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Phenylketonurias clinical trials

The registry lists 191 studies for Phenylketonurias across more than 10 countries. 40 are recruiting or about to start. 182 of them are mainly about Phenylketonurias (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-10.

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Status

Completed
112
Recruiting
31
Terminated
18
Active, not recruiting
8
Status unknown
6
Withdrawn
6

Phase

Phase 2
27
Phase 1
25
Phase 3
19
Phase 4
9
Early Phase 1
3

Where

United States
93
United Kingdom
35
France
17
Germany
17
Italy
13
Canada
13

Who runs them

BioMarin Pharmaceutical (35) · Vitaflo International, Ltd (11) · Nutricia UK Ltd (6) · Emory University (6) · Otsuka Pharmaceutical Development & Commercialization, Inc. (6) · University Hospital, Tours (5) · University of Missouri-Columbia (5) · Boston Children's Hospital (5)

What is being tested here

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

DrugOpenPhase 3All
Repinatrabit426
Pegvaliase1410
Sepiapterin134
Methotrexate101
Sapropterin0927
Carnitine001
Carbohydrates001
Proteins001
Caseins001
Amino Acids001

Coming up

4phase 3 studies under way
17studies 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 31 Phenylketonurias studies recruiting now, 14 (45%) set no upper age limit and 17 (55%) stop at 75 or younger. 15 (48%) take children under 18. 1 (3%) are for women only and 0 (0%) for men only; 8 (26%) take healthy volunteers.

Recruiting studies that take each age, of 31
AgeWomenMen
1012 (39%)12 (39%)
1826 (84%)25 (81%)
3026 (84%)25 (81%)
4026 (84%)25 (81%)
5025 (81%)24 (77%)
6519 (61%)18 (58%)
7515 (48%)14 (45%)
8514 (45%)13 (42%)

Across all 191 studies, recruiting or not: 56% stop at 75 or younger and 50% at 65; 61% take children; 3% are for women only and 2% for men only; 24% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.

How long Phenylketonurias studies take

Of 33 interventional studies with no phase under Phenylketonurias, half reached primary completion within 22 months of starting, counting those still running.

Start to primary completionEvery sponsorIndustryNon-industry
Interventional, no phase22 mo9–28 · 33 studies——
Observational45 mo17–95 · 22 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 registered1

Studies

Recruiting first, then most recently updated. Showing 50 of 191. Browse all 191 → or search them.