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Mitochondrial Diseases clinical trials

The registry lists 189 studies for Mitochondrial Diseases across more than 10 countries. 60 are recruiting or about to start. 83 of them are mainly about Mitochondrial Diseases (how this is decided). Per ClinicalTrials.gov, last updated 2026-10-28. Counting its 6 narrower conditions too, such as Friedreich Ataxia: 399 studies, 104 open.

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A study of drug A after surgery for early breast cancer

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Status

Completed
93
Recruiting
40
Not yet recruiting
13
Terminated
11
Status unknown
10
Active, not recruiting
5

Phase

Phase 2
36
Phase 1
19
Phase 3
16
Phase 4
8
Early Phase 1
1

Where

United States
83
France
25
United Kingdom
23
Italy
17
Germany
15
Denmark
12

Who runs them

Children's Hospital of Philadelphia (8) · Centre Hospitalier Universitaire de Nice (7) · GenSight Biologics (7) · Khondrion BV (6) · Columbia University (6) · Rigshospitalet, Denmark (5) · PTC Therapeutics (4) · Massachusetts General Hospital (4)

What is being tested here

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

DrugOpenPhase 3All
Coenzyme Q10125
Trolox115
Napazimone104
Idebenone112
Modafinil111
Tributyrin101
Deoxycytidine101
Thymidine101
Acetylcysteine101
Alpha-tocotrienol quinone016

Coming up

5phase 3 studies under way
18studies finish main data collection within a year1 of them phase 3

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

The 10 nearest, phase 3 first

Who these studies take

Of the 40 Mitochondrial Diseases studies recruiting now, 20 (50%) set no upper age limit and 13 (33%) stop at 75 or younger. 19 (48%) take children under 18. 3 (8%) are for women only and 0 (0%) for men only; 17 (43%) take healthy volunteers.

Recruiting studies that take each age, of 40
AgeWomenMen
1018 (45%)17 (43%)
1835 (88%)34 (85%)
3035 (88%)32 (80%)
4035 (88%)32 (80%)
5034 (85%)33 (83%)
6532 (80%)31 (78%)
7529 (73%)28 (70%)
8525 (63%)24 (60%)

Across all 189 studies, recruiting or not: 44% stop at 75 or younger and 36% at 65; 47% take children; 4% are for women only and 2% for men only; 33% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.

How long Mitochondrial Diseases studies take

Of 16 phase 2 interventional studies under Mitochondrial Diseases, half reached primary completion within 27 months of starting, counting those still running.

Start to primary completionEvery sponsorIndustryNon-industry
Phase 227 mo17–85 · 16 studies——
Interventional, no phase36 mo16–60 · 26 studies——
Observational169 mo26–… · 33 studies——

Each figure is the month by which half the studies had reached primary completion, with the range in which the middle half did. Studies still running count until their last registry update; studies that stopped early are left out. Phase 1 includes early phase 1; observational means prospective or not stated, as retrospective and cross-sectional studies date a window of data rather than a study. How these figures are worked out

What changed recently

From the registry's own updates since Oct 13, 2026.

Newly registered1

Studies

Recruiting first, then most recently updated. Showing 50 of 189. Browse all 189 → or search them.