OTC

Ornithine transcarbamylase, mitochondrial · P00480 · OTC on Sugi Atlas →

3,349 patent compounds predicted against OTC, 2,043 high-confidence (nearest known ligand ≥ 0.5)

Top predicted compounds (top 20 by supporting neighbours, then confidence)

Compound	Confidence	Supporting neighbours
SCHEMBL450041	0.63	1/20
SCHEMBL2237070	0.63	1/20
SCHEMBL2237074	0.63	1/20
SCHEMBL5548101	0.63	1/20
Arginine (SCHEMBL508141)	0.62	1/20
Arginine (SCHEMBL508142)	0.62	1/20
Medronic Acid (SCHEMBL367902)	0.61	1/20
SCHEMBL2236944	0.61	1/20
SCHEMBL2236948	0.61	1/20
SCHEMBL2236773	0.59	1/20
SCHEMBL2236779	0.59	1/20
SCHEMBL2237597	0.59	1/20
SCHEMBL2238970	0.59	1/20
SCHEMBL2238971	0.59	1/20
Arginine (SCHEMBL3784234)	0.59	1/20
L-Citrulline (SCHEMBL20587)	0.59	1/20
L-Citrulline (SCHEMBL20588)	0.59	1/20
L-Citrulline (SCHEMBL215403)	0.59	1/20
L-Citrulline (SCHEMBL288641)	0.59	1/20
L-Citrulline (SCHEMBL437142)	0.59	1/20

Clinical trials — most relevant to OTC by text similarity (a relevance ranking, not an exhaustive list)

Trial	Phase	Status
L-ornithine L-aspartate in Overt Hepatic Encephalopathy	PHASE4	COMPLETED
N-Carbamylglutamate (Carbaglu) In The Treatment Of Hyperammonemia	PHASE2/PHASE3	UNKNOWN
Efficacy of L-Ornithine-L-Aspartate in Cirrhotics With Hepatic Encephalopathy	PHASE3	COMPLETED
Halting Ornithine Transcarbamylase Deficiency With Recombinant AAV in ChildrEn	PHASE1/PHASE2	RECRUITING
A Phase I/II Open-label Safety and Efficacy Study of LNP.UCD.ABE in Patients With Urea Cycle Disorders.	PHASE1/PHASE2	NOT_YET_RECRUITING
Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency	PHASE1/PHASE2	COMPLETED
Safety, Tolerability and PK/PD Evaluation of Intravenous Administration of MRT5201 in Patients With OTC Deficiency	PHASE1/PHASE2	WITHDRAWN
Phase II Study of Sodium Phenylbutyrate, Sodium Benzoate, Sodium Phenylacetate, and Dietary Intervention for Urea Cycle Disorders	PHASE2	COMPLETED
Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate	PHASE2	WITHDRAWN
Metabolic Cofactor Supplementation in Alzheimer's Disease (AD) and Parkinson's Disease (PD) Patients	PHASE2	COMPLETED
The KHENERGY Study	PHASE2	COMPLETED
Study of Treatment and Metabolism in Patients With Urea Cycle Disorders	PHASE1	UNKNOWN
Valproic Acid in Childhood Progressive Brain Tumors	PHASE1	WITHDRAWN
Anaplerotic Therapy in Propionic Acidemia	PHASE1	COMPLETED
Trial of Oxaloacetate in Alzheimer's Disease (TOAD)	PHASE1	COMPLETED
Valproic Acid-associated Hypoalbuminemia in Medically Fragile Patients	nan	COMPLETED
PCORI Urea Cycle Disorder Study	nan	COMPLETED
Study in Healthy Subjects, Patients With Urea Cycle Disorders (UCD) and Carriers of UCD Mutations to Evaluate Urea Cycle Function	nan	COMPLETED
Longitudinal Study of Urea Cycle Disorders	nan	RECRUITING
Neuroimaging and Neuropsychological Outcomes in Urea Cycle Disorders	nan	RECRUITING
Gyrate Atrophy Ocular and Systemic Study	nan	ACTIVE_NOT_RECRUITING
Nitric Oxide Supplementation on Neurocognitive Functions in Patients With ASLD	nan	COMPLETED
Neurologic Injuries in Adults With Urea Cycle Disorders	nan	COMPLETED
Effects of Parenteral L-carnitine Supplementation in Premature Neonates	nan	COMPLETED
At Home Ammonia Monitoring of Inborn Errors of Ammonia Metabolism	nan	RECRUITING
Multi-omics Study in Citrin Deficiency	nan	ACTIVE_NOT_RECRUITING
The Effect of Arginine and Citrulline Supplementation on Endothelial Dysfunction in Mitochondrial Diseases	nan	COMPLETED
Orphan Drugs for Inherited Metabolic Diseases	nan	COMPLETED
MNGIE Natural History Study	nan	RECRUITING
Metabolism of Ornithine Phenylacetate	nan	COMPLETED

Related proteins — ESM-2 sequence neighbours (a OTC binder may also engage these)

Protein	Name	Similarity
P11725	—	1.000	P11725 →
P00481	—	1.000	P00481 →
P31326	—	1.000	P31326 →
P84010	—	1.000	P84010 →
Q9N1U7	—	0.999	Q9N1U7 →
Q9YHY9	—	0.998	Q9YHY9 →
P14995	—	0.992	P14995 →
Q0T9E0	—	0.991	Q0T9E0 →
P05150	—	0.991	P05150 →
Q72T26	—	0.991	Q72T26 →
P57876	—	0.991	P57876 →
Q0TUR4	—	0.991	Q0TUR4 →
Q7MZ18	—	0.991	Q7MZ18 →
Q8KDE2	—	0.991	Q8KDE2 →
A8F7Z9	—	0.991	A8F7Z9 →
Q8F2D1	—	0.991	Q8F2D1 →
P31317	—	0.990	P31317 →
Q8CMW2	—	0.990	Q8CMW2 →
O19072	—	0.990	O19072 →
A7ZVD3	—	0.990	A7ZVD3 →
O53089	—	0.989	O53089 →
Q7VHJ0	—	0.989	Q7VHJ0 →
Q8GND4	—	0.989	Q8GND4 →
Q4FT48	—	0.989	Q4FT48 →
A5FQE1	—	0.989	A5FQE1 →
B3R5Y0	—	0.989	B3R5Y0 →
A6UYL2	—	0.988	A6UYL2 →
Q6FUD0	—	0.988	Q6FUD0 →
Q8BPC6	—	0.988	Q8BPC6 →
A3M663	—	0.988	A3M663 →