HNF Partner Applied Therapeutics is Currently Enrolling SORD Deficiency Patients for the INSPIRE Phase III Clinical Trial

Applied Therapeutics, a clinical stage biopharmaceutical company, is conducting an international phase III randomized interventional trial for patients with Sorbitol Dehydrogenase (SORD) Deficiency called INSPIRE.  This trial is designed to investigate the ability of AT-007 versus placebo to reduce toxic sorbitol levels, and to evaluate the effect of AT-007 on improving symptoms of the disease over a longer period of time.

Applied Therapeutics is enrolling participants between the ages of 18 and 55 living with SORD Deficiency into the INSPIRE trial. 

The objectives of the INSPIRE clinical trial include understanding the ability of AT-007 to reduce sorbitol levels and determining the impact AT-007 has on SORD Deficiency clinical symptoms and outcomes.  This clinical trial is supported by doctors, nurses, and other healthcare professionals. The commitment of each participant and the entire study team is important to help meet the objectives of the clinical trial. This INSPIRE trial follows strict ethical and governmental guidelines to ensure that participants’ rights are protected while the information is being collected.

Participants will need to have a genetic confirmation of SORD Deficiency.  To help determine eligibility, participants will be seen by a healthcare team that will review medical history, perform blood and urine tests, conduct a physical exam, and verify a diagnosis of SORD Deficiency.  Genetic testing for SORD Deficiency will be provided at no charge to those potentially eligible for the trial.

While participating in the INSPIRE trial, participants will still see their regular doctors.  The INSPIRE trial healthcare team will monitor each participant’s progress throughout the trial over a 24-month period.  Trial staff will work with each participant to coordinate visits within their schedule.  The healthcare team will routinely ask participants about their experience during the INSPIRE trial.  They will also conduct regular health assessments that will include medical evaluations, physical examinations, and samples of blood and urine.

This is a placebo-controlled trial as required by the FDA.  A placebo-controlled trial helps doctors determine how the trial drug performs compared to placebo.  For every 2 participants who get the active drug, 1 participant will get placebo.  Participants who receive placebo treatment in the main study, will have the opportunity to transition to the open-label extension part of the study and receive AT-007 for 24 months.

For more information or to inquire about participating in the INSPIRE trial of AT-007 in SORD Deficiency, please email [email protected]