Hydroxyurea for Sickle Cell Anemia
(OPTIMA Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests hydroxyurea to assist individuals with sickle cell disease, a condition affecting red blood cells. It aims to determine if pharmacokinetic testing, which assesses how the body processes the drug, can help patients achieve the optimal dose more quickly than standard methods. Participants will be divided into three groups: two groups will start hydroxyurea using either the new test or standard bloodwork, and one group will consist of individuals who have been on hydroxyurea for over a year to check drug levels. The trial seeks children and teens with sickle cell disease who are either beginning hydroxyurea or have been on it for at least a year. As a Phase 2 and Phase 3 trial, this research measures the treatment's effectiveness in an initial, smaller group and represents the final step before FDA approval, offering participants a chance to contribute to advancing treatment options.
Do I need to stop my current medications to join the trial?
The trial information does not specify if you need to stop taking your current medications. However, it does require participants to take hydroxyurea for the study duration.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Research has shown that hydroxyurea is generally safe for people with sickle cell anemia. In one study, hydroxyurea reduced the risk of hospitalization by 85%. Another study found that people taking hydroxyurea had a lower chance of dying over nine years. This treatment also helps children by reducing their need for emergency room visits.
Hydroxyurea has been used for many years to treat sickle cell disease and is approved by the FDA for this condition, indicating that most people tolerate it well. However, like any treatment, it can have side effects, so discussing them with a doctor is important.12345Why are researchers excited about this trial?
Researchers are excited about hydroxyurea for sickle cell anemia because it offers a unique approach by potentially increasing fetal hemoglobin levels, which can help reduce the sickling of red blood cells. Unlike standard treatments that primarily focus on managing pain or preventing infections, hydroxyurea targets the disease at a cellular level, aiming to decrease the frequency of painful episodes and other complications. Additionally, its ability to potentially reach maximum tolerated dose (MTD) while being monitored for pharmacokinetics (PK) makes it an intriguing option for personalized treatment plans. These features offer hope for more effective management of sickle cell anemia, differentiating hydroxyurea from conventional care options.
What is the effectiveness track record for hydroxyurea in treating sickle cell anemia?
Research has shown that hydroxyurea effectively treats sickle cell anemia. Studies have found that it increases hemoglobin levels, aiding oxygen transport in the blood, and reduces white blood cell counts, which can decrease inflammation. In children with sickle cell disease, hydroxyurea has lowered emergency room visits and hospital stays over time. At doses above 20 mg/kg/day, it can prevent organ damage and increase fetal hemoglobin levels by about 18%. Overall, hydroxyurea has demonstrated lasting benefits for individuals with sickle cell disease. Participants in this trial will be assigned to different groups to evaluate the pharmacokinetics and effects of hydroxyurea over a 12-month period.45678
Are You a Good Fit for This Trial?
This trial is for patients with moderate to severe sickle cell anemia. Participants must be able to undergo pharmacokinetic testing and have not reached their maximum tolerated dose of hydroxyurea. Specific inclusion and exclusion criteria details are not provided.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive hydroxyurea with dosage adjustments based on pharmacokinetic testing or standard bloodwork over 12 months
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Hydroxyurea
Trial Overview
The study tests if using pharmacokinetic testing can help patients find the best dose of hydroxyurea faster than standard bloodwork follow-up. Patients are randomly assigned to two groups: one uses pharmacokinetic results (Group A) and the other follows standard care (Group B). There's also a Group C for those already on hydroxyurea for 12 months, checked once.
How Is the Trial Designed?
3
Treatment groups
Experimental Treatment
Active Control
Patient will undergo one PK sample procurement to evaluate level of HU-AUC after 12 months of taking hydroxyurea.
Patient starting hydroxyurea that will have PK sample procurements at different timpoints over a period of 12 months until HU-AUC shows MTD
Patient starting hydroxuyrea that will be followed as per standard of care over a period of 12 months until MTD has been reached
Find a Clinic Near You
Who Is Running the Clinical Trial?
Yves Pastore
Lead Sponsor
St. Justine's Hospital
Collaborator
Published Research Related to This Trial
Citations
The Current Role of Hydroxyurea in the Treatment of Sickle ...
This study showed that the MTD achieved greater increases in HbF and total hemoglobin levels, as well as a more significant reduction in white ...
2.
ashpublications.org
ashpublications.org/bloodadvances/article/9/14/3585/536727/Evaluating-the-long-term-benefits-of-hydroxyureaEvaluating the long-term benefits of hydroxyurea in pediatric ...
This study demonstrates that hydroxyurea has sustained clinical benefits in reducing ED visits and hospital days across years of use in children with SCA.
3.
hematology.org
hematology.org/newsroom/press-releases/2025/hydroxyurea-effective-long-term-in-children-living-with-scdHydroxyurea Effective Long Term in Children Living With ...
Hydroxyurea remains effective long-term in reducing emergency department visits and hospital days for children living with sickle cell disease (SCD), according ...
4.
hematologyadvisor.com
hematologyadvisor.com/features/hydroxyurea-for-sickle-cell-anemia-in-children/Hydroxyurea for Sickle Cell Anemia in Children
Researchers determined long-term treatment with hydroxyurea is beneficial for children with sickle cell anemia (SCA).
5.
systematicreviewsjournal.biomedcentral.com
systematicreviewsjournal.biomedcentral.com/articles/10.1186/s13643-024-02461-zRole of hydroxyurea therapy in the prevention of organ ...
Our study found that a hydroxyurea dose above 20 mg/kg/day with a mean rise in HbF by 18.46% post-hydroxyurea therapy had a beneficial role.
The Risks and Benefits of Long-term Use of Hydroxyurea in ...
A randomized, controlled clinical trial established the efficacy and safety of short-term use of hydroxyurea in adult sickle cell anemia.
Thirty Years of Hydroxyurea for Sickle Cell Anemia
Additional follow-up research involving patients in the MSH trial found that, after 9 years, hydroxyurea was associated with reduced mortality.
8.
ashpublications.org
ashpublications.org/bloodadvances/article/7/16/4319/495797/Efficacy-safety-and-pharmacokinetics-of-a-newEfficacy, safety, and pharmacokinetics of a new, ready-to-use ...
The risk of hospitalization was reduced by 85% after initiation of hydroxyurea treatment (odds ratio 0.15 [95% confidence interval, 0.05-0.45]; ...
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