AT-007 for Sensory and Autonomic Neuropathy

(INSPIRE Trial)

Recruiting at 9 trial locations

Overseen By Michael E Shy, MD

Age: 18 - 65

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: Applied Therapeutics, Inc.

Must not be taking: Prescription medications

Disqualifiers: Diabetes, Neuropathy, Myopathy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 24 months

56 Participants Needed

Trial Summary

What is the purpose of this trial?

This study is designed to assess the efficacy and safety of AT-007 treatment in patients with SORD Deficiency. This randomized, double-blind study will assess the effect of AT-007 compared to Placebo in SORD Deficiency patients for 24 months.

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.

How does the drug AT-007 differ from other treatments for sensory and autonomic neuropathy?

AT-007 is unique because it is being specifically studied for its effects on sensory and autonomic neuropathy, a condition with limited standard treatment options. Unlike other treatments that may focus on immune modulation or symptom management, AT-007's mechanism of action and potential benefits are still being explored in this context, making it a novel option for patients with this condition. ^{1 2 3 4 5}

Research Team

Michael E Shy, MD

Principal Investigator

University of Iowa

Eligibility Criteria

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

I cannot have children or agree to use birth control during the study.

I am a male who is either surgically sterile or will use birth control during and after the study.

Willing and able to provide signed and dated informed consent prior to any study-related procedures and willing and able to comply with all study procedures

Exclusion Criteria

I do not have any major health issues that could affect the study's safety or results.

Body Mass Index (BMI) >35 kg/m2 or clinically relevant underweight, weight loss suggestive of a pathology unrelated to SORD deficiency, or BMI < 17.5 kg/m2

History of significant drug allergy or drug hypersensitivity

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either AT-007 or placebo daily for 24 months

24 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• AT-007
• Placebo

Trial Overview The trial tests the efficacy and safety of AT-007 against a placebo over 24 months in patients with SORD Deficiency. It's randomized and double-blind, meaning neither the researchers nor participants know who receives the actual drug versus the placebo.

Participant Groups

Group I: AT-007 Active Control 1 Intervention

AT-007 is an Aldose reductase inhibitor

Group II: Placebo Placebo Group 1 Intervention

Is an non-active control

Who Is Running the Clinical Trial?

Applied Therapeutics, Inc.

Lead Sponsor

Trials

5

Recruited

900+

References

1. England pubmed.ncbi.nlm.nih.gov

Validation of the composite autonomic symptom scale 31 (COMPASS-31) in patients with and without small fiber polyneuropathy. [2022]

2. United States pubmed.ncbi.nlm.nih.gov

Randomized trial of l-serine in patients with hereditary sensory and autonomic neuropathy type 1. [2022]

3. Japan pubmed.ncbi.nlm.nih.gov

[Autoimmune autonomic ganglionopathy and acute autonomic and sensory neuropathy]. [2019]

4. England pubmed.ncbi.nlm.nih.gov

Clinicopathological features of acute autonomic and sensory neuropathy. [2010]

5. Germany pubmed.ncbi.nlm.nih.gov

Acute autonomic and sensory neuropathy: a case report. [2019]