Clinical Trials

Tocofersolan has been evaluated in several Phase 2 and Phase 3 clinical trials assessing its efficacy across metabolic and malabsorptive disorders, including vitamin E deficiency, cirrhosis, short bowel syndrome, and hypobetalipoproteinemias. Sponsored primarily by academic and medical institutions such as Boston Children's Hospital, University Hospital Geneva, and Hospices Civils de Lyon, overall recruitment statuses for these investigations range from completed studies examining absorption and acanthocytosis to withdrawn pediatric trials.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT03582384 WITHDRAWN
Short Bowel Syndrome; Vitamin E Deficiency
Boston Children's Hospital
 2020-12 PHASE2
NCT01457690 COMPLETED
Hypobetalipoproteinemias
Hospices Civils de Lyon
 2011-10 PHASE3
NCT01463735 COMPLETED
Cirrhosis
University Hospital, Geneva
 2011-11 PHASE2
NCT01463735 Completed
Cirrhosis
University Hospital Geneva
 2011-11 Phase 2

(data from https://clinicaltrials.gov, updated on 2020-05-13)

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

Compliance for Clinical Use

Mechanism and Biochemical Profile

Tocofersolan functions as an amphipathic, water-soluble derivative of vitamin E that incorporates into cellular lipid bilayers and neutralizes reactive oxygen species, thereby preventing lipid peroxidation and membrane degradation. By facilitating micellar solubilization and intestinal absorption of lipophilic antioxidants independently of bile secretion, it restores systemic vitamin E status to counteract oxidative cell injury in patients with vitamin E deficiency, cirrhosis, short bowel syndrome, and hypobetalipoproteinemias.

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>).

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Note: Technical data last updated: Sep 1, 2026.