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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 belowSelect...
I agree to use contraception and not donate sperm.
Select...
I can take my medication by myself or with help.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ baseline to 2 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline to 2 years
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 • NCT0350054941%
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
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have had a bone marrow transplant.I am 18 or older, have PNH, and benefited from a pegcetacoplan trial without major side effects.I am currently taking medication that inhibits the complement system.I am vaccinated against meningitis, pneumonia, and Hib or have a documented nonresponder status.I have hepatitis B, C, or HIV.I agree to use contraception and not donate sperm.I can take my medication by myself or with help.I have a genetic issue affecting my immune system.I have severe aplastic anemia.I have had meningococcal disease before.I have PNH and finished a pegcetacoplan trial without getting the drug but might benefit from starting it.
Research Study Groups:
This trial has the following groups:- Group 1: 1,080 mg pegcetacoplan administered subcutaneously
Awards:
This trial has 3 awards, including:- All Individual Drugs Already Approved - Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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