Table of contents
- Overview of the clinical research
- Study design and phase
- Who took part in the trial
- What the trial measured
- Trial status and size
- Patient-friendly explanation of key terms
Overview of the clinical research
The available study investigated Mercaptamine-Pantetheine Disulfide Acetate in people with mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes (MELAS).[1] The main goal was to see whether the oral treatment could improve symptoms and function while also checking safety and tolerability.[1]
This trial was designed to compare the treatment with placebo, which is a look-alike control treatment with no active drug, so the results could be tested more fairly.[1]
Study design and phase
The study was a multi-center, randomized, double-blind, placebo-controlled trial.[1] Multi-center means it was carried out at more than one site, randomized means participants were assigned by chance, and double-blind means neither the participants nor the study team knew who received the treatment or placebo during the study.[1]
This was a Phase 2 study.[1] Phase 2 trials usually focus on early evidence of benefit and continue to collect important safety information in a small group of patients.[1]
Who took part in the trial
The study included patients with MELAS, a rare mitochondrial disease.[1] The trial enrolled 12 participants in total.[1]
The intervention listed in the trial record was oral TTI-0102, with placebo control using Pearlitol® 100 SD (mannitol).[1] The study summary states that the treatment was given for up to 6 months.[1]
What the trial measured
The primary outcome was change in functional capacity, measured by the 12-minute walking test (12-MWT) from baseline to Week 24 or study exit.[1] Functional capacity means how well a person can perform physical activity in daily life.[1]
The study also measured safety and tolerability outcomes, including the number, severity, and relationship of adverse events to the study treatment.[1] In addition, the trial tracked changes in clinical laboratory tests, physical examination findings, vital signs, 12-lead electrocardiography, and seizure activity.[1]
Another stated objective was to assess pharmacokinetics and pharmacodynamics.[1] Pharmacokinetics describes how the body handles a treatment, while pharmacodynamics describes the treatment’s effects on the body.[1]
Trial status and size
The trial record lists the study as Completed.[1] Because the enrollment was small, this study provides limited but important early clinical information about Mercaptamine-Pantetheine Disulfide Acetate in MELAS.[1]
Patient-friendly explanation of key terms
Randomized: people are put into study groups by chance, which helps make the comparison more reliable.[1]
Double-blind: the participants and the study team do not know who gets the active treatment or the placebo during the trial.[1]
Placebo-controlled: the treatment is compared with a placebo so researchers can see whether the study drug makes a difference.[1]
Adverse events: unwanted medical problems that happen during a study, whether or not they are caused by the treatment.[1]
Electrocardiography: a test that records the heart’s electrical activity.[1]
Seizure activity: signs or episodes of seizures that the study team watches for during treatment.[1]
