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Ornithine Carbamoyltransferase Deficiency Disease clinical trials

The registry lists 59 studies for Ornithine Carbamoyltransferase Deficiency Disease across more than 10 countries. 13 are recruiting or about to start. 30 of them are mainly about Ornithine Carbamoyltransferase Deficiency Disease (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-05.

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Status

Completed
25
Terminated
10
Recruiting
10
Active, not recruiting
7
Not yet recruiting
3
Withdrawn
2

Phase

Phase 1
13
Phase 2
12
Phase 3
4
Phase 4
1

Where

United States
28
United Kingdom
10
France
9
Spain
9
Canada
6
Italy
6

Who runs them

Ultragenyx Pharmaceutical Inc (6) · Arcturus Therapeutics, Inc. (4) · Assiut University (3) · Baylor College of Medicine (2) · Abbott Medical Devices (2) · iECURE, Inc. (2) · Andrea Gropman (2) · Korro Bio, Inc. (1)

Who develops what

Save this set

Every programme with a company-led study here in the last two years, whatever its size: 1 programme from 1 company, 0 with this as their studies’ main aim; 0 with a live phase 3 study; 1 primary completion in the next 12 months.

Comparator & background therapies

What other companies’ phase 2+ studies give as the comparator or the background here (its leading trial sponsor and partners left out): a sign of what new drugs are measured against, not by itself the standard of care, its prescriptions or its market.

None given by two companies or more yet.

Programmes

“Meets, by arms” reads each study’s arms: vs its arm against the therapy’s; + X vs a combination against it; added to it plus the therapy against the therapy; studied with given together only. The next primary completion is the next phase-3 study’s when there is one.

The 1 programme — sign in to see the whole list, free: filter by phase and aim, sort by late development, direct comparisons or the next primary completion.

Primary completion in the next 12 months (1)

Live phase 2 and 3 studies of these programmes. Primary completion is when the main measure’s data collection ends, as the registry states it (most are estimates); results come later, if they are announced.

  • Aug 2027 est.KRRO-121 · Korro Bio, Inc. · Phase 2 registry mapping onlyNCT07773246 Phase 1/2 Study of KRRO-121 in Healthy Volunteers and Patients With UCD — primary endpoint: Incidence of dose limiting toxicities (DLT) (Safety and Tolerability) · 30 enrolled

Read from ClinicalTrials.gov, industry-led studies. Main aim: this is the study’s judged main condition; named, not the main aim: the sponsor names it, the study is about something else; registry mapping only: only the registry’s mapping put it here. Leading trial sponsor is the company leading the most industry studies of a drug, a measure of trial activity, not of rights. US and EU rights, and Approved: from the regulators’ records (FDA’s Drugs@FDA via openFDA; EMA’s medicines data) — the holder of a marketed or authorised original, generics and other companies’ versions; not rights elsewhere, nor approval for this condition. Phase 4 trial recorded, where neither lists it: a phase 4 study exists somewhere. Not read yet: labels by indication, exclusivity, licences, line of therapy, academic comparators, sales.

What is being tested here

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

DrugOpenPhase 3All
ARCT-810104
Sodium Acetate011
Cytidine Diphosphate Choline011
Carglumic acid001
Fingolimod001

Coming up

2phase 3 studies under way
4studies 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.

All 4, phase 3 first

Who these studies take

Of the 10 Ornithine Carbamoyltransferase Deficiency Disease studies recruiting now, 3 (30%) set no upper age limit and 6 (60%) stop at 75 or younger. 7 (70%) take children under 18. 1 (10%) are for women only and 0 (0%) for men only; 3 (30%) take healthy volunteers.

Recruiting studies that take each age, of 10
AgeWomenMen
105 (50%)4 (40%)
187 (70%)7 (70%)
305 (50%)5 (50%)
405 (50%)5 (50%)
505 (50%)5 (50%)
655 (50%)5 (50%)
754 (40%)4 (40%)
853 (30%)3 (30%)

Across all 59 studies, recruiting or not: 44% stop at 75 or younger and 42% at 65; 44% take children; 2% 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.

Studies

Recruiting first, then most recently updated. Showing 50 of 59. Browse all 59 → or search them.