The main criteria for participation
The clinical trial is recruiting 12 patients aged 16 to 60 years in France and the Netherlands, with a moderate form of MELAS syndrome, with at least two symptoms (diabetes, myopathy, seizures, etc.) and able to walk at least 150 meters and a maximum of 1,000 meters in 12 minutes. Eight patients will receive the treatment and four will receive the placebo.
The objective
This trial evaluates the efficacy, safety, and tolerability of orally administered TTI-0102 compared to placebo. It is a randomized, double-blind study: patients are randomly assigned to the treatment group and the placebo group, and neither the patient nor the medical team knows who is receiving what. This is a common step before being able to offer a treatment to all patients, subject, of course, to its safety and efficacy.
In practice
The trial lasts six months.
During the first week, the patient takes a half-dose of the drug candidate or placebo daily, and if all goes well, they will then receive the normal dose.
During the first two months, follow-up alternates between two hospital visits and two phone calls from the medical team. For the following four months, only monthly hospital visits will be maintained. The first and last visits will assess walking ability, fatigue, and quality of life. At the end of the six-month follow-up, any patient who wishes to receive the treatment can do so.
The study is being conducted at the Angers University Hospital.
Where are we?
Recruitment is underway in France.
Contacts :
Thiogenesis Therapeutics Laboratory