AT-007 for Sensory and Autonomic Neuropathy
(INSPIRE Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial explores the effectiveness of a new treatment called AT-007 for individuals with SORD Deficiency, a condition affecting nerves responsible for sensation and autonomic functions like sweating. The study compares AT-007 to a placebo (a non-active substance) to determine if it can safely improve symptoms over two years. Candidates for this trial include those with a confirmed diagnosis of SORD Deficiency and stable medication routines. As a Phase 2, Phase 3 trial, this research evaluates the treatment's effectiveness in an initial group and represents the final step before FDA approval, offering participants the opportunity to contribute to a potentially groundbreaking therapy.
Will I have to stop taking my current medications?
The trial protocol does not specify if you must stop taking your current medications, but it excludes those using prescription medications likely to interfere with the study drug. It's best to discuss your current medications with the study team.
Is there any evidence suggesting that AT-007 is likely to be safe for humans?
Research has shown that AT-007 has been tested in other conditions, such as Classic Galactosemia, with strong safety results over three years in 185 patients. Most patients tolerated the treatment well. In studies for Sorbitol Dehydrogenase Deficiency, AT-007 was tested for 24 months and demonstrated promising safety outcomes. This treatment is an aldose reductase inhibitor, which helps prevent certain sugars from accumulating in the body. Overall, evidence suggests that AT-007 is well-tolerated, with no major safety concerns reported.12345
Why do researchers think this study treatment might be promising?
Most treatments for sensory and autonomic neuropathy focus on managing symptoms with medications like antidepressants, anticonvulsants, or pain relievers. But AT-007 works differently by targeting the root cause of the condition. It's an aldose reductase inhibitor, which means it aims to prevent nerve damage by stopping the buildup of harmful sugar alcohols in nerve cells. Researchers are excited because this new approach could potentially slow down or even halt disease progression, offering hope for more long-term relief compared to traditional symptom management.
What evidence suggests that AT-007 might be an effective treatment for SORD Deficiency?
Research has shown that AT-007, a medication under study in this trial, offers promising results for conditions like Classic Galactosemia, which resembles SORD Deficiency. Specifically, studies involving 185 patients over three years demonstrated the medication's effectiveness and safety. Patients experienced slower disease progression and improved health outcomes, with harmful substances like sorbitol reduced for up to 24 months. These findings suggest that AT-007 could also effectively treat SORD Deficiency by targeting similar pathways and reducing toxic buildup.13678
Who Is on the Research Team?
Michael E Shy, MD
Principal Investigator
University of Iowa
Are You a Good Fit for This Trial?
This trial is for adults aged 18-55 with SORD Deficiency, confirmed by medical records and gene analysis. Participants must have elevated sorbitol levels and agree to use birth control if they can bear children. Excluded are those with very severe disease, significant other health issues, high or low BMI outside set limits, substance abuse history, recent blood donations or participation in another drug study.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive either AT-007 or placebo daily for 24 months
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- AT-007
- Placebo
Find a Clinic Near You
Who Is Running the Clinical Trial?
Applied Therapeutics, Inc.
Lead Sponsor