Long-Term Pegcetacoplan for Paroxysmal Nocturnal Hemoglobinuria

Recruiting at 61 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

160 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, but you cannot take other complement inhibitors like eculizumab or ravulizumab while participating.

What data supports the effectiveness of the drug Pegcetacoplan for treating paroxysmal nocturnal hemoglobinuria?

Research shows that Pegcetacoplan helps improve hemoglobin levels and reduces the need for blood transfusions in patients with paroxysmal nocturnal hemoglobinuria (PNH). It has been found to be more effective than eculizumab, another treatment, in managing anemia and improving quality of life for PNH patients. ^{1 2 3 4 5}

How is the drug pegcetacoplan different from other treatments for paroxysmal nocturnal hemoglobinuria (PNH)?

Pegcetacoplan is unique because it is the first drug to target C3, a part of the immune system involved in PNH, providing more complete control of hemolysis (breakdown of red blood cells) compared to existing C5 inhibitors like eculizumab. It is administered subcutaneously (under the skin) and has shown to improve hemoglobin levels and reduce the need for blood transfusions, enhancing the quality of life for patients. ^{1 2 3 4 5}

What is the purpose of this trial?

This is an Open-label, Non-Randomized, Multi-Center Extension Study. Eligible subjects will have previously completed a pegcetacoplan study.

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

References

1. England 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. [2023]

2. United States pubmed.ncbi.nlm.nih.gov

C3 inhibition with pegcetacoplan in subjects with paroxysmal nocturnal hemoglobinuria treated with eculizumab. [2021]

3. England pubmed.ncbi.nlm.nih.gov

Safety and efficacy of pegcetacoplan in paroxysmal nocturnal hemoglobinuria. [2023]

4. New Zealand pubmed.ncbi.nlm.nih.gov

Pegcetacoplan: A Review in Paroxysmal Nocturnal Haemoglobinuria. [2023]

5. United States pubmed.ncbi.nlm.nih.gov

Pegcetacoplan controls hemolysis in complement inhibitor-naive patients with paroxysmal nocturnal hemoglobinuria. [2023]