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Monoclonal Antibodies

Long-Term Pegcetacoplan for Paroxysmal Nocturnal Hemoglobinuria

Phase 3
Waitlist Available
Research Sponsored by Apellis Pharmaceuticals, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Males must agree to use contraception and refrain from sperm donation
Willing and able to self-administer pegcetacoplan (administration by caregiver allowed)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline to 2 years
Awards & highlights

Study Summary

This trial is for subjects who have already completed a study on the drug pegcetacoplan and are eligible for this extension study.

Who is the study for?
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.Check my eligibility
What is being tested?
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.See study design
What are the potential side effects?
Specific side effects aren't listed here but may include reactions related to subcutaneous administration, potential hypersensitivity issues similar to other compounds in its class, and risks associated with immune system alterations.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I agree to use contraception and not donate sperm.
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I can take my medication by myself or with help.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline to 2 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to 2 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Incidence and severity of treatment-emergent adverse events

Side effects data

From 2020 Phase 3 trial • 80 Patients • NCT03500549
41%
Injection site erythema
15%
Injection site pruritus
13%
Headache
13%
Injection site swelling
13%
Diarrhoea
10%
Injection site reaction
9%
Nausea
8%
Pyrexia
8%
Injection site pain
6%
Fatigue
6%
Injection site induration
5%
Dyspnoea
5%
Nasopharyngitis
4%
Contusion
4%
Dizziness
4%
Chromaturia
4%
Vaccination site pain
4%
Pain in extremity
4%
Back pain
4%
Myalgia
3%
Vaccination complication
3%
Anxiety
3%
Thrombocytopenia
3%
Cough
3%
Abdominal pain
3%
Abdominal discomfort
3%
Arthralgia
3%
Injection site bruising
3%
Sepsis
1%
Sinusitis
1%
Oral herpes
1%
Haemolysis
1%
Anaemia
1%
Asthenia
1%
Palpitations
1%
Abdominal distension
1%
Vomiting
1%
Constipation
1%
Urinary tract infection
100%
80%
60%
40%
20%
0%
Study treatment Arm
Run-in Period: Pegcetacoplan + Eculizumab
Open-label Period: Pegcetacoplan
RCP: Eculizumab
RCP: Pegcetacoplan

Trial Design

1Treatment groups
Experimental Treatment
Group I: 1,080 mg pegcetacoplan administered subcutaneouslyExperimental Treatment1 Intervention
1,080mg pegcetacoplan administered subcutaneously twice weekly or every three days.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Pegcetacoplan
FDA approved

Find a Location

Who is running the clinical trial?

Apellis Pharmaceuticals, Inc.Lead Sponsor
24 Previous Clinical Trials
3,865 Total Patients Enrolled

Media Library

Pegcetacoplan (Monoclonal Antibodies) Clinical Trial Eligibility Overview. Trial Name: NCT03531255 — Phase 3
Paroxysmal Nocturnal Hemoglobinuria Research Study Groups: 1,080 mg pegcetacoplan administered subcutaneously
Paroxysmal Nocturnal Hemoglobinuria Clinical Trial 2023: Pegcetacoplan Highlights & Side Effects. Trial Name: NCT03531255 — Phase 3
Pegcetacoplan (Monoclonal Antibodies) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03531255 — Phase 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
~28 spots leftby Jun 2025