REX-001 Phase 3 clinical trial for CLTI

For more information on participating in our clinical trial, visit our trial website.

Following highly encouraging results from three Phase 2 studies, Ixaka initiated a pivotal, placebo-controlled, double-blind, adaptive Phase 3 trial in patients with Rutherford category 5 chronic limb-threatening ischemia (CLTI) and diabetes mellitus (DM).

The design of the trial, including the patient population and primary endpoint, was developed with our internationally renowned clinical advisors in consultation with the US Food and Drug Administration (FDA) and European Medicines Agency (EMA).

The Efficacy and Safety of REX-001 to Treat Ischemic Ulcers in Subjects with CLI Rutherford Category 5 and DM (NCT03174522)

We are currently recruiting patients with CLTI and DM for the clinical trial.

The study is a confirmatory Phase 3 clinical trial, and its design has been endorsed by the EMA, including the primary endpoint.

In the Phase 3 study, the efficacy and safety of REX-001 will be assessed in patients with Rutherford stage 5 CLTI with a primary endpoint of complete ulcer healing.
Amputation-free survival is included as a secondary endpoint.

Topline data will be available 12 months following the enrolment of the last patient in the trial.

Previous clinical trials

REX-001 has been administered to over 100 patients in three previous clinical trials.

  1. Phase 2 trial in patients with CLTI and diabetes (NCT00987363)
  2. Phase 2 trial in patients with CLTI without diabetes (NCT01408381)
  3. Phase 1/2 trial in patients with CLTI and diabetes (NCT00872326)

Results from these three Phase 2 studies in patients with CLTI are highly encouraging. Patients were classified according to the Rutherford scale as category 5 (ulcers) or category 4 (rest pain without ulceration).

Following treatment with REX-001, after 12 months, most patients were free of CLTI with significant improvement in their clinical condition (as assessed by change in Rutherford category) and complete ulcer healing and alleviation of rest pain in the majority of patients.

Blood vessel regeneration by REX-001

Angiographic imaging was used to assess revascularization following treatment with REX-001. In most treated patients there was a major improvement in blood vessels in the affected limb, which is likely to be responsible for the corresponding improvement in patient’s clinical condition.

Angiograms showing blood vessels prior to treatment (left) and 6 months after treatment with REX-001 (right)

Complete healing of ischemic ulcers by REX-001
Patients with CLTI often experience painful ischemic ulcers which may partially resolve with intensive wound management but are rarely completely healed. Following treatment with REX-001, most patients in the three Phase 2 studies with ulcers experienced complete or partial healing.

Diabetic ulcer at baseline (left) and after 6 months following treatment with REX-001 (right)
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