Actively Recruiting
Study to Assess Oral TTI-0102 Versus Placebo in Patients With MELAS Syndrome
Led by Thiogenesis Therapeutics, Inc. · Updated on 2025-09-09
12
Participants Needed
2
Research Sites
8 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the effects of oral TTI-0102 compared to placebo in patients diagnosed with MELAS syndrome, a condition characterized by mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes. This randomized, double-blind, placebo-controlled study aims to assess the efficacy, safety, tolerability, pharmacokinetics, and pharmacodynamics of TTI-0102 over a treatment period of up to six months in patients aged 16 to 60 years with moderate disease severity. Participants will be randomly assigned to receive either TTI-0102 or a placebo. Treatment begins with a half dose of 2.75 grams once daily for the first week to monitor tolerance, followed by a full dose of 5.5 grams once daily for the remaining weeks. The study includes an initial screening visit, a Day 1 dosing visit, weekly visits during the first week, and alternating clinic visits and phone calls over the first eight weeks. Monthly clinic visits continue from Week 12 through Week 20, with a Study Exit visit at Week 24. Participants may opt to continue in an open-label extension study after completing the initial 24-week period. Throughout the study, participants will undergo various assessments including a 12-minute walk test, safety evaluations, and blood sampling for pharmacokinetic and pharmacodynamic analyses. The primary outcomes measured are changes in functional capacity and the incidence of treatment-emergent adverse events from baseline to Week 24. Secondary outcomes include fatigue levels, quality of life, and pharmacokinetic parameters like peak concentration and exposure. Safety and efficacy will be closely monitored with interim data reviews to guide study continuation.
CONDITIONS
Brief Title
A Study to Assess TTI-0102 vs Placebo in MELAS Patients
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patient or legally authorized representative has given written informed consent and understands the study risks and purpose
- Male or female aged between 16 and 60 years at screening
- Diagnosis of MELAS with specific mtDNA mutations and at least two related symptoms such as diabetes or stroke-like episodes
- Moderate disease severity with a Newcastle Mitochondrial Disease Adult Scale score between 15 and 45
- Able to complete a 12-minute walk test covering at least 150 meters
- Regular use of certain dietary supplements for at least 3 months prior and agreement to continue during the study
- Stable dose of prescribed seizure medications for at least 30 days before screening
- Willing and able to take the study drug orally as required
- Female participants must be nonchildbearing or agree to effective contraception if of childbearing potential
- Male participants must agree to abstain from sperm donation and use contraception if sexually active with females of childbearing potential
- Suitable venous access for blood sampling
- Willing and able to comply with all study assessments and protocol requirements
You will not qualify if you...
- Diagnosis of other inborn metabolic errors
- Recent hospitalization related to mitochondrial disease within 60 days prior to screening
- Comorbidities preventing safe exercise such as cardiovascular or neurological disorders
- Treatment with taurine in the prior month without willingness to discontinue
- Low blood counts (platelets, lymphocytes, hemoglobin) below normal limits
- Liver enzyme levels greater than 2.5 times upper limit of normal
- High bilirubin levels above 1.2 g/dL
- Renal insufficiency requiring dialysis or with poor kidney function
- Severe gastrointestinal diseases like gastroparesis
- Conditions affecting drug absorption or metabolism
- Severe heart failure causing lactic acidosis
- Suspected increased intracranial pressure or similar conditions
- Recent heart disease or surgery within 2 years
- History of drug or alcohol abuse
- History of pancreatitis
- Known allergy or hypersensitivity to cysteamine or study drug ingredients
- Recent or current Helicobacter pylori infection
- Recent live vaccinations except flu and COVID-19
- Pregnancy or breastfeeding in women of childbearing potential
- Recent significant blood donation or transfusion
- Participation in another investigational trial within specified recent periods
- Any other condition or therapy that may interfere with safe study participation or compliance
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 24 weeks
Participants take the oral study drug TTI-0102 or placebo as assigned to treat MELAS syndrome.
Regular visits during treatment as scheduled by the study team
Trial Site Locations
Total: 2 locations
1
Angers University Hospital Center (CHU Angers)
Angers, France, 49100
Actively Recruiting
2
Radboud University Medical Center
Nijmegen, Netherlands, 6500 HB
Actively Recruiting
Research Team
T
TTI-MITO-001 Clinical Trial Recruitment
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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