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Leigh Disease clinical trials

The registry lists 20 studies for Leigh Disease across more than 10 countries. 9 are recruiting or about to start. 11 of them are mainly about Leigh Disease (how this is decided). Per ClinicalTrials.gov, last updated 2026-10-28.

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· Sites: 4 added, including Seoul and Busan

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Status

Recruiting
6
Completed
5
Terminated
2
Withdrawn
2
Enrolling by invitation
2
Not yet recruiting
1

Phase

Phase 2
9
Phase 3
2
Phase 1
1

Where

United States
14
Italy
3
Japan
2
France
2
Poland
2
Germany
2

Who runs them

PTC Therapeutics (5) · Khondrion BV (2) · Amgen (2) · Columbia University (2) · LMU Klinikum (1) · Aadi Bioscience, Inc. (1) · Matthew Demczko (1) · Thiogenesis Therapeutics, Inc. (1)

What is being tested here

Drugs under test in Leigh Disease studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted

DrugOpenPhase 3All
Sirolimus101
Cysteamine-pantetheine disulfide101
Alpha-tocotrienol quinone024
Cysteamine002
Trolox002
Sirolimus Albumin-bound Nanoparticles001

Coming up

0phase 3 studies under way
3studies finish main data collection within a year

Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.

All 3

Who these studies take

Of the 6 Leigh Disease studies recruiting now, 4 (67%) set no upper age limit and 0 (0%) stop at 75 or younger. 6 (100%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 2 (33%) take healthy volunteers.

Recruiting studies that take each age, of 6
AgeWomenMen
106 (100%)6 (100%)
186 (100%)6 (100%)
306 (100%)6 (100%)
406 (100%)6 (100%)
506 (100%)6 (100%)
656 (100%)6 (100%)
756 (100%)6 (100%)
856 (100%)6 (100%)

Across all 20 studies, recruiting or not: 55% stop at 75 or younger and 55% at 65; 95% take children; 0% are for women only and 5% for men only; 20% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.

Studies

Recruiting first, then most recently updated. Showing 20 of 20. search them.