Long-Term Pegcetacoplan for Paroxysmal Nocturnal Hemoglobinuria

Not currently recruiting at 67 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Apellis Pharmaceuticals, Inc.

Must be taking: Pegcetacoplan

Must not be taking: Complement inhibitors

Disqualifiers: Hepatitis B, C, HIV, others

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

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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: Long-term

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial examines the effectiveness of pegcetacoplan, a new treatment for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that causes premature destruction of red blood cells. The study aims to determine if continued treatment with pegcetacoplan maintains health benefits for those who have previously used it successfully in past trials. It is suitable for adults with PNH who have found pegcetacoplan helpful and are comfortable self-administering the medication. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants the opportunity to contribute to the potential availability of a new treatment.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot take other complement inhibitors like eculizumab or ravulizumab while participating.

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

Research has shown that pegcetacoplan is generally well-tolerated by people with paroxysmal nocturnal hemoglobinuria (PNH). Studies have found that patients using pegcetacoplan for up to three years experienced mostly mild or moderate side effects, such as reactions at the injection site. Serious side effects occurred less frequently. This suggests that pegcetacoplan is generally safe and tolerable for long-term use in people with PNH. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for Paroxysmal Nocturnal Hemoglobinuria (PNH), which often involve complement inhibitors like eculizumab, pegcetacoplan stands out by blocking part of the complement system known as C3. This unique mechanism of action allows it to prevent the destruction of red blood cells more effectively. Additionally, pegcetacoplan is administered subcutaneously, giving patients a potentially more convenient option compared to intravenous treatments. Researchers are excited because this approach may improve anemia and reduce the need for blood transfusions in PNH patients.

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

Research has shown that pegcetacoplan effectively treats paroxysmal nocturnal hemoglobinuria (PNH). Studies have found that it increases hemoglobin levels, lowers lactate dehydrogenase (LDH) levels—an enzyme linked to cell damage—and reduces fatigue. In a direct comparison, pegcetacoplan proved more effective than eculizumab, a common PNH treatment. Patients experienced quick improvements within weeks, and these benefits lasted a long time. This treatment has improved the quality of life for people with PNH. ^{5 6 7 8 9}

Are You a Good Fit for This Trial?

Adults with Paroxysmal Nocturnal Hemoglobinuria (PNH) who've been in a pegcetacoplan study and seen benefits or are expected to benefit can join. They must be vaccinated against certain infections, able to self-administer the drug or have help, not pregnant, using contraception, and without conditions that increase risk.

Inclusion Criteria

I am 18 or older, have PNH, and benefited from a pegcetacoplan trial without major side effects.

I am vaccinated against meningitis, pneumonia, and Hib or have a documented nonresponder status.

I agree to use contraception and not donate sperm.

Exclusion Criteria

I have had a bone marrow transplant.

I am currently taking medication that inhibits the complement system.

I have hepatitis B, C, or HIV.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive 1,080 mg pegcetacoplan administered subcutaneously twice weekly or every three days

Long-term

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Pegcetacoplan

Trial Overview The trial is testing the long-term safety and effectiveness of Pegcetacoplan for PNH patients. It's an open-label extension study meaning everyone gets Pegcetacoplan and knows it; there's no placebo or comparison group.

How Is the Trial Designed?

Group I: 1,080 mg pegcetacoplan administered subcutaneously Experimental Treatment 1 Intervention

1,080mg pegcetacoplan administered subcutaneously twice weekly or every three days.

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+

Published Research Related to This Trial

In a real-world study involving 61 patients with paroxysmal nocturnal hemoglobinuria (PNH) treated with pegcetacoplan for an average of 1.3 to 14.8 months, there was a significant increase in hemoglobin levels by 2.5 g/dL and a 27.4% reduction in patients with elevated lactate dehydrogenase (LDH) levels, indicating effective treatment outcomes.

Patient and physician satisfaction with pegcetacoplan was over 90%, and both groups reported improvements in fatigue and health-related quality of life (HRQoL), suggesting that pegcetacoplan may be a preferred option compared to previous treatments.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pegcetacoplan in paroxysmal nocturnal haemoglobinuria: Its use, its clinical effectiveness, and its influence on health-related quality of life and productivity. Wilson, K., Rich, C., Hakimi, Z., et al.[2023]

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/37994576/

Pegcetacoplan in paroxysmal nocturnal haemoglobinuria These real-world data support the effectiveness of PEG through positive effects on haemoglobin, LDH, fatigue and HRQoL.

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 ... Baseline data highlight a need for improved treatment. After 6 months of pegcetacoplan treatment, pts experienced rapid improvements in ...

4. investors.apellis.com investors.apellis.com/news-releases/news-release-details/apellis-reports-positive-top-line-results-phase-3-head-head

Apellis Reports Positive Top-line Results from Phase 3 ... Top-line data show that pegcetacoplan met the study's primary efficacy endpoint, demonstrating superiority to eculizumab with a statistically ...

5. 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 markedly and rapidly (within 4 weeks) improved all efficacy measures, which stabilized close to (hemoglobin, FACIT-Fatigue) or ...

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

Efficacy and Safety Maintained up to 3 Years in Adults with ... Introduction: Pegcetacoplan, the first C3 and C3b inhibitor for paroxysmal nocturnal hemoglobinuria (PNH), demonstrated efficacy and safety in C5 inhibitor- ...

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11052769/

Safety and Efficacy of Pegcetacoplan in Adult Patients with ... Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, life-threatening disease characterized by complement-mediated hemolysis and thrombosis.

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. 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 ...