Table of Contents
- Trial overview
- Study design and treatment groups
- Who could participate
- What was measured
- Why this trial matters
Trial overview
The available data describe one interventional study of Mercaptamine-Pantetheine Disulfide Acetate listed as oral TTI-0102 in the trial record.[1] It studied patients with mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes (MELAS), a rare condition that affects energy-making parts of cells and can cause brain and muscle problems.[1]
The study was Phase 2, which means it was an early clinical research stage focused on signs of benefit and continued safety testing in a small group of people.[1] The trial status was completed, and it enrolled 12 participants.[1]
Study design and treatment groups
This study was randomized, so people were assigned to a study group by chance rather than by choice.[1] It was also double-blind and placebo-controlled, meaning neither the participants nor the study team knew who received the study treatment and who received the placebo, which is a look-alike treatment with no active drug.[1]
The title says the study aimed to assess the efficacy, safety, tolerability, pharmacokinetics, and pharmacodynamics of oral TTI-0102 compared with placebo for up to 6 months in patients with MELAS.[1] In simple terms, the researchers wanted to see whether the treatment may help, whether it was acceptable to take, and how the body handled it and responded to it.[1]
The intervention list includes oral TTI-0102 and Pearlitol® 100 SD (mannitol), which was used as the placebo comparator in the source data.[1]
Who could participate
The trial targeted patients with MELAS.[1] The source data do not provide more detailed entry rules such as age limits, disease severity, or other medical requirements.[1]
Because the study was small, only a limited number of people could take part.[1] This kind of study is often used to learn more about a treatment in a carefully selected group before larger trials are done.[1]
What was measured
The main outcome was change in functional capacity, measured with the 12-minute walking test (12-MWT) from Day 1 or baseline to Week 24 or study exit.[1] Functional capacity means how well a person can do physical tasks in daily life, and the walking test is a simple way to measure that ability.[1]
Safety and tolerability were also measured.[1] The study tracked the number, severity, and relationship of adverse events, which are unwanted health problems that happen during a trial, whether or not they are caused by the study treatment.[1]
Other safety checks included changes in laboratory tests, physical exam findings, vital signs, 12-lead electrocardiography, and seizure activity.[1] A 12-lead ECG is a heart tracing test, and seizure activity refers to episodes of abnormal electrical activity in the brain.[1]
Why this trial matters
This trial focuses on a rare disease with serious symptoms, so even a small study can help researchers learn whether a treatment deserves further study.[1] The design used a placebo comparison and blinded assignment, which helps make the results more reliable.[1]
Because the study measured both walking ability and safety markers, it looked at whether the treatment might help patients function better while also checking for harm.[1] The completed Phase 2 design shows that the trial was meant to give early answers about possible benefit in people with MELAS.[1]
