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Study on the Effects of TTI-0102 for Patients with MELAS Syndrome

Verified siteInvestigationalNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a rare condition known as Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like episodes (MELAS). This is a genetic disorder that affects the energy-producing structures in cells, leading to symptoms like muscle weakness, seizures, and stroke-like episodes. The study will test a new treatment called TTI-0102, which is an oral solution. TTI-0102 is a prodrug derivative, meaning it is a modified version of an existing medication called Cystagon®, which is used to treat similar conditions.

The purpose of the study is to evaluate how effective and safe TTI-0102 is for people with MELAS. Participants in the study will be randomly assigned to receive either TTI-0102 or a placebo, which is a substance with no active medication. The study will last for up to six months, during which participants will take the medication orally. Throughout the study, participants will have regular check-ups to monitor their health and any changes in their condition. These check-ups will include tests to measure how well they can walk over a 12-minute period, as well as assessments of their overall quality of life and fatigue levels.

In addition to monitoring the main symptoms of MELAS, the study will also look at how the body processes TTI-0102 by measuring levels of certain substances in the blood, such as cysteamine and pantothenic acid (vitamin B5). This will help researchers understand how the drug works in the body. The study aims to provide valuable information on whether TTI-0102 can be a safe and effective treatment option for people living with MELAS.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial visit

    Upon joining the study, an initial visit is conducted to confirm eligibility. This involves a review of medical history and a physical examination.

    Blood samples are taken to ensure suitable venous access and to check for specific genetic markers associated with mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes (MELAS).

  2. Step 2

    Baseline assessments

    Baseline assessments are performed, including a 12-minute walk test (12-MWT) to measure functional capacity. This test involves walking as far as possible in 12 minutes.

    Additional assessments include the Fatigue Severity Scale (FSS) and the Quality of Life questionnaire (WHOQOL-BREF).

  3. Step 3

    Medication administration

    Participants are randomly assigned to receive either the study medication TTI-0102 or a placebo. The medication is administered as an oral solution.

    The dosage and frequency of administration are determined by the study protocol and are followed for up to 6 months.

  4. Step 4

    Regular follow-up visits

    Regular follow-up visits occur at Weeks 4, 8, 12, 16, and 20. These visits include assessments of safety, tolerability, and efficacy.

    Blood samples are collected to monitor pharmacokinetic (PK) and pharmacodynamic (PD) parameters, including levels of certain biomarkers.

  5. Step 5

    Study exit visit

    At Week 24, a final study exit visit is conducted. This includes a repeat of the 12-MWT and other assessments to evaluate changes in functional capacity.

    Participants are monitored for any adverse events and changes in clinical laboratory parameters.

Who can join the trial?

12 criteria

  • The patient or their legally designated representative must provide written informed consent before any study-related activities begin. This means they understand the purpose of the trial and any possible risks.
  • Male participants must agree to avoid unprotected sex until the end of the study and not donate sperm. If they have a female partner who can become pregnant, they must use a highly effective form of birth control.
  • The patient must have suitable veins for taking blood samples.
  • The patient must be willing and able to follow all study assessments and adhere to the study schedule and rules.
  • The patient must be between 16 and 60 years old at the time of screening.
  • The patient must have a diagnosis of mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes (MELAS). This includes having a specific genetic mutation associated with MELAS and showing two or more symptoms like diabetes, muscle weakness, seizures, a history of stroke-like episodes, or difficulty with exercise.
  • The patient must have a moderate level of disease severity, measured by a specific scale called the Newcastle Mitochondrial Disease Adult Scale (NMDAS), with a score between 15 and 45.
  • The patient must be able to walk at least 150 meters but no more than 1000 meters in a 12-minute walk test within 30 days before or at the time of screening.
  • If the patient is taking dietary supplements like creatine, alpha-lipoic acid, CoQ10, B vitamins, or levocarnitine, they must have been taking them for at least 3 months before the study and agree to continue taking them during the study.
  • The patient must agree not to start any new dietary supplements or non-prescribed medications during the study, unless allowed by the study doctor. If they are taking medication for seizures, the dose must have been stable for at least 30 days before the screening.
  • The patient must be willing and able to take the study medication by mouth.
  • Female participants must either be unable to have children (due to surgery or menopause) or, if they can have children, agree not to donate eggs, not try to become pregnant, and use highly effective birth control if having sex with a male partner.

Who cannot join the trial?

9 criteria

  • Patients who have any other serious health conditions that could interfere with the study.
  • Patients who are currently participating in another clinical trial.
  • Patients who have had a recent surgery or are planning to have surgery during the study period.
  • Patients who are pregnant or breastfeeding.
  • Patients who have a history of allergic reactions to the study medication or similar medications.
  • Patients who are unable to follow the study procedures or take the study medication as directed.
  • Patients who have a history of substance abuse or alcohol dependency.
  • Patients who have a mental health condition that could affect their ability to participate in the study.
  • Patients who have a condition that affects their ability to understand or communicate effectively.
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Investigated drugs

TTI-0102 is an experimental medication being tested for its potential to help people with a condition called MELAS, which stands for mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes. This condition affects the energy-producing parts of cells, leading to various symptoms like muscle weakness, headaches, and seizures. The medication is taken by mouth and is being studied to see if it can improve the symptoms of MELAS, make patients feel better, and be safe to use over a period of up to six months. Researchers are also looking at how the body processes the medication and how it affects the body’s functions.

What is already known about the treatment

TTI-0102 – TTI-0102 is an oral medication currently being studied for its potential to treat patients with mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes (MELAS). This medication is in the clinical trial phase, where researchers are evaluating its effectiveness, safety, and how well patients tolerate it. TTI-0102 is designed to help manage the symptoms of MELAS, a rare genetic disorder that affects energy production in cells. The drug works at the molecular level by targeting specific pathways involved in energy metabolism, aiming to improve cellular function. It falls under the pharmacological classification of treatments for mitochondrial disorders.

Investigated diseases

Mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes (MELAS) – This is a genetic disorder that affects the mitochondria, which are the energy-producing structures in cells. It typically begins in childhood and is characterized by muscle weakness and pain, recurrent headaches, loss of appetite, vomiting, and seizures. As the disease progresses, individuals may experience stroke-like episodes that can lead to temporary muscle weakness or paralysis on one side of the body, altered consciousness, and vision abnormalities. These episodes can occur repeatedly and may result in cumulative brain damage. Lactic acidosis, a buildup of lactic acid in the body, is also common and can cause nausea, vomiting, and extreme fatigue. Over time, MELAS can lead to a decline in cognitive function and other neurological impairments.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IITrial ID2023-506723-28-00Protocol codeTTI-MITO-001Estimated enrolment12 patientsSponsorThiogenesis Therapeutics

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

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