ALXN2050 for Paroxysmal Nocturnal Hemoglobinuria

Recruiting at 23 trial locations

Overseen By Alexion Pharmaceuticals Inc.

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Alexion

Must be taking: Eculizumab, Danicopan

Disqualifiers: Organ transplant, Aplastic anemia, 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: 12

Duration: 12 weeks

29 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications. However, if you are currently on eculizumab, you must have been on a stable dose for at least 8 weeks. If you are on danicopan, you must have received it during a specific previous study.

What is the purpose of this trial?

This trial tests a new oral medication, ALXN2050, for patients with PNH. It targets those who are either new to treatment or not fully responding to their current medication. The medication works by blocking a protein that causes red blood cells to break down.

Eligibility Criteria

This trial is for adults with Paroxysmal Nocturnal Hemoglobinuria (PNH) who either haven't been treated, still have anemia after eculizumab treatment, or are on danicopan. They must have certain levels of blood cells and enzymes indicating PNH. People can't join if they've had organ transplants, other bone marrow issues not related to PNH, or severe kidney problems.

Inclusion Criteria

You have a specific type of blood cell clone that is larger than 10%.

Your LDH levels in the blood are higher than 1.5 times the normal limit.

Your blood has low levels of hemoglobin (Hgb <10.5 g/dL).

Exclusion Criteria

I have a bleeding disorder or condition causing anemia not related to PNH.

I have had a major organ or bone marrow transplant.

I have a bone marrow condition needing a stem cell transplant or have been on immunosuppressants for less than 6 months.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive ALXN2050 monotherapy for 12 weeks to assess efficacy and safety

12 weeks

Periodic visits through Week 12

Long-term Extension

Participants continue on ALXN2050 treatment beyond 12 weeks

Long-term

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• ALXN2050

Trial Overview The study tests ALXN2050 as a solo treatment for PNH. It checks how well it works and its safety in new patients, those previously on eculizumab with ongoing anemia, and those taking danicopan alone. Participants will be checked regularly up to Week 12 then continue into a long-term part of the study.

Participant Groups

Group I: Open-label ALXN2050 Monotherapy Experimental Treatment 1 Intervention

Experimental: Open-label ALXN2050 Monotherapy ALXN2050 orally administered Group 1: Patients with PNH who are treatment naïve Group 2: Patient with PNH who have received complement component 5 (C5) inhibition with eculizumab for at least 6 months, who continue to experience anemia and reticulocytes above the upper limit of normal (ULN) Group 3: Patients with PNH who have received danicopan monotherapy during study ACH471-103

Who Is Running the Clinical Trial?

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

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