What side effects are associated with this type of intervention?

Common treatments for Paroxysmal Nocturnal Hemoglobinuria (PNH) include eculizumab and ravulizumab, which are complement inhibitors. Known side effects of these treatments include headaches, upper respiratory tract infections, and infusion-related reactions. For treatments similar to the Factor D inhibitor ALXN2050, such as eculizumab, patients may also experience anemia and reticulocytosis. Overall, while these treatments are generally well-tolerated, monitoring for infections and managing infusion reactions are important aspects of patient care.

What prior FDA approvals exist?

The FDA has approved several treatments for Paroxysmal Nocturnal Hemoglobinuria (PNH), primarily focusing on complement inhibition. Eculizumab (Soliris) was the first complement inhibitor approved, targeting the C5 protein to prevent hemolysis. Ravulizumab (Ultomiris), a longer-acting C5 inhibitor, was subsequently approved to offer extended dosing intervals. These treatments are similar to the Factor D inhibitor ALXN2050 being studied, as they all aim to mitigate the effects of complement-mediated hemolysis in PNH patients.

What other types of research exist?

Promising novel alternatives to ALXN2050 for PNH patients include eculizumab and danicopan. Eculizumab is a well-established treatment, while danicopan is another option currently used in monotherapy. Both are viable alternatives for patients seeking treatment in 2024.

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Factor D Inhibitor

ALXN2050 for Paroxysmal Nocturnal Hemoglobinuria

Phase 2

Waitlist Available

Research Sponsored by Alexion

Eligibility Criteria Checklist

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

Must have

Diagnosis of PNH

Be older than 18 years old

Must not have

Known underlying bleeding disorders or any other conditions leading to anemia not primarily associated with PNH

History of a major organ transplant or hematopoietic stem cell/marrow transplant

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from first dose of study drug up to week 217

Awards & highlights

No Placebo-Only Group

Summary

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.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Possible side effects aren't specified here but may include reactions typical of oral medications targeting the immune system such as gastrointestinal symptoms, potential liver enzyme changes, and increased risk of infections.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been diagnosed with PNH.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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

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I have had a major organ or bone marrow transplant.

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

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I have been treated with a complement inhibitor before.

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My kidney function is very low or I am on dialysis.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from first dose of study drug up to week 217

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from first dose of study drug up to week 217

This trial's timeline: 3 weeks for screening, Varies for treatment, and from first dose of study drug up to week 217 for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Change From Baseline in Hgb at Week 12

Secondary study objectives

Change From Baseline in Absolute Reticulocyte Count at Week 12

Change From Baseline in Component 3 (C3) Fragment Deposition on PNH RBCs at Week 12

Change From Baseline in FACIT-Fatigue Scale (Version 4) Scores at Week 160

+5 more

Awards & Highlights

No Placebo-Only Group

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

Trial Design

1Treatment groups

Experimental Treatment

Group I: Open-label ALXN2050 MonotherapyExperimental Treatment1 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

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

ALXN2050

2021

Completed Phase 1

~250

0 / 6Eligibility criteria met

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

Paroxysmal Nocturnal Hemoglobinuria (PNH) is commonly treated with complement inhibitors such as eculizumab and ravulizumab, which block the complement protein C5, preventing the formation of the membrane attack complex that leads to red blood cell destruction. Factor D inhibitors, like the investigational drug ALXN2050, target an earlier step in the complement pathway by inhibiting Factor D, a crucial enzyme in the alternative pathway activation. This inhibition prevents the amplification of the complement cascade, reducing hemolysis and thrombosis risk. These treatments are vital for PNH patients as they address the underlying cause of hemolysis, improving quality of life and reducing complications associated with the disease.

[The Outcome of Severe Aplastic Anemia Children Treated with Reduced Dose of Cyclophosphamide Combined Cyclosporine A].