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Long-term vatiquinone treatment in Japanese patients with Leigh encephalomyelopathy (extension study)
jRCT2031200224Completed
- Sponsor
- PTC Therapeutics, Inc.
- Start
- Dec 2020
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Open-label, single-arm trial of vatiquinone in Leigh syndrome, sponsored by PTC Therapeutics, Inc. Primary endpoints: Safety: adverse events and adverse drug reactions, Safety: 12-lead electrocardiography and 11 more. Started Dec 2020; status: completed.
02
Drugs
vatiquinone
03
Conditions
Leigh syndrome
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