In short
Clinical trials are investigating GOVORESTAT in people with Sorbitol Dehydrogenase (SORD) Deficiency. These studies look at whether GOVORESTAT can improve clinical benefit and measure changes in blood sorbitol and walking or running ability. The main target group is patients 16 to 55 years old.
Key points
- Clinical trials are studying GOVORESTAT in people with Sorbitol Dehydrogenase (SORD) Deficiency. The available trial is a completed interventional study with 72 participants. It includes patients 16 to 55 years old and measures blood sorbitol and performance on a 10-meter walk/run test. The study is described as Phase 4 in the trial record, while the title also calls it a phase 2/3 study. The main goal is to understand whether long-term treatment gives clinical benefit.
Trial overview
The available clinical trial is studying GOVORESTAT in people with Sorbitol Dehydrogenase (SORD) Deficiency, a rare condition named in the study record. The trial is interventional, which means researchers gave a study drug or placebo and then measured the results.
The trial title describes it as a phase 2/3 study, while the record lists the phase as Phase 4. The study status is Completed and the enrollment was 72 participants.
Who can participate
The study focused on patients with SORD Deficiency who were 16 to 55 years old. This age range is important because it shows the group the researchers wanted to follow over time.
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.
What is being measured
The main outcomes were blood sorbitol at Month 12 and the 10 m walk/run test (10MWRT) at Month 24. Blood sorbitol is a lab measure, while the walk/run test checks how well a person can move over a short distance.
The study brief also says the goal was to evaluate the effect of long-term treatment on clinical benefit and on blood sorbitol levels. In simple terms, the researchers wanted to see whether the study treatment could help with both body measurements and day-to-day physical function.
Trial design and phase
The trial compared GOVORESTAT with a placebo, which is a look-alike treatment without the active study drug. This type of design helps researchers compare outcomes more fairly.
The intervention listed in the record was oral use of AT-007 at 20 mg/kg and an oral placebo suspension. The source data use the name AT-007 in the intervention section, while the article topic uses GOVORESTAT as requested.
The phase information is mixed in the source: the title says phase 2/3, but the structured phase field says Phase 4. Because both are in the record, both are shown here without guessing which one is more important.
Key patient terms
Enrollment means the number of people who joined the study. In this trial, 72 people were enrolled.
Baseline means the starting point before changes are measured. The study compared results from baseline with Month 12 and Month 24.
Primary outcome means the main result the researchers planned to measure. Here, the main results were blood sorbitol and the walk/run test.
What the record shows
The trial was completed, so the study has already finished collecting the planned data. The available record does not provide final results, so this article focuses on the study design and what it was trying to measure.
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. That makes it a study about both biological change and possible patient benefit.