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Voxelotor for Sickle Cell Disease
Study Summary
This trial tests if a drug can improve blood flow & brain function in adolescents & young adults with SCD.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowTimeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Trial Design
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Who is running the clinical trial?
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- I have had a blood transfusion in the last 3 months or am scheduled for one.I am not pregnant or breastfeeding.I needed strong pain medication through an IV due to a blood flow blockage event within the last 28 days.I am currently experiencing symptoms of COVID-19.I am a woman who can have children and have a negative pregnancy test.I am currently being treated for a serious infection.I am not on medications that strongly affect liver enzyme activity.I have severe kidney problems or am on dialysis.I am not currently on antibiotics for an acute bacterial infection.I am between 12 and 30 years old.I have severe narrowing in one or more of my brain's main arteries.My anemia is caused by my bone marrow not working properly.I have had a stem cell transplant or gene therapy.I do not have active hepatitis A, B, C, or HIV.I have taken Adakveo within the last 90 days.I have not had surgery in the last 8 weeks and do not plan to have surgery during the study.I have had a stroke, mini-stroke, or spinal cord injury.I have given or, if under 18, my guardian has given consent to participate.I have taken Oxbryta within the last 90 days.I have been on a stable dose of hydroxyurea for at least 90 days without changes.I have sickle cell disease with HbSS or Hbβ0 thalassemia genotype.I have been on a stable dose of ESAs for at least 12 weeks with no changes expected.
- Group 1: Active Drug
- Approved for 5 Other Conditions - This treatment demonstrated efficacy for 5 other conditions.
- All Individual Drugs Already Approved - Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
- Drug Has Already Been Approved - The FDA has already approved this drug, and is just seeking more data.
- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
To what extent could Active Drug pose a risk to those who use it?
"Because this treatment is approved, our assessment of the safety of Active Drug was a 3 on a 1 to 3 scale."
Can subjects still join this research endeavor?
"The current recruitment status is active, as reflected on clinicaltrials.gov. An initial posting date of August 9th 2023 and a last updated date of August 23rd 2023 can be found there."
Does the qualification criteria for this research involve an age limit of 45 years or younger?
"This trial is only available to those aged 12-30, however there are 120 clinical trials that accept applicants under the age of 18 and 79 for individuals over 65."
What is the number of individuals that have opted into this research project?
"Affirmative. According to the information available from clinicaltrials.gov, this investigation is currently enrolling participants that were first published on August 9th of 2023 and has been updated recently on August 23rd. 24 individuals are being sought out over 2 research sites for participation in the trial."
What criteria must be met in order to participate in this clinical trial?
"This medical trial is looking to enrol 24 participants between 12 and 30 years old, suffering from sickle cell disease. To be eligible for this study, the individual must conform to these requirements: Male or female with HbSS/Hbβ0 thalassemia genotype; haemoglobin level ranging from 5.5-10.5 g/dL; capability of MRI scans; no dose alteration in hydroxyurea/erythropoiesis-stimulating agents (ESAs) for 90 days/12 weeks respectively before enrollment; highly effective contraception methods if female or barrier contraception if male during drug administration period & up"
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