Pharmacodynamic EffIcacy and Clinical Benefit of AT 007 in Patients With Sorbitol Dehydrogenase (SORD) Deficiency (INSPIRE)
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|ClinicalTrials.gov Identifier: NCT05397665|
Recruitment Status : Recruiting
First Posted : May 31, 2022
Last Update Posted : May 31, 2022
|Condition or disease||Intervention/treatment||Phase|
|Hereditary Neuropathy Caused by SORD Deficiency||Drug: AT-007 Drug: Placebo||Phase 2 Phase 3|
This international, multi-center, randomized, double-blinded, placebo-controlled, phase 2-3 study is designed to assess the pharmacodynamic (PD) efficacy and clinical benefit of long term administration of AT 007 versus placebo in male and non-pregnant female subjects with genetically confirmed SORD Deficiency aged 18-55 and able to ambulate with a 10MWRT time of <10 seconds.
The study will be conducted at up to 12 sites worldwide. Genetically confirmed SORD Deficiency patients with blood sorbitol levels >10,000 ng/ml will be screened and randomized in a 2:1 ratio to receive either AT-007 daily or a matching placebo for 24 months. A total of up to 72 subjects will be enrolled.
The primary clinical outcome measure, 10-meter walk-run test (10MWRT), will be assessed at 24 months and compared to baseline.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||72 participants|
|Intervention Model:||Parallel Assignment|
|Intervention Model Description:||Randomized, double blind study|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Masking Description:||AT-007 and its matching placebo will have the same presentation, the same aspect and taste in order to be indistinguishable, and they will be supplied and used in the same conditions.|
|Official Title:||A RandomIzed, Double-Blind, Placebo-CoNtrolled, Two-Part Study to Evaluate the Pharmacodynamic EffIcacy and Clinical Benefit of AT 007 in Patients With SoRbitol Dehydrogenase (SORD) DEficiency|
|Actual Study Start Date :||January 1, 2022|
|Estimated Primary Completion Date :||July 31, 2024|
|Estimated Study Completion Date :||July 31, 2026|
Active Comparator: AT-007
AT-007 is an Aldose reductase inhibitor
AT-007, aldose reductase inhibitor
Other Name: Govorestat
Sham Comparator: Placebo
Is an non-active control
Liquid oral suspension
Other Name: Liquid oral vehicle suspension
- 10-meter walk-run test (10MWRT). [ Time Frame: baseline and up to month 24 ]The 10MWRT measures the time necessary to walk or run a distance of 10 meters by the study population
- blood sorbitol levels [ Time Frame: Baseline and up to month 24 ]
Patients with SORD Deficiency are unable to metabolize sorbitol. All patients with SORD Deficiency have highly elevated levels of blood sorbitol.
The measurement of Sorbitol will provide evidence of the efficacy of the treatment (AT-007) used in the study
- Muscle MRI (Magnetic Resonance Imaging) [ Time Frame: Baseline and up to month 24 month ]Patients will undergo MRI of their legs to evaluate the fat deposition and the muscle size.
- CMT-Fom (Charcot Marie Tooth Functional Outcome Measure) [ Time Frame: Baseline and up to month 24 ]The CMT-Fom is a performance-based assessment that measures the functional ability of patients with neuropathies
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05397665
|Contact: Riccardo Perfetti, MD, PhDemail@example.com|
|Contact: Michael E Shy, MDfirstname.lastname@example.org|
|United States, New Jersey|
|Hassman Research Institute||Recruiting|
|Berlin, New Jersey, United States, 08009|
|Contact: Michael Hassman, MD 856-753-7355 ext 316 email@example.com|
|Study Chair:||Michael E Shy, MD||University of Iowa|