What side effects are associated with this type of intervention?

Common side effects of complement inhibitor therapies for Paroxysmal Nocturnal Hemoglobinuria (PNH), such as those similar to BCX9930 (a Factor D inhibitor), include headache, upper respiratory tract infections, and infusion-related reactions. Serious but less frequent side effects can include an increased risk of meningococcal infections due to the suppression of the complement system.

What prior FDA approvals exist?

The FDA has approved several complement inhibitors for the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH). Eculizumab (Soliris) and ravulizumab (Ultomiris) are two prominent C5 inhibitors that have been approved for PNH treatment. These drugs work by inhibiting the complement system, which is overactive in PNH patients. BCX9930, a Factor D inhibitor, represents a different approach within the complement pathway, targeting a different component to achieve similar therapeutic effects. This trial aims to evaluate the efficacy and safety of BCX9930 as a monotherapy for PNH patients not receiving other complement inhibitors.

What other types of research exist?

Promising, novel alternatives to BCX9930 for PNH patients in 2024 could include other complement inhibitors such as ravulizumab and pegcetacoplan. These therapies target different components of the complement system and have shown efficacy in reducing hemolysis and improving clinical outcomes in PNH patients.

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Complement Inhibitor

BCX9930 for Paroxysmal Nocturnal Hemoglobinuria (REDEEM-2 Trial)

Phase 2

Waitlist Available

Led By David J Kuter, MD, DPhil

Research Sponsored by BioCryst Pharmaceuticals

Eligibility Criteria Checklist

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

Must have

Body weight ≥ 40 kg

Documented diagnosis of PNH

Must not have

Known history of or existing diagnosis of hereditary complement deficiency

Treatment with anti-thymocyte globulin within 180 days prior to screening

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from week 4 to week 12

Summary

This trial tests an oral medication called BCX9930 for adults with PNH who are not on standard treatments. BCX9930 aims to block proteins that cause PNH symptoms, helping to reduce red blood cell breakdown and improve health.

Who is the study for?

Adults over 18 with PNH who haven't had complement inhibitor therapy for at least a year can join this trial. They must weigh over 40 kg, have a PNH clone of ≥10%, hemoglobin ≤10.5 g/dL, and high lactate dehydrogenase levels. Participants need current vaccinations or agree to get them and cannot access other PNH treatments.

What is being tested?

The study is testing BCX9930 alone against a placebo in patients with PNH who aren't on other complement inhibitors. The goal is to see if BCX9930 is effective and safe as the only treatment for controlling their condition.

What are the potential side effects?

Specific side effects are not listed here, but generally, participants may experience reactions related to the immune system's response to the drug or placebo effect which could include headaches, nausea, or potential allergic reactions.

Eligibility Criteria

Inclusion Criteria

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

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My weight is at least 40 kg.

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I have been diagnosed with PNH.

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I cannot use or access standard complement inhibitor treatments.

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I haven't taken complement inhibitor therapy in the last year.

Exclusion Criteria

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

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I have a genetic issue with my immune system.

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I have not received anti-thymocyte globulin treatment in the last 6 months.

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I have had or am a candidate for a bone marrow or organ transplant.

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I haven't changed my iron supplement dose in the last 28 days.

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

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from week 4 to week 12

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from week 4 to week 12

This trial's timeline: 3 weeks for screening, Varies for treatment, and from week 4 to week 12 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 hemoglobin

Secondary study objectives

Change from baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scale score

Blood Transfusion

Percent change from baseline in lactate dehydrogenase

+1 more

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: BCX9930 monotherapyExperimental Treatment1 Intervention

In Part 1, participants are randomized to 2:1 to receive BCX9930 monotherapy or placebo under double-blind conditions In Part 2, all participants receive open-label BCX9930 monotherapy

Group II: PlaceboPlacebo Group1 Intervention

In Part 1, participants are randomized to 2:1 to receive BCX9930 monotherapy or placebo under double-blind conditions

0 / 9Eligibility 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 target the complement protein C5 to prevent the formation of the membrane attack complex that leads to red blood cell destruction. BCX9930, a Factor D inhibitor, works by blocking an earlier step in the complement pathway, specifically the activation of the alternative pathway, which is crucial in PNH pathophysiology. This inhibition prevents the downstream effects that lead to hemolysis and thrombosis. These treatments are vital for PNH patients as they reduce hemolysis, decrease the risk of thrombosis, and improve overall quality of life by addressing the underlying cause of the disease.

Factor VIIa in patients with C1-inhibitor deficiency.Plasma-derived factor X concentrate compassionate use for hereditary factor X deficiency: Long-term safety and efficacy in a retrospective data-collection study.An overview of inherited factor VII deficiency.