ALXN1820 for Sickle Cell Disease

(PHOENIX Trial)

Recruiting at 15 trial locations

Overseen By Alexion Pharmaceuticals, Inc.

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Alexion Pharmaceuticals, Inc.

Must be taking: Hydroxyurea

Must not be taking: Voxelotor, Crizanlizumab, Erythropoetins

Disqualifiers: Hepatitis, Severe renal impairment, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 12 weeks

2 Participants Needed

Trial Summary

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.

What is the purpose of this trial?

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.

Eligibility Criteria

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

My vaccinations for Hib and pneumonia are current as per guidelines for sickle cell disease.

I have been diagnosed with sickle cell disease.

I've had between 1 and 10 pain crises in the last year.

Exclusion Criteria

History of complement deficiency

I have not had a serious infection in the last 14 days.

I haven't taken complement inhibitors in the last 6 months.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

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

12 weeks

Weekly or bi-weekly visits depending on dosing regimen

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• ALXN1820

Trial Overview 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).

Participant Groups

Group I: ALXN1820 600 mg once every 4 weeks Experimental Treatment 1 Intervention

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

Group II: ALXN1820 300 mg once weekly Experimental Treatment 1 Intervention

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

Group III: ALXN1820 300 mg once every 2 weeks (Optional cohort) Experimental Treatment 1 Intervention

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

Who Is Running the Clinical Trial?

Alexion Pharmaceuticals, Inc.

Lead Sponsor

Trials

267

Recruited

141,000+

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

Trials

247

Recruited

38,600+

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

Trials

231

Recruited

36,700+

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