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Friedreich Ataxia clinical trials

The registry lists 111 studies for Friedreich Ataxia across more than 10 countries. 28 are recruiting or about to start. 102 of them are mainly about Friedreich Ataxia (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-12.

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Status

Completed
66
Recruiting
18
Active, not recruiting
9
Not yet recruiting
7
Withdrawn
3
Enrolling by invitation
3

Phase

Phase 2
36
Phase 1
23
Phase 3
16
Phase 4
2
Early Phase 1
2

Where

United States
71
France
22
Italy
21
Germany
17
Australia
13
Spain
12

Who runs them

Larimar Therapeutics, Inc. (8) · PTC Therapeutics (6) · Children's Hospital of Philadelphia (6) · Biogen (6) · IRCCS Eugenio Medea (4) · Design Therapeutics, Inc. (4) · University of South Florida (4) · Santhera Pharmaceuticals (4)

What is being tested here

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

DrugOpenPhase 3All
Nomlabofusp216
Alpha-tocotrienol quinone136
GV101101
Idebenone047
Interferon gamma-1b034
Omaveloxolone012
Resveratrol002
Interferon Gamma002
Epoetin Alfa002
Deferiprone002

Coming up

4phase 3 studies under way
12studies 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 18 Friedreich Ataxia studies recruiting now, 9 (50%) set no upper age limit and 9 (50%) stop at 75 or younger. 15 (83%) take children under 18. 1 (6%) are for women only and 0 (0%) for men only; 3 (17%) take healthy volunteers.

Recruiting studies that take each age, of 18
AgeWomenMen
108 (44%)8 (44%)
1818 (100%)17 (94%)
3018 (100%)17 (94%)
4018 (100%)17 (94%)
5016 (89%)15 (83%)
6512 (67%)11 (61%)
759 (50%)8 (44%)
859 (50%)8 (44%)

Across all 111 studies, recruiting or not: 60% stop at 75 or younger and 56% at 65; 64% take children; 2% are for women only and 1% for men only; 15% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.

How long Friedreich Ataxia studies take

Of 12 phase 2 interventional studies under Friedreich Ataxia, half reached primary completion within 22 months of starting, counting those still running.

Start to primary completionEvery sponsorIndustryNon-industry
Phase 222 mo14–34 · 12 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 14, 2026.

Newly registered2

Studies

Recruiting first, then most recently updated. Showing 50 of 111. Browse all 111 → or search them.