Cellular therapy with ixmyelocel-T to treat critical limb ischemia: The randomized, double-blind, placebo-controlled RESTORE-CLI trial

Richard J. Powell, William A. Marston, Scott A. Berceli, Raul Guzman, Timothy D. Henry, Amy T. Longcore, Theresa P. Stern, Sharon Watling, Ronnda L. Bartel

Research output: Contribution to journalArticlepeer-review

129 Scopus citations

Abstract

Ixmyelocel-T is a patient-specific, expanded, multicellular therapy evaluated in patients with lower extremity critical limb ischemia (CLI) with no options for revascularization. This randomized, double-blind, placebo-controlled, phase 2 trial (RESTORE-CLI) compared the efficacy and safety of intramuscular injections of ixmyelocel-T with placebo. Patients received one-time injections over 20 locations in a single leg and were followed for 12 months. Safety assessments included occurrence of adverse events. Efficacy assessments included time to first occurrence of treatment failure (TTF; major amputation of injected leg; all-cause mortality; doubling of total wound surface area from baseline; de novo gangrene) and amputation-free survival (AFS; major amputation of injected leg; all-cause mortality). A total of 77 patients underwent bone marrow or sham aspiration; 72 patients received ixmyelocel-T (48 patients) or placebo (24 patients). Adverse event rates were similar. Ixmyelocel-T treatment led to a significantly prolonged TTF (P = 0.0032, logrank test). AFS had a clinically meaningful 32% reduction in event rate that was not statistically significant (P = 0.3880, logrank test). Treatment effect in post hoc analyses of patients with baseline wounds was more pronounced (TTF: P < 0.0001, AFS: P = 0.0802, logrank test). Ixmyelocel-T treatment was well tolerated and may offer a potential new treatment option.

Original languageEnglish (US)
Pages (from-to)1280-1286
Number of pages7
JournalMolecular Therapy
Volume20
Issue number6
DOIs
StatePublished - Jun 2012
Externally publishedYes

Bibliographical note

Funding Information:
We thank William Hiatt (University of Colorado Denver School of Medicine) for his comments and review and Lisa McCormick (LSM Pharmaceutical Consulting) for her assistance in manuscript preparation. This trial was funded by Aastrom Biosciences. R.J.P. has been paid consulting fees by AnGes and Aastrom Biosciences. W.A.M. has been paid consulting fees by Aastrom. A.T.L., T.P.S., S.W., and R.L.B. are paid employees of Aastrom.

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