← Back to Search

Complement Inhibitor

Pegcetacoplan for Paroxysmal Nocturnal Hemoglobinuria

Phase 2
Recruiting
Research Sponsored by Apellis Pharmaceuticals, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Weigh at least 20 kg (approx. 44 lbs)
Either not being treated with an approved complement inhibitor (eculizumab or ravulizumab) prior to start of pegcetacoplan dosing, AND have hemolytic anemia defined as Hb < LLN and LDH >1.5 times ULN; OR currently receiving treatment with an approved complement inhibitor (eculizumab or ravulizumab) AND have evidence of ongoing anemia defined as Hb < LLN and ARC > ULN
Timeline
Screening 3 weeks
Treatment Varies
Follow Up week 16-52
Awards & highlights

Study Summary

This trial will test the safety and effectiveness of pegcetacoplan in adolescents with PNH.

Who is the study for?
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.Check my eligibility
What is being tested?
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.See study design
What are the potential side effects?
While specific side effects for Pegcetacoplan in this age group aren't detailed here, similar medications may cause injection site reactions, headaches, nausea, diarrhea, fatigue, fever and potential increased risk of infections.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I weigh at least 44 pounds.
Select...
I have not used eculizumab or ravulizumab, or if I have, I still have anemia.
Select...
I have been diagnosed with PNH through a specific blood test.
Select...
I am between 12 and 17 years old.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~week 16-52
This trial's timeline: 3 weeks for screening, Varies for treatment, and week 16-52 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Change from baseline to Wk 16 in hemoglobin (Hb)
Change from baseline to wk 16 absolute reticulocyte count (ARC)
Change from baseline to wk 16 lactate dehydrogenase (LDH)
+2 more
Secondary outcome measures
Erythrocytes
Change from baseline to Week 16 and to Week 52 in Health-Related Quality of Life (HRQOL) assessments
Change from baseline to Week 52, and from Week 16 to Week 52 , in Hb
+6 more

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%
Haemolysis
1%
Vomiting
1%
Sinusitis
1%
Oral herpes
1%
Anaemia
1%
Asthenia
1%
Palpitations
1%
Abdominal distension
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: PegcetacoplanExperimental Treatment1 Intervention
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Pegcetacoplan
2015
Completed Phase 3
~380

Find a Location

Who is running the clinical trial?

Apellis Pharmaceuticals, Inc.Lead Sponsor
24 Previous Clinical Trials
4,013 Total Patients Enrolled

Media Library

Pegcetacoplan (Complement Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT04901936 — Phase 2
Paroxysmal Nocturnal Hemoglobinuria Research Study Groups: Pegcetacoplan
Paroxysmal Nocturnal Hemoglobinuria Clinical Trial 2023: Pegcetacoplan Highlights & Side Effects. Trial Name: NCT04901936 — Phase 2
Pegcetacoplan (Complement Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04901936 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there other ongoing or completed studies using Pegcetacoplan?

"11 different clinical trials are underway to research the efficacy of Pegcetacoplan. Of these, 5 are in Phase 3. The locations for these trials span from California to London, with a total of 593 sites running studies."

Answered by AI

Does this research stand alone or has it been done before?

"As of right now, there are 11 ongoing Pegcetacoplan studies in 164 different cities and 26 countries. The first clinical trial for this medication was back in 2018 and it completed Phase 2 drug approval that year. Since then, 8 more trials have been conducted."

Answered by AI

Has the FDA cleared Pegcetacoplan for use?

"Pegcetacoplan has received a 2 for safety. This is due to the lack of efficacy data, as this is only a Phase 2 trial."

Answered by AI

How many volunteers are enrolling in this clinical trial?

"The answer is affirmative. According to the information available on clinicaltrials.gov, this particular clinical trial is still recruiting participants. The trial was originally posted on February 4th, 2021 and was updated for the last time on July 18th, 2022. They are looking for a total of 12 participants from 1 site."

Answered by AI

Are people still being considered for participation in this trial?

"Yes, you're right. The website says that the trial is still looking for patients. They began recruiting on February 4th 2021 and the listing was updated last on July 18th, 2022. So far, 1 site has enrolled 12 people but they need more participants."

Answered by AI

Does this trial have an age limit?

"This study is enrolling adolescent patients who are between 12 and 17 years old."

Answered by AI

Could I potentially qualify to participate in this research?

"The inclusion criteria for this clinical trial requires that patients have hemoglobinuria, paroxysmal and are between the ages of 12-17. The study is looking to enroll a total of 12 participants."

Answered by AI
Recent research and studies
clinicaltrials.govStudy
A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
2021. "A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04901936.
~3 spots leftby Apr 2025
Unbiased ResultsWe believe in providing patients with all the options.
Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.
Back to top