Trial reportBritish journal of clinical pharmacology2019
Pharmacokinetics and pharmacodynamics of voxelotor (GBT440) in healthy adults and patients with sickle cell disease.
Trial report in British journal of clinical pharmacology, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 53 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
53 citing papers in PubMed, 97 citations in OpenAlex.
- Safety, Pharmacokinetics, and Pharmacodynamics of Osivelotor for Sickle Cell Disease: First-in-Human Studies in Healthy Participants and Patients.Clinical pharmacology and therapeutics · 2026Trial
- Effect of voxelotor on cerebral perfusion and cerebral oxygen metabolism and cardiac stress in adult patients with sickle cell disease.American journal of hematology · 2025Trial
- One-year safety and efficacy of mitapivat in sickle cell disease: follow-up results of a phase 2, open-label study.Blood advances · 2023Trial
- Pharmacokinetics of Voxelotor in Patients With Renal and Hepatic Impairment.Journal of clinical pharmacology · 2021Trial
- Pharmacokinetics and pharmacodynamics of voxelotor (GBT440) in healthy adults and patients with sickle cell disease.British journal of clinical pharmacology · 2019Trial
- Utilization of Model-Informed Drug Development in Early Clinical Development: Case Study of a Hb Modulator for Treatment of Sickle Cell Disease.CPT: pharmacometrics & systems pharmacology · 2026Article
- Hypoxia Preserves Chromatin Integrity and Delays Cellular Senescence through Epigenetic Regulation.Biomolecules & therapeutics · 2026Review
- Potent Antisickling Furaldehyde Analogs for Acute Sickle Cell Therapy: Enhanced Efficacy and Intravenous Formulation Potential.ChemMedChem · 2025Article
- In Silico Post-screening of Anti-polymerization Agents to Treat Sickle Cell Disease.bioRxiv : the preprint server for biology · 2025Article
- Discovery of Osivelotor (GBT021601): A Potent, Next-Generation Sickle Hemoglobin Polymerization Inhibitor.ACS medicinal chemistry letters · 2025Article
- Impact of Voxelotor on Red Blood Cell Exchange Therapeutic Procedures: Evaluation of Multi-Institutional Procedure Data.Journal of clinical apheresis · 2025Article
- Michael Acceptor Compounds as Hemoglobin Oxygen Affinity Modulators for Reversing Sickling of Red Blood Cells.Pharmaceuticals (Basel, Switzerland) · 2025Article
- Promising role of voxelotor in managing sickle cell disease in children: a narrative review.Clinical and experimental pediatrics · 2025Article
- A review on disease modifying pharmacologic therapies for sickle cell disease.Annals of hematology · 2025Review
- Hypoxia as a medicine.Science translational medicine · 2025Review
- Hemoglobin Variants as Targets for Stabilizing Drugs.Molecules (Basel, Switzerland) · 2025Review
- Metabolic profile characterization of voxelotor in human urine based on in vivo and in vitro models for doping control.Analytical and bioanalytical chemistry · 2024Article
- Sickle Cell Disease.Transfusion medicine and hemotherapy : offizielles Organ der Deutschen Gesellschaft fur Transfusionsmedizin und Immunhamatologie · 2024Review
- Gene-environmental influence of space and microgravity on red blood cells with sickle cell disease.NPJ genomic medicine · 2024Review
- Sickle Cell Disease: Current Drug Treatments and Functional Foods with Therapeutic Potential.Current issues in molecular biology · 2024Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
9 authors at 3 institutions in 3 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
aimsVoxelotor (previously GBT440) is a haemoglobin (Hb) modulator that increases Hb-oxygen affinity, thereby reducing Hb polymerization and sickling of red blood cells (RBCs), being developed as a once-daily oral drug to treat sickle cell disease (SCD). This first-in-human study evaluated the safety, tolerability, pharmacokinetics and pharmacodynamics of voxelotor in healthy volunteers and SCD patients.
methodsA total of 40 healthy volunteers (100, 400, 1000, 2000 or 2800 mg) and 8 SCD patients (1000 mg) were randomly assigned to a single dose of voxelotor once daily (n = 6 per group) or placebo (n = 2 per group). Twenty-four healthy volunteers received multiple doses of voxelotor once daily for 15 days (300, 600 or 900 mg, n = 6 per group) or placebo (n = 2 per group).
resultsVoxelotor was well tolerated and exhibited a linear pharmacokinetic profile and a half-life ranging from 61 ± 7 h to 85 ± 7 h. High partitioning into the RBC compartment provides evidence of highly specific binding to Hb. Voxelotor exhibited a concentration-dependent left-shift of oxygen equilibrium curves. Percent Hb modification following 900 mg voxelotor for 15 days was 38 ± 9%. Terminal half-life of voxelotor in SCD patients (50 ± 3 h) was shorter than in healthy volunteers. Evaluation of erythropoietin, exercise testing, and haematologic parameters were consistent with normal oxygen delivery during both rest and exercise.
conclusionThis first-in-human study demonstrates voxelotor was well tolerated in SCD patients and healthy volunteers and established proof of mechanism on increasing Hb-oxygen affinity.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.