What side effects are associated with this type of intervention?

Common treatments for Sickle Cell Disease include hydroxyurea and L-glutamine. Hydroxyurea can cause mild myelotoxicity, gastrointestinal symptoms, and has teratogenic potential, requiring discontinuation before conception. L-glutamine is generally well-tolerated but may cause mild gastrointestinal symptoms. Chronic transfusions, another treatment option, can lead to iron overload and alloimmunization. Investigational therapies, such as those similar to ALXN1820, may have varying side effects that are still under study.

What prior FDA approvals exist?

Hydroxyurea was the first FDA-approved drug for Sickle Cell Disease (SCD), known for increasing fetal hemoglobin (Hb F) levels to reduce complications. L-glutamine (Endari) was later approved to reduce acute complications. Voxelotor (Oxbryta) inhibits hemoglobin polymerization, and crizanlizumab (Adakveo) targets P-selectin to reduce vaso-occlusive crises. These treatments represent various mechanisms to manage SCD effectively.

What other types of research exist?

Promising novel alternatives to ALXN1820 for Sickle Cell Disease patients include L-glutamine, voxelotor, and crizanlizumab. These drugs have been approved by the FDA for the prophylaxis and treatment of SCD complications. Additionally, sevuparin, a novel non-anticoagulant low molecular weight heparinoid with anti-adhesive properties, is being investigated for its potential to shorten vaso-occlusive crisis duration.

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ALXN1820 for Sickle Cell Disease (PHOENIX Trial)

Phase 2

Waitlist Available

Research Sponsored by Alexion Pharmaceuticals, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, week 12

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new medication called ALXN1820, given as an injection under the skin, for people with Sickle Cell Disease. The main goal is to see if it is safe and if patients can tolerate it.

Who is the study for?

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.

What is being tested?

The trial is testing the safety of ALXN1820 given under the skin to adults with Sickle Cell Disease. It aims to understand how well it's tolerated and its effects on the body (pharmacokinetics/dynamics).

What are the potential side effects?

While specific side effects for ALXN1820 are not listed here, common risks may include reactions at injection site, potential allergic responses due to ingredients like polysorbate 80, and general discomfort.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, week 12

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, week 12

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, week 12 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Secondary study objectives

Change From Baseline or Percent Change From Baseline in Complement Biomarkers Through Week 12 (Cohorts 1 and 2)

Hemolytic Complement

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

3Treatment groups

Experimental Treatment

Group I: ALXN1820 600 mg once every 4 weeksExperimental Treatment1 Intervention

Participants will receive 600 mg once every 4 weeks (Q4W).

Group II: ALXN1820 300 mg once weeklyExperimental Treatment1 Intervention

Participants will receive 300 milligrams (mg) once weekly (QW).

Group III: ALXN1820 300 mg once every 2 weeks (Optional cohort)Experimental Treatment1 Intervention

Participants will receive 300 mg once every 2 weeks (Q2W).

Do I qualify?Apply below to find out

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Sickle Cell Disease (SCD) include hydroxyurea, which increases fetal hemoglobin (Hb F) production to reduce red blood cell sickling and vaso-occlusive events, and red blood cell transfusions, which lower the proportion of sickled cells in the bloodstream to prevent complications. Investigational therapies, such as those in trials like ALXN1820, often focus on molecular modifications to reduce sickling and improve nitric oxide availability. These treatments are vital for SCD patients as they address the disease's root causes, aiming to alleviate pain, prevent complications, and enhance quality of life.

[Sickle cell disease].The controversial role of red cell transfusions for sickle cell pain.Optimizing hydroxyurea therapy for sickle cell anemia.