CMXI signed an agreement with NIH to collaborate on a P2 clinical study in patients with intermittent claudication (IC).
The Bright Cell Therapy Study in Peripheral Arterial Disease (PAD) will be … funded by National Heart, Lung, and Blood Institute (NHLBI) of NIH in Collaboration with Cardiovascular Cell Therapy Research Network (CCTRN)
IC is caused by peripheral arterial disease (PAD), a condition causing reduced flow of blood and oxygen to muscles of the leg. The study funds from the NHLBI/NIH will be managed by the … Cardiovascular Cell Therapy Research Network (CCTRN), which is also responsible for enrolling patients that includes 7 centers in the US with experience and expertise in stem cell clinical trials studying treatments for cardiovascular heart diseases.
The P2 PACE (Patients with Intermittent Claudication Injected with ALDH Bright Cells) study is an 80 patient, double-blind, placebo-controlled clinical trial intended to demonstrate the safety and efficacy of ALD-301 (Bright Cells) in patients diagnosed with IC. ALD-301 is a population of autologous pluripotent stem cells isolated from the patients' bone marrow using CMXI's proprietary Bright Cell technology. These adult stem cells express high levels of the enzyme ALDH, an indicator of biological activity in heterogeneous early stage stem cells.
The primary endpoints of the study are safety and the change in peak walking time at 6 months compared to baseline. Additionally, changes in leg collateral arterial anatomy, calf muscle blood flow, and tissue perfusion as determined by magnetic resonance imaging (MRI) will be examined. These novel MRI techniques are incorporated into the study to assess perfusion, providing a unique set of data potentially supporting the angiogenic mechanism of Bright Cells.
The Bottom Line: The clinical study has received IND approval from the FDA and is expected to begin enrollment in Q1/13 upon the IRB approvals from the participating centers. This is the 1st randomized clinical trial to look at the benefits of autologous stem cell therapy in PAD patients with IC. It will collect important mechanistic and clinical information on the efficacy and safety of the direct injection of Bright Cells into these patients. It will also evaluate the utility of advanced imaging endpoints that could be used in the future to further understand the impact of novel therapies in this patient population. This clinical study builds on the strong data showing increased blood flow and improved clinical status from CMXI's previous P1/2 study of ALD-301 in CLI published last year. In the PACE study, ALD-301 will be delivered in the same manner, via direct, intramuscular injection in a grid pattern of the affected lower limb. CMXI will be responsible for manufacturing ALD-301 for the clinical trial and will have certain rights to data generated during the trial.
Intermittent claudication is a serious consequence of arteriosclerosis which, if left untreated, will likely progress to pain at rest and possibly open wounds. CMXI's experience with the AutoloGel product and the clinical treatment of lower extremity wounds resulting from CLI has provides a full appreciation of the difficult clinical outcomes associated with this compromised patient population. PAD is a major unmet medical need affecting approximately 8 to 10 M patients in the U.S. IC is a significant subset of the PAD population and is characterized by pain in the lower legs while in motion that resolves upon rest. Critical limb ischemia (CLI) is the advanced form of PAD, and is associated with poor clinical outcomes and increased morbidity. An important goal of medical intervention is to attempt to prevent the progression of patients from IC to CLI.
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