Clinical Trials

Multiple clinical trials sponsored by Vifor (International) Inc. have evaluated VIT-2763 in Phase 2 studies for hematologic disorders, including beta-thalassemia, non-transfusion-dependent thalassemia, and sickle cell disease. Several Phase 2a clinical trials assessing the safety, tolerability, and pharmacodynamics of VIT-2763 in non-transfusion-dependent thalassemia and sickle cell disease have been completed. In contrast, a Phase 2b clinical trial examining multiple doses in adult patients with beta-thalassemia was withdrawn.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT04817670 COMPLETED
Sickle Cell Disease
Vifor (International) Inc.
 2021-11-18 PHASE2
NCT04938635 WITHDRAWN
Beta-Thalassemia
Vifor (International) Inc.
 2021-09-20 PHASE2
NCT04364269 COMPLETED
Beta-Thalassemia; Non-transfusion-dependent Thalassemia
Vifor (International) Inc.
 2020-06-11 PHASE2

(data from https://clinicaltrials.gov, updated on 2025-02-18)

Check the VIT-2763 product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

VIT-2763 functions as an oral small-molecule inhibitor of ferroportin that displaces hepcidin with an IC50 of 24 nM, thereby blocking regulated iron export across cell membranes. By normalizing cellular iron transport and reducing organ iron loading, this targeted inhibition restores iron balance and enhances erythropoiesis, providing clinical therapeutic potential for treating anemia in conditions such as beta-thalassemia and sickle cell disease.

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.