ALXN1820 for Sickle Cell Disease
(PHOENIX Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to determine if ALXN1820, an experimental treatment, is safe and well-tolerated for people with Sickle Cell Disease (SCD). SCD can cause painful episodes and other health issues, and the study will explore different dosing schedules to identify the most effective one. Participants will receive either a weekly or monthly dose of ALXN1820. The trial is suitable for those who have experienced 1 to 10 painful crises related to SCD in the past year and are currently stable on their SCD treatment. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to significant advancements in SCD treatment.
Will I have to stop taking my current medications?
The trial requires that if you are taking hydroxyurea, you must have been on a stable dose for at least 3 months before joining and cannot change the dose during the study. You cannot be taking Voxelotor or crizanlizumab within 60 days before starting the trial, and you cannot be on certain other medications like recombinant human erythropoetins or complement inhibitors.
Is there any evidence suggesting that ALXN1820 is likely to be safe for humans?
Research is examining the safety of ALXN1820 for people with sickle cell disease. Earlier studies found that most patients tolerated ALXN1820 well. These studies did not identify any serious side effects directly linked to the treatment, suggesting it might be safe for most people. However, as the trials remain in the early stages, researchers continue to gather more safety information. Participants should consider this and consult their doctor if they have any concerns.12345
Why are researchers excited about this trial's treatments?
Researchers are excited about ALXN1820 for sickle cell disease because it offers a novel approach compared to existing treatments. While typical treatments like hydroxyurea and blood transfusions focus on managing symptoms or complications, ALXN1820 targets the underlying disease process more directly. ALXN1820 is unique because it is designed to be administered in varied doses and schedules, including 300 mg weekly, 600 mg every four weeks, and an optional 300 mg every two weeks, potentially offering flexibility and convenience for patients. This versatility, combined with the promise of a more targeted mechanism, makes ALXN1820 a promising candidate for improving sickle cell disease management.
What evidence suggests that ALXN1820 might be an effective treatment for Sickle Cell Disease?
Research shows that ALXN1820 is under testing to determine its potential benefits for people with Sickle Cell Disease (SCD). Participants in this trial will receive varying dosages of ALXN1820: 300 mg once weekly, 600 mg once every 4 weeks, or 300 mg once every 2 weeks. Although detailed results on its effectiveness in humans are not yet available, this treatment aims to target areas in the body that might reduce SCD symptoms, potentially leading to less pain and improved blood flow. Early findings suggest it might help manage some challenging symptoms of SCD. Further studies are underway to confirm its effectiveness and safety for patients.13467
Are You a Good Fit for This Trial?
Adults with Sickle Cell Disease (HbSS, or HbSβ0-thalassemia) weighing at least 40 kg and having a hemoglobin level between 5.5 and 10 g/dL can join. They must be on stable hydroxyurea doses if applicable, vaccinated against certain infections, not planning major treatment changes, without severe kidney issues or recent transfusions/infections, and not pregnant.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive ALXN1820 with different dosing regimens: 300 mg once weekly, 600 mg once every 4 weeks, or 300 mg once every 2 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- ALXN1820
Find a Clinic Near You
Who Is Running the Clinical Trial?
Alexion Pharmaceuticals, Inc.
Lead Sponsor
Dr. Alberto R. Martinez
Alexion Pharmaceuticals, Inc.
Chief Medical Officer since 2010
MD from University of Sao Paulo
Marc Dunoyer
Alexion Pharmaceuticals, Inc.
Chief Executive Officer since 2021
PhD in Molecular Biology
Alexion
Lead Sponsor
Marc Dunoyer
Alexion
Chief Executive Officer since 2021
PhD in Molecular Biology, University of Brussels
Christophe Hotermans
Alexion
Chief Medical Officer since 2021
MD, University of Leuven
Alexion Pharmaceuticals
Lead Sponsor
Marc Dunoyer
Alexion Pharmaceuticals
Chief Executive Officer since 2021
B.A. in Psychology from the University of New Hampshire
Christophe Hotermans
Alexion Pharmaceuticals
Chief Medical Officer since 2023
MD