HNF partner Applied Therapeutics, a clinical-stage biopharmaceutical company, is conducting an international Phase III randomized trial called INSPIRE for patients with Sorbitol Dehydrogenase (SORD) Deficiency, a form of inherited neuropathy. The trial is designed to evaluate whether AT-007 can reduce toxic sorbitol levels and improve symptoms of the disease over time compared to placebo.

Who Can Enroll

The INSPIRE trial is enrolling participants between the ages of 18 and 55 who have been diagnosed with SORD Deficiency. Participants must have genetic confirmation of the diagnosis. To determine eligibility, a healthcare team will review medical history, perform blood and urine tests, conduct a physical exam, and verify the diagnosis. Genetic testing for SORD Deficiency will be provided at no cost to those who may be eligible.

What Participation Involves

Participants continue to see their regular doctors throughout the trial. The INSPIRE trial healthcare team will monitor each participant’s progress over a 24-month period and will coordinate visit schedules to work around participants’ routines. Regular health assessments will include medical evaluations, physical examinations, and blood and urine samples.

This is a placebo-controlled trial, as required by the FDA. For every two participants who receive AT-007, one will receive placebo. Participants who receive placebo during the main study will have the opportunity to transition to an open-label extension and receive AT-007 for an additional 24 months.

How to Get More Information

For more information or to inquire about participating in the INSPIRE trial of AT-007, email SORD@appliedtherapeutics.com.

To learn more about SORD Deficiency and other CMT research initiatives, visit our GRIN registry page or explore what is CMT.