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Amino Acid Metabolism, Inborn Errors clinical trials

The registry lists 15 studies for Amino Acid Metabolism, Inborn Errors across more than 10 countries. 7 are recruiting or about to start. 2 of them are mainly about Amino Acid Metabolism, Inborn Errors (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-05. Counting its 9 narrower conditions too, such as Phenylketonurias: 403 studies, 91 open.

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Status

Recruiting
6
Completed
5
Status unknown
1
Active, not recruiting
1
Not yet recruiting
1
Terminated
1

Phase

Phase 2
5
Phase 1
3
Phase 3
1

Where

United States
8
United Kingdom
6
France
4
Spain
3
Switzerland
3
Canada
3

Who runs them

ModernaTX, Inc. (2) · National Center for Research Resources (NCRR) (2) · iECURE, Inc. (2) · University Children's Hospital, Zurich (1) · PTC Therapeutics (1) · Food and Nutrition Research Institute, Philippines (1) · Brendan Lee (1) · Rigshospitalet, Denmark (1)

What is being tested here

Drugs under test in Amino Acid Metabolism, Inborn Errors studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted

DrugOpenPhase 3All
Phenylbutyric acid002
Arginine001
Sodium Benzoate001
Ataluren001

Coming up

1phase 3 study under way
2studies 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 2

Who these studies take

Of the 6 Amino Acid Metabolism, Inborn Errors studies recruiting now, 3 (50%) set no upper age limit and 3 (50%) stop at 75 or younger. 5 (83%) 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
104 (67%)4 (67%)
185 (83%)5 (83%)
304 (67%)4 (67%)
404 (67%)4 (67%)
504 (67%)4 (67%)
653 (50%)3 (50%)
753 (50%)3 (50%)
853 (50%)3 (50%)

Across all 15 studies, recruiting or not: 67% stop at 75 or younger and 60% at 65; 80% take children; 7% are for women only and 7% for men only; 27% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.

Studies

Recruiting first, then most recently updated. Showing 15 of 15. search them.