Can a new oral drug help tame leigh syndrome?
NCT ID NCT06990984
First seen Jul 24, 2026 · Last updated Jul 24, 2026
Summary
This trial tests an experimental oral drug, TTI-0102, in people aged 5 to 55 with Leigh syndrome spectrum, a rare genetic disorder that damages the brain and nerves. The study aims to find the right dose and check safety over 12 weeks of twice-daily treatment. Participants will have regular clinic visits for blood tests and monitoring.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- an oral drug called TTI-0102 (cysteamine-pantetheine disulfide)
- What this could lead to
- If successful, this could point toward a treatment to slow or manage Leigh syndrome, a rare and severe neurological disorder.
- What could go wrong
- This is an early, small trial with only 18 participants, focused on safety and dosing—not yet testing effectiveness. Side effects are possible.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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