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Compares treatment options for MELAS Syndrome
Official title A Study to Assess TTI-0102 vs Placebo in MELAS Patients
ClinicalTrials.gov ID: NCT06644534
What this study is testing
What is TTI-0102?
TTI-0102 is an investigational medicine, given as an once-daily pill taken by mouth, being studied as a potential treatment for melas syndrome.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This is a randomized, double-blind, placebo-controlled study. Prior to treatment, patients will undergo a screening visit.
- Phase 2: a mid-size study of how well it works
- Time commitment: about 6 months
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 16 to 60
You may be able to join if
- Patient or Patient's legally designated representative has given written informed consent before any study-related activities are carried out and is...
- Males and females between 16 and 60 years of age at screening.
- Diagnosis of mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes (MELAS), defined as: \- mtDNA mutation known to be associated...
- Moderate disease severity defined as Newcastle Mitochondrial Disease Adult Scale (NMDAS) score between 15 to 45 inclusive.
- Able to complete a 12-minute walk test (12-MWT) distance of at least 150 meters and no more than 1000 meters within 30 days prior to, or at time of...
You likely can't join if
- Documented diagnosis of concurrent inborn errors of metabolism.
- Non-elective hospitalization related to their mitochondrial disease or direct complication of disease within 60 days prior to the Screening Visit.
- Overt comorbidity preventing them from safely performing an exercise. In particular, patients suffering from cardiovascular, neurological disorders...
- Treatment with taurine during the previous month, and not willing to discontinue for the duration of the trial.
- Platelet count, lymphocyte count or hemoglobin level below the lower limit of normal (LLN) at screening.
- Hepatic insufficiency with liver enzyme tests (alkaline phosphatase, AST or ALT) greater than 2.5 times to upper limit of normal (ULN) at screening.
See the full eligibility criteria
Who can join
- Patient or Patient's legally designated representative has given written informed consent before any study-related activities are carried out and is able to understand the full nature and purpose of the trial, including...
- Males and females between 16 and 60 years of age at screening.
- Diagnosis of mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes (MELAS), defined as: \- mtDNA mutation known to be associated with MELAS and MELAS phenotype (Emmanuele et al., 2022) including but...
- Moderate disease severity defined as Newcastle Mitochondrial Disease Adult Scale (NMDAS) score between 15 to 45 inclusive.
- Able to complete a 12-minute walk test (12-MWT) distance of at least 150 meters and no more than 1000 meters within 30 days prior to, or at time of screening.
- people regularly taking dietary supplements including but not limited to creatine, alpha-lipoic acid, CoQ10, B vitamins, levocarnitine shall have been taking them for at least 3 months pre-study and will agree to...
- With respect to concomitant medications, the subject must:
- Be willing to abstain from initiating new dietary supplements and non-prescribed medications, except as permitted by the Investigator throughout the study.
- Be on a stable dose of medications prescribed for seizure management and prevention. Stable dose in this context means unchanged for at least 30 days prior to the Screening Visit.
- Willing and able to comply with study drug dosing requirements, i.e., able to ingest study drug solution orally.
- Female participants:
- Must be of nonchildbearing potential (i.e., surgically sterilized [hysterectomy, bilateral salpingectomy, bilateral oophorectomy at least 6 weeks before the screening visit]) or postmenopausal (where postmenopausal is...
- If of childbearing potential, must agree not to donate ova, not to attempt to become pregnant and, if engaging in sexual intercourse with a male partner, must agree to the use of acceptable forms of highly effective...
- Male participants:
- Engaging in any sexual intercourse, including those who are infertile and do not produce sperm (e.g. post-vasectomy), must abstain from unprotected sex until the Study Exit visit.
- Must agree to abstain from sperm donation, and if engaging in sexual intercourse with a female of child bearing potential must agree to the use of an acceptable form of highly effective contraception (refer to protocol...
- Have suitable venous access for blood sampling.
- Be willing and able to comply with all study assessments and adhere to the protocol schedule and restrictions.
What rules you out
- Documented diagnosis of concurrent inborn errors of metabolism.
- Non-elective hospitalization related to their mitochondrial disease or direct complication of disease within 60 days prior to the Screening Visit.
- Overt comorbidity preventing them from safely performing an exercise. In particular, patients suffering from cardiovascular, neurological disorders (e.g. ataxia, sequel blindness from pseudostroke, peripheral...
- Treatment with taurine during the previous month, and not willing to discontinue for the duration of the trial.
- Platelet count, lymphocyte count or hemoglobin level below the lower limit of normal (LLN) at screening.
- Hepatic insufficiency with liver enzyme tests (alkaline phosphatase, AST or ALT) greater than 2.5 times to upper limit of normal (ULN) at screening.
- Bilirubin \> 1.2 g/dL at screening.
- Renal insufficiency, defined as 1) a requirement for chronic dialysis or 2) serum creatinine ≥1.2 mg/dl or creatinine clearance \<60 ml/min
- Severe gastrointestinal disease including gastroparesis.
- Presence or having sequelae of gastrointestinal, liver, kidney, or other conditions known to interfere with the absorption, distribution, metabolism, or excretion of drugs. Examples: malabsorption requiring TPN, chronic...
- Severe end-organ hypo-perfusion syndrome secondary to cardiac failure resulting in lactic acidosis.
- Patients with suspected elevated intracranial pressure, pseudotumor cerebri (PTC) and/or papilledema.
- History of angina, myocardial infarction, or cardiac surgery within 2 years prior to screening.
- History of drug or alcohol abuse.
- History of pancreatitis.
- Known or suspected hypersensitivity to cysteamine.
- Allergy to any medicine containing mercaptamine, penicillamine or known hypersensitivity to any of the study drug ingredients.
- Evidence of or verbal attestation of Helicobacter pylori infection, presently, or within the last 90 days prior to Screening.
- Use of any live vaccinations within 30 days prior to the first study drug administration except for the influenza vaccine (note that COVID-19 vaccine is permitted).
- For women of childbearing potential, a positive urine pregnancy test and confirmatory positive serum test at screening. Must not be currently breastfeeding.
- Donation of blood or plasma within 30 days prior to first study drug administration, or loss of whole blood of more than 500 mL within 30 days prior to first study drug administration, or receipt of a blood transfusion...
- Participation in another investigational clinical trial within 30 days if a drug, or 90 days for a biologic or device, prior to screening.
- Any other condition or prior therapy that in the opinion of the Investigator would make the subject unsuitable for this study, including inability to cooperate fully with the requirements of the study protocol or...
The study team makes the final eligibility decision.
Where it's taking place
- Angers, France
- Nijmegen, Netherlands
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 6 months per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 16 years to 60 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Angers, France; Nijmegen, Netherlands. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.