Pegcetacoplan for Paroxysmal Nocturnal Hemoglobinuria

Recruiting at 9 trial locations

Overseen By Rushi Shah

Age: < 18

Sex: Any

Trial Phase: Phase 2

Sponsor: Apellis Pharmaceuticals, Inc.

Must be taking: Complement inhibitors

Disqualifiers: Adults, Hereditary fructose intolerance, 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

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 16 weeks

12 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores the safety and effectiveness of pegcetacoplan for treating paroxysmal nocturnal hemoglobinuria (PNH), a rare blood condition, in adolescents. PNH can cause issues like anemia (low red blood cell count) and fatigue. The trial seeks participants aged 12-17 who have PNH and either experience ongoing anemia while on certain medications or have hemolytic anemia (a condition where red blood cells break down too quickly) without current treatment. Participants will receive pegcetacoplan, and researchers will study its efficacy and interaction with the body. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

Do I have to stop taking my current medications for the trial?

The trial does not specify if you need to stop taking your current medications. However, if you are already on a complement inhibitor like eculizumab or ravulizumab, you can continue with it while participating in the trial.

Is there any evidence suggesting that pegcetacoplan is likely to be safe for humans?

Research has shown that pegcetacoplan is well-tolerated by adults with paroxysmal nocturnal hemoglobinuria (PNH). Studies found it remained safe and effective for up to three years, with most adults experiencing no serious side effects over this period.

These studies also noted improvements in hemoglobin levels, crucial for oxygen transport in the blood, and a decrease in fatigue and the need for blood transfusions. This indicates that the treatment not only alleviated symptoms but also proved safe over time.

While this information pertains to adult patients, it offers a hopeful outlook for its potential safety in teenagers. However, since the trial is in an earlier stage for younger patients, safety in this age group is still under investigation. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Pegcetacoplan is unique because it targets the complement system, specifically C3, which plays a key role in the destruction of red blood cells in paroxysmal nocturnal hemoglobinuria (PNH). Most current treatments for PNH, like eculizumab and ravulizumab, inhibit the C5 protein further down the complement pathway. By intervening earlier in the pathway, pegcetacoplan has the potential to offer more comprehensive protection against red blood cell destruction. Researchers are excited about this treatment because it could improve hemoglobin levels and reduce the need for blood transfusions, enhancing the quality of life for patients with PNH.

What evidence suggests that pegcetacoplan might be an effective treatment for paroxysmal nocturnal hemoglobinuria?

Research has shown that pegcetacoplan, the treatment under study in this trial, effectively treats paroxysmal nocturnal hemoglobinuria (PNH). In previous studies, patients experienced lasting improvements in hemoglobin levels, enhancing their blood's ability to carry oxygen. Pegcetacoplan also reduced fatigue and the need for blood transfusions. Long-term studies found these benefits lasted up to three years. This treatment has succeeded in adults, suggesting it might also benefit teenagers with PNH. ^{1 6 7 8 9}

Are You a Good Fit for This Trial?

This trial is for adolescents aged 12-17 with paroxysmal nocturnal hemoglobinuria (PNH). They can be new to treatment or already receiving certain drugs but still have anemia. Participants must weigh at least 44 pounds and have specific blood cell counts. It's not for those over 18, with hereditary fructose intolerance, complement deficiencies, a history of bone marrow transplant or meningococcal disease, nor pregnant or breastfeeding females.

Inclusion Criteria

I weigh at least 44 pounds.

I have not used eculizumab or ravulizumab, or if I have, I still have anemia.

I have been diagnosed with PNH through a specific blood test.

Exclusion Criteria

I am not pregnant or breastfeeding.

I have a history of complement deficiency, bone marrow transplant, or meningococcal disease.

I am 18 or older with PNH.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

4 weeks

Run-in

Participants switching from a C5 inhibitor undergo a run-in period

4 weeks

Treatment

Participants receive pegcetacoplan via subcutaneous infusion twice a week at home

16 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

8 weeks

Long-term extension

Participants may enter a long-term extension period for continued treatment

What Are the Treatments Tested in This Trial?

Interventions

• Pegcetacoplan

Trial Overview The study tests Pegcetacoplan's safety and effectiveness in treating PNH in young patients. It looks at how the body processes this medication and its impact on the disease's symptoms like anemia caused by red blood cells breaking down too quickly.

How Is the Trial Designed?

Group I: Pegcetacoplan Experimental Treatment 1 Intervention

Pegcetacoplan is already approved in United States, European Union for the following indications:

Approved in United States as Empaveli for:

• Paroxysmal Nocturnal Hemoglobinuria (PNH)

Approved in European Union as Aspaveli for:

• Paroxysmal Nocturnal Hemoglobinuria (PNH)

Who Is Running the Clinical Trial?

Apellis Pharmaceuticals, Inc.

Lead Sponsor

Trials

26

Recruited

4,300+

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40720060/

Efficacy and Safety Maintained up to 3 Years in Adults with ... Conclusion: Pegcetacoplan efficacy and safety was sustained for up to 3 years in patients with PNH, with or without prior C5 inhibitor treatment, verifying the ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT05776472

Study Details | NCT05776472 | A Real World Effectiveness ... This is a 36-month, long-term, multicenter, observational study designed to describe the real world effectiveness of pegcetacoplan in patients with PNH.

3. ashpublications.org ashpublications.org/blood/article/144/Supplement%201/5669/527339/A-First-Report-from-the-COMPLETE-Study-on-the-Real

A First Report from the COMPLETE Study on the Real-World ... Median (IQR) percentage increase in Hb was 24.2% (5.4%-34.1%) and 55% of pts had a Hb increase of ≥2 g/dL. There were no pegcetacoplan-related ...

4. link.springer.com link.springer.com/article/10.1007/s12325-024-02827-8

Safety and Efficacy of Pegcetacoplan in Adult Patients with ... Pegcetacoplan sustained long-term improvements in hemoglobin concentrations, fatigue reduction, and transfusion burden.

5. onlinelibrary.wiley.com onlinelibrary.wiley.com/doi/full/10.1002/ajh.27242

Real‐world experience of pegcetacoplan in paroxysmal ... Pegcetacoplan significantly improves outcomes for patients with paroxysmal nocturnal hemoglobinuria (PNH) experiencing extravascular hemolysis (EVH) on ...

6. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/38573482/

Safety and Efficacy of Pegcetacoplan in Adult Patients with ... Pegcetacoplan sustained long-term improvements in hemoglobin concentrations, fatigue reduction, and transfusion burden.

7. clinicaltrials.gov clinicaltrials.gov/study/NCT03500549

NCT03500549 | Study to Evaluate the Efficacy and Safety ... Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH). ClinicalTrials.gov ID NCT03500549. Sponsor ...

8. ashpublications.org ashpublications.org/blood/article/142/Supplement%201/574/502392/Efficacy-and-Safety-Is-Maintained-in-Adult

Efficacy and Safety Is Maintained in Adult Patients with ... Efficacy and Safety Is Maintained in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria Receiving Pegcetacoplan for up to 3 Years Available.

9. link.springer.com link.springer.com/article/10.1007/s12325-025-03310-8

Efficacy and Safety Maintained up to 3 Years in Adults with ... Pegcetacoplan efficacy and safety was sustained for up to 3 years in patients with PNH, with or without prior C5 inhibitor treatment, verifying ...