Table of Contents

• Trial overview
• Who can participate
• What is being measured
• Trial design and phase
• Key patient terms
• What the record shows

Trial overview

The available clinical trial is studying GOVORESTAT in people with Sorbitol Dehydrogenase (SORD) Deficiency, a rare condition named in the study record.^[1] The trial is interventional, which means researchers gave a study drug or placebo and then measured the results.^[1]

The trial title describes it as a phase 2/3 study, while the record lists the phase as Phase 4.^[1] The study status is Completed and the enrollment was 72 participants.^[1]

Who can participate

The study focused on patients with SORD Deficiency who were 16 to 55 years old.^[1] This age range is important because it shows the group the researchers wanted to follow over time.^[1]

The trial data do not list other joining rules in detail, so the clearest known group is adults and older teens within that age range.^[1]

What is being measured

The main outcomes were blood sorbitol at Month 12 and the 10 m walk/run test (10MWRT) at Month 24.^[1] Blood sorbitol is a lab measure, while the walk/run test checks how well a person can move over a short distance.^[1]

The study brief also says the goal was to evaluate the effect of long-term treatment on clinical benefit and on blood sorbitol levels.^[1] In simple terms, the researchers wanted to see whether the study treatment could help with both body measurements and day-to-day physical function.^[1]

Trial design and phase

The trial compared GOVORESTAT with a placebo, which is a look-alike treatment without the active study drug.^[1] This type of design helps researchers compare outcomes more fairly.^[1]

The intervention listed in the record was oral use of AT-007 at 20 mg/kg and an oral placebo suspension.^[1] The source data use the name AT-007 in the intervention section, while the article topic uses GOVORESTAT as requested.^[1]

The phase information is mixed in the source: the title says phase 2/3, but the structured phase field says Phase 4.^[1] Because both are in the record, both are shown here without guessing which one is more important.^[1]

Key patient terms

Enrollment means the number of people who joined the study.^[1] In this trial, 72 people were enrolled.^[1]

Baseline means the starting point before changes are measured.^[1] The study compared results from baseline with Month 12 and Month 24.^[1]

Primary outcome means the main result the researchers planned to measure.^[1] Here, the main results were blood sorbitol and the walk/run test.^[1]

What the record shows

The trial was completed, so the study has already finished collecting the planned data.^[1] The available record does not provide final results, so this article focuses on the study design and what it was trying to measure.^[1]

Overall, the GOVORESTAT trial is centered on long-term follow-up in people with SORD Deficiency, with outcomes that combine lab testing and physical function.^[1] That makes it a study about both biological change and possible patient benefit.^[1]