Orotic acid (6-Carboxyuracil) Clinical Trials

Size	Price	Qty
100mg	USD 147

Clinical Trials

Several clinical trials evaluate therapeutic interventions for conditions associated with pyrimidine metabolism, specifically focusing on hereditary orotic aciduria and broader inborn errors of metabolism. Encompassing Phase 2 and Phase 3 designs, these studies include both completed trials and those with unknown recruitment statuses. Funding has been provided by industry sponsors, such as Wellstat Therapeutics, alongside governmental and academic institutions like the Eunice Kennedy Shriver National Institute of Child Health and Human Development.

NCT Number	Recruitment	Conditions	Sponsor/Collaborators	Start Date	Phases
NCT02110147	Completed	Hereditary Orotic Aciduria	Wellstat Therapeutics	2014-04	Phase 3
NCT00843921	Unknown status	Inborn Errors of Metabolism	Mendel Tuchman\|Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)\|Children''s National Research Institute	2008-08	Phase 2\|Phase 3

(data from https://clinicaltrials.gov, updated on 2024-05-22)

Check the Orotic acid (6-Carboxyuracil) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Orotic acid functions as a key precursor in the de novo pyrimidine biosynthetic pathway by serving as a substrate for orotate phosphoribosyltransferase to generate orotidine 5'-monophosphate. Disruption or enzymatic deficiencies within this metabolic pathway impair cellular nucleic acid synthesis and alter nucleotide pools, directly underlying metabolic dysfunctions observed in conditions such as hereditary orotic aciduria and related inborn errors of metabolism.

Appendix RUO and cGMP Quality Standards

Quality Dimension	RUO (Research Use Only)	cGMP (Current Good Manufacturing Practice)
Clinical Applicability	Prohibited in human clinical trials or medical diagnostics.	Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status	Non-regulated grade; exempt from drug manufacturing laws.	Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment	Unclassified analytical or research laboratories.	Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control	Basic purity and activity testing.	Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation	Basic equipment calibration; no process validation required.	Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance	Vendor self-declared without required formal QMS.	Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact	High risk of IND rejection if used as a critical raw material.	Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).