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VLCAD deficiency clinical trials

The registry lists 26 studies for VLCAD deficiency across more than 10 countries. 6 are recruiting or about to start. 17 of them are mainly about VLCAD deficiency (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-02.

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A study of drug A after surgery for early breast cancer

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· Sites: 4 added, including Seoul and Busan

· Enrollment: 300 → 450 participants

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Status

Completed
12
Recruiting
4
Active, not recruiting
3
Terminated
2
Status unknown
2
Available
1

Phase

Phase 2
8
Phase 1
2
Early Phase 1
1
Phase 3
1

Where

United States
12
Denmark
3
Germany
3
Italy
2
Austria
2
Spain
2

Who runs them

Ultragenyx Pharmaceutical Inc (6) · Oregon Health and Science University (4) · Minovia Therapeutics Ltd. (2) · Rigshospitalet, Denmark (2) · Children's National Research Institute (1) · University Hospital Freiburg (1) · LMU Klinikum (1) · Assistance Publique - Hôpitaux de Paris (1)

What is being tested here

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

DrugOpenPhase 3All
Triheptanoin014
Soybean oil, phospholipid emulsion001
Heparin001
(R)-3-hydroxybutyl (R)-3-hydroxybutyrate001
Bezafibrate001
Alpha-tocotrienol quinone001

Coming up

1phase 3 study under way
2studies 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 2, phase 3 first

Who these studies take

Of the 4 VLCAD deficiency studies recruiting now, 1 (25%) set no upper age limit and 3 (75%) stop at 75 or younger. 4 (100%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 1 (25%) take healthy volunteers.

Recruiting studies that take each age, of 4
AgeWomenMen
104 (100%)4 (100%)
184 (100%)4 (100%)
301 (25%)1 (25%)
401 (25%)1 (25%)
501 (25%)1 (25%)
651 (25%)1 (25%)
751 (25%)1 (25%)
851 (25%)1 (25%)

Across all 26 studies, recruiting or not: 46% stop at 75 or younger and 42% at 65; 85% take children; 4% are for women only and 0% for men only; 19% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.

What changed recently

From the registry's own updates since Oct 13, 2026.

Newly registered1

Studies

Recruiting first, then most recently updated. Showing 26 of 26. search them.