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Voxelotor

Clinical trials are investigating Voxelotor in people with sickle cell disease. These studies look at whether it can improve markers of hemolysis, which means red blood cell breakdown, and help with organ damage. The trial data here focus on safety-related study design details, biological effects, and measured outcomes in a small patient group.

Table of Contents

Trial overview

The trial listed for Voxelotor is HEMolyse and Organ damage imPROvement in sickle cell disease by VoxElotor, with the study ID NCT05199766.[1] It is a Phase 2, interventional study in sickle cell disease.[1] The brief summary says the study aimed to evaluate the biological activity of Voxelotor on the reduction of intravascular hemolysis, measured by plasma hemoglobin.[1]

Study population and design

The target population in this trial was people with sickle cell disease.[1] The study used an intervention, meaning participants received the study treatment and the researchers measured the effects over time.[1] The listed intervention was Oxbryta 500 mg film-coated tablets given by oral use, with a total dose shown in the source as 1500 mg.[1]

The source does not provide extra details about age limits, sex limits, or other entry rules.[1] Because of that, the only clearly stated participation group is people with sickle cell disease.[1]

Outcomes and endpoints

The main endpoint, also called the primary outcome, was improvement of intravascular hemolysis.[1] This was defined as at least a 20% decrease in plasma hemoglobin between week 0 and week 48.[1]

In simple words, the researchers were checking whether a blood marker linked to red blood cell breakdown got better during the study.[1] The trial title also mentions organ damage improvement, which suggests the researchers were interested in whether the treatment might help with damage caused by the disease.[1]

Trial status and enrollment

This study is marked as Completed.[1] The enrollment was 35 participants.[1] That is a small study, which is common in early clinical research when the goal is to look for signs of biological effect.[1]

What this trial means for patients

For patients, this trial is important because it tested whether Voxelotor could change a measurable sign of disease activity in sickle cell disease.[1] The study did not focus on a broad range of conditions; it focused on one disease group and one main blood-based outcome.[1] The available data are centered on study design, patient group, and measured results, rather than on a full list of clinical benefits or harms.[1]

Trial ID Phase Condition studied Status Enrollment
NCT05199766 Phase 2 Sickle cell disease Completed 35

FAQ

  • What is being studied in Voxelotor clinical trials? The trial data focus on whether Voxelotor can improve intravascular hemolysis, which is red blood cell breakdown inside blood vessels. The main measure is a change in plasma hemoglobin over time.
  • Who can take part in these trials? The study listed here involved people with sickle cell disease. The trial data do not give more detailed entry rules in the source provided.
  • What phase is the Voxelotor trial? The trial listed here is a Phase 2 study. Phase 2 trials usually look at early signs of benefit and continue to collect study data in a defined patient group.
  • What outcome did the trial measure? The main outcome was improvement in intravascular hemolysis. This was defined as at least a 20% decrease in plasma hemoglobin between week 0 and week 48.
  • How many people were enrolled in the trial? The trial enrolled 35 people. It was an interventional study, meaning the researchers gave a study treatment and measured the results.

Ongoing Clinical Trials on Voxelotor

  • Study on Voxelotor for Reducing Hemolysis in Patients with Sickle Cell Disease

    Not recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    France

Glossary

  • Sickle cell disease: A blood condition studied in this trial. It affects red blood cells and can lead to ongoing health problems.
  • Intravascular hemolysis: Breakdown of red blood cells inside blood vessels. The trial measured whether this got better over time.
  • Plasma hemoglobin: Hemoglobin found in the liquid part of blood. In this study, it was used as a marker of red blood cell breakdown.
  • Primary outcome: The main result the researchers planned to measure. This trial used a change in plasma hemoglobin as the primary outcome.
  • Phase 2: A mid-stage clinical trial. It usually checks whether a treatment shows signs of benefit in a group of patients.
  • Interventional study: A study in which researchers give a treatment or intervention and then measure the effects.
  • Enrollment: The number of people who joined the study.
  • Organ damage: Harm to body organs. The trial title suggests the study was also interested in whether Voxelotor could help with this problem.

References