients with Critical Limb Ischemia (CLI)
Official title: A Prospective, Open Label, Controlled, Randomized, Multicenter 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 Substudy to Assess the Safety and Potential Efficacy of CLBS12 in Patients with CLI Due to Buerger's Disease (BD)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Critical Limb Ischemia
Buerger Disease
Thromboangiitis Obliterans
Atherosclerosis Obliterans
Keywords: CLI, ASO, TAO
Lead sponsor: Lisata Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2015-07-17
Enrollment: 33
Sex: ALL
Minimum age (years): 20
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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).

Detailed 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.

Eligibility criteria: Inclusion Criteria:

* subject has CLI caused by ASO or BD

Exclusion Criteria:

* \< 20 years old

Primary outcomes:
measure: Time to continuous CLI-free status
timeFrame: 1 year
Secondary outcomes:
Other outcomes:
Locations:
city: Midorigaoka
country: Japan
facility: Asahikawa Medical University Hospital - 1-1-1 Higashi-2jou
state: Asahikawa-shi
city: Fukuoka
country: Japan
facility: Fukuoka Sanno Hospital
city: Kamakura
country: Japan
facility: Shonan Kamakura General Hospital
city: Kobe
country: Japan
facility: Kobe City Medical Center General Hospital
city: Kobe
country: Japan
facility: Shinsuma General Hospital
city: Oita
country: Japan
facility: Oita Oka Hospital
city: Osaka
country: Japan
facility: Osaka Saiseikai Nakatsu Hospital
city: Tokyo
country: Japan
facility: Nippon Medical School Hospital
city: Tokyo
country: Japan
facility: Keio University Hospital
city: Tokyo
country: Japan
facility: Toho University Medical Center Ohashi Hospital
city: Tokyo
country: Japan
facility: Tokyo Medical University Hospital
city: Tokyo
country: Japan
facility: Tokyo Women's Medical University
Study officials:
affiliation: Lisata Therapeutics, Inc.
name: Kristen K Buck, MD
role: STUDY_DIRECTOR
References: