Clinical Trials

Multiple clinical trials, spanning Phase 1 through Phase 4 studies and observational registries, evaluate the therapeutic application of voxelotor. Research primarily targets sickle cell disease and associated complications—including pediatric sickle cell anemia, leg ulcers, and nephropathy—alongside secondary indications such as acute lung injury and end-stage lung disease. Sponsored by pharmaceutical companies like Pfizer and Novartis as well as academic institutions such as Duke University and Emory University, these studies encompass recruitment statuses ranging from active and recruiting to completed and terminated.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT06023199 COMPLETED
Sickle Cell Disease
Inova Health Care Services
 2023-10-23 PHASE2
NCT03573882 TERMINATED
Sickle Cell Disease
Pfizer
 2018-06-06 PHASE3
NCT04188509 TERMINATED
Sickle Cell Disease
Pfizer
 2019-11-18 PHASE3
NCT05018728 TERMINATED
Sickle Cell Anemia in Children
Emory University
 2022-03-28 PHASE2
NCT04335721 TERMINATED
Sickle Cell Disease; Sickle Cell Nephropathy
University of Illinois at Chicago
 2021-03-16 PHASE1; PHASE2
NCT05561140 TERMINATED
Sickle Cell Disease; Leg Ulcers
Pfizer
 2022-05-30 PHASE3
NCT05199766 UNKNOWN
Sickle Cell Disease
Assistance Publique - Hôpitaux de Paris
 2023-03-22 PHASE2
NCT04218084 TERMINATED
Sickle Cell Disease
Pfizer
 2020-11-11 PHASE3
NCT05981365 COMPLETED
Sickle Cell Disease
Pfizer
 2023-04-17 PHASE1
NCT02850406 TERMINATED
Sickle Cell Disease
Pfizer
 2016-07-05 PHASE2
NCT06023199 Recruiting
Sickle Cell Disease
Inova Health Care Services|Pfizer
 2023-10-23 Phase 2
NCT05228821 WITHDRAWN
Sickle Cell Disease
Pfizer
 2023-08-09 PHASE4
NCT05289570 TERMINATED
Acute Lung Injury; End Stage Lung Disease
Duke University
 2022-05-03 PHASE2
NCT05228834 TERMINATED
Sickle Cell Disease
Pfizer
 2022-06-24 PHASE3
NCT04400487 COMPLETED
Sickle Cell Disease; Sickle Cell Anemia
Pfizer
 2020-12-21 PHASE4
NCT05289570 Terminated
Acute Lung Injury|End Stage Lung Disease
Duke University
 2022-05-03 Phase 2
NCT05561140 Active not recruiting
Sickle Cell Disease|Leg Ulcers
Pfizer
 2022-05-30 Phase 3
NCT05018728 Recruiting
Sickle Cell Anemia in Children
Amy Tang|Pfizer|Emory University
 2022-03-28 Phase 2
NCT04581356 COMPLETED
Sickle Cell Anemia
Elizabeth Yang, MD, PhD
 2020-09-21 PHASE4
NCT05494541 Completed
Sickle Cell Disease
Novartis Pharmaceuticals|Novartis
 2021-08-30 --
NCT04247594 TERMINATED
Sickle Cell Disease
Pfizer
 2020-01-09 PHASE2
NCT03036813 COMPLETED
Sickle Cell Disease
Pfizer
 2016-12 PHASE3

(data from https://clinicaltrials.gov, updated on 2026-08-31)

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

Compliance for Clinical Use

Mechanism and Biochemical Profile

Voxelotor functions as a small-molecule hemoglobin modifier that selectively binds to the N-terminal alpha-subunit of sickle hemoglobin, stabilizing its oxygenated state and inhibiting deoxygenation-induced polymerization. By preventing hemoglobin polymerization, voxelotor reduces erythrocyte sickling and membrane damage, directly addressing the underlying pathogenesis in clinical trial conditions such as sickle cell disease, pediatric sickle cell anemia, and associated vascular complications.

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.