Actively Recruiting
A Study to Assess TTI-0102 vs Placebo in MELAS Patients
Led by Thiogenesis Therapeutics, Inc. · Updated on 2025-09-09
12
Participants Needed
2
Research Sites
59 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This is a randomized, double-blind, placebo-controlled study. Prior to treatment, patients will undergo a screening visit. If eligible, each subject will return for a Day 1 visit and will receive their first dose of investigational product (TTI-0102 or placebo). At the end of the first week of treatment, subjects will return for a Week 1/Day 8 study visit to assess study drug dosing/tolerance and instruct on dosing for the upcoming second week of treatment. For the first 8 weeks of treatment, subjects will alternate between returning to the clinic for detailed assessments (Weeks 4 and 8) and receiving a telephone call from the Investigator team to assess safety and TTI-0102 dose (Weeks 2 and 6) and the potential need for an immediate unscheduled study visit. After the first 8 weeks of treatment, subjects will continue to return to the clinic for monthly assessments at Weeks 12, 16, 20. The Study Exit visit will occur at Week 24, and subjects will be offered to continue on an open-label extension study of TTI-0102. If a subject does not complete the study, they will be asked to return for a Study Exit visit 4 weeks after last study drug dose. Primary Objective The primary objective of this study is to assess the efficacy, safety and tolerability of oral TTI 0102 compared to placebo, for up to 6 months in patients with MELAS. Secondary Objective The secondary objectives of this study are to assess the efficacy, pharmacokinetics (PK) and pharmacodynamics (PD) of cysteamine after oral administration of TTI-0102 at steady state, in patients with MELAS on a stable dose of TTI-0102. This is a randomized, double-blind, placebo-controlled study. Prior to treatment, patients will undergo a screening visit. If eligible, each subject will return for a Day 1 visit and will receive their first dose of investigational product (TTI-0102 or placebo). At the end of the first week of treatment, subjects will return for a Week 1/Day 8 study visit to assess study drug dosing/tolerance and instruct on dosing for the upcoming second week of treatment. For the first 8 weeks of treatment, subjects will alternate between returning to the clinic for detailed assessments (Weeks 4 and 8) and receiving a telephone call from the Investigator team to assess safety and TTI-0102 dose (Weeks 2 and 6) and the potential need for an immediate unscheduled study visit. After the first 8 weeks of treatment, subjects will continue to return to the clinic for monthly assessments at Weeks 12, 16, 20. The Study Exit visit will occur at Week 24, and subjects will be offered to continue on an open-label extension study of TTI-0102. If a subject does not complete the study, they will be asked to return for a Study Exit visit 4 weeks after last study drug dose. Study Drug Dosing To prevent any manifestation of intolerance at the initiation of drug treatment, only half a dose (2.75 grams) will be given once a day for the first week of treatment. During the following weeks of treatment, patients will be given a full dose of 5.5 grams once a day. Interim Data Review After nine (9) patients have completed three months of treatment (the Week 12 visit) an interim data cut will take place to assess safety and potential efficacy signals. Even if no indications of efficacy are detected at this early stage, the trial itself will not be terminated unless there is a serious safety concern (i.e., protocol-defined Stopping Criteria are met).
CONDITIONS
Official 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 trial risks and purpose
- Male or female between 16 and 60 years of age at screening
- Diagnosis of MELAS confirmed by mitochondrial DNA mutation with >50% heteroplasmy plus two or more clinical symptoms such as diabetes, myopathy, seizures, stroke-like episodes, or exercise intolerance
- Moderate disease severity with Newcastle Mitochondrial Disease Adult Scale score between 15 and 45
- Able to complete a 12-minute walk test distance between 150 and 1000 meters within 30 days prior to screening
- Taking dietary supplements regularly for at least 3 months before study and willing to continue during study
- Stable dose of seizure medications for at least 30 days prior to screening
- Willing and able to take study drug orally and comply with dosing requirements
- Female participants must be non-childbearing or use effective contraception if of childbearing potential
- Male participants must abstain from unprotected sex and sperm donation during the study
- 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 errors of metabolism
- Non-elective hospitalization related to mitochondrial disease within 60 days before screening
- Severe comorbidities preventing safe exercise, such as cardiovascular or neurological disorders
- Treatment with taurine in the previous month and unwillingness to discontinue
- Low platelet, lymphocyte, or hemoglobin levels below normal at screening
- Liver enzyme levels more than 2.5 times the upper normal limit or bilirubin >1.2 g/dL
- Renal insufficiency requiring dialysis or serum creatinine ≥1.2 mg/dL or creatinine clearance <60 ml/min
- Severe gastrointestinal diseases including gastroparesis or conditions affecting drug absorption
- Severe cardiac failure causing lactic acidosis
- Suspected elevated intracranial pressure or pseudotumor cerebri
- History of angina, heart attack, or cardiac surgery within 2 years prior to screening
- History of drug or alcohol abuse or pancreatitis
- Known hypersensitivity to cysteamine or study drug ingredients
- Current or recent Helicobacter pylori infection
- Use of live vaccines within 30 days prior to first study drug, except influenza and COVID-19 vaccines
- Positive pregnancy test or breastfeeding in women of childbearing potential
- Recent blood donation or transfusion within specified timeframes
- Participation in another investigational trial within 30 or 90 days prior to screening
- Any other condition making the subject unsuitable or likely noncompliant with study requirements
AI-Screening
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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
CONTACT
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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