Triheptanoin Clinical Trials

Size	Price	Qty
25mg	USD 97

Clinical Trials

Multiple clinical trials in Phase 2 and Phase 3, sponsored by academic research institutions and pharmaceutical sponsors, have evaluated triheptanoin across metabolic energy disorders including long-chain fatty acid oxidation disorders, Rett syndrome, and Glycogen Storage Disease Type V. These investigations assess its therapeutic utility in specific enzymatic deficiencies—such as carnitine palmitoyltransferase, very long-chain acyl-CoA dehydrogenase, and trifunctional protein deficiencies—by evaluating its capacity as an anaplerotic substrate to restore cellular energy homeostasis.

NCT Number	Recruitment	Conditions	Sponsor/Collaborators	Start Date	Phases
NCT05933200	Recruiting	Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)	Ultragenyx Pharmaceutical Inc	2023-02-28	Phase 3
NCT04632953	Recruiting	Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)	Ultragenyx Pharmaceutical Inc	2021-11-30	--
NCT03059160	Unknown status	Rett Syndrome	Sheba Medical Center\|Ultragenyx Pharmaceutical Inc	2017-04-01	Phase 2
NCT02919631	Unknown status	Glycogen Storage Disease Type V	Institut National de la Santé Et de la Recherche Médicale France\|Rigshospitalet Denmark	2016-10	Phase 2
NCT02432768	Completed	Glycogen Storage Disease Type V	Rigshospitalet Denmark\|Groupe Hospitalier Pitie-Salpetriere\|University of Texas Southwestern Medical Center\|Ultragenyx Pharmaceutical Inc	2015-04	Phase 2
NCT02214160	Completed	Carnitine Palmitoyltransferase (CPT I or CPT II) Deficiency\|Very Long Chain Acyl-CoA Dehydrogenase (VLCAD) Deficiency\|Long-chain 3-hydroxy-acyl-CoA Dehydrogenase (LCHAD) Deficiency\|Trifunctional Protein (TFP) Deficiency\|Carnitine-acylcarnitine Translocase (CACT) Deficiency	Ultragenyx Pharmaceutical Inc	2014-12-09	Phase 2

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

Check the Triheptanoin product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Triheptanoin functions as an anaplerotic precursor that hydrolyzes into odd-chain heptanoate fatty acids, bypassing impaired enzymatic steps to directly resupply acetyl-CoA and propionyl-CoA to the tricarboxylic acid cycle. This replenishment of key metabolic intermediates restores mitochondrial energy output, mitigating bioenergetic failure in long-chain fatty acid oxidation disorders and related metabolic conditions.

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>).