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Fabry Disease clinical trials

The registry lists 244 studies for Fabry Disease across more than 10 countries. 55 are recruiting or about to start. 213 of them are mainly about Fabry Disease (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-03.

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Status

Completed
120
Recruiting
45
Status unknown
28
Terminated
16
Active, not recruiting
13
Withdrawn
10

Phase

Phase 2
34
Phase 3
30
Phase 1
23
Phase 4
13
Early Phase 1
1

Where

United States
94
United Kingdom
52
France
43
Germany
32
Italy
31
Canada
30

Who runs them

Amicus Therapeutics (24) · Genzyme, a Sanofi Company (17) · Shire (12) · National Institute of Neurological Disorders and Stroke (NINDS) (11) · Chiesi Farmaceutici S.p.A. (8) · CENTOGENE GmbH Rostock (6) · Sanofi (6) · Protalix (6)

What is being tested here

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

DrugOpenPhase 3All
Migalastat21120
Pegunigalsidase alfa2810
Agalsidase alfa1518
Agalsidase beta1416
Venglustat024
Larazotide acetate003
Larazotide003
Paricalcitol001
Dapagliflozin001
Apabetalone001

Coming up

8phase 3 studies under way
12studies finish main data collection within a year2 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 45 Fabry Disease studies recruiting now, 32 (71%) set no upper age limit and 11 (24%) stop at 75 or younger. 20 (44%) take children under 18. 2 (4%) are for women only and 4 (9%) for men only; 7 (16%) take healthy volunteers.

Recruiting studies that take each age, of 45
AgeWomenMen
1016 (36%)14 (31%)
1838 (84%)40 (89%)
3037 (82%)39 (87%)
4037 (82%)39 (87%)
5037 (82%)39 (87%)
6538 (84%)38 (84%)
7535 (78%)34 (76%)
8532 (71%)31 (69%)

Across all 244 studies, recruiting or not: 37% stop at 75 or younger and 29% at 65; 43% take children; 3% are for women only and 16% for men only; 16% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.

How long Fabry Disease studies take

Of 22 phase 3 interventional studies under Fabry Disease, half reached primary completion within 41 months of starting, counting those still running.

Start to primary completionEvery sponsorIndustryNon-industry
Phase 222 mo20–24 · 11 studies——
Phase 341 mo31–64 · 22 studies——
Observational81 mo36–… · 61 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.

Started recruiting1

Newly registered2

Studies

Recruiting first, then most recently updated. Showing 50 of 244. Browse all 244 → or search them.