Paroxysmal Nocturnal Hemoglobinuria (PNH)Phase 2NCT04901936
A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
A Phase 2 study testing Pegcetacoplan in people with Paroxysmal Nocturnal Hemoglobinuria (PNH) and Paroxysmal Hemoglobinuria, run by Apellis Pharmaceuticals, Inc. at 12 sites in 9 countries.
Trial summary
The purpose of this study is to evaluate the safety, effectiveness, and biological activity (how the investigational medication is processed by the body) of pegcetacoplan in 12-17 year-olds (adolescents) who have paroxysmal nocturnal hemoglobinuria (PNH). Read more about this study
- Who is it for?
- Ages 12 to 17 years
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria (PNH), Paroxysmal Hemoglobinuria
- What is being tested?
- Pegcetacoplan
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 12 sitesin 9 countries
- Study dates
- 4 Feb 2021to Dec 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Are 12-17 years old at the time of screening
- Weigh at least 20 kg (approx. 44 lbs)
- Have the diagnosis of PNH, confirmed by high-sensitivity flow cytometry (granulocyte or monocyte clone >10%)
- EITHER:
- Not being treated with an approved complement inhibitor (eculizumab or ravulizumab) prior to start of pegcetacoplan dosing, AND have hemolytic anemia. Hemolytic anemia is defined as hemoglobin (Hb) less than the lower limit of normal (Hb < LLN) and LDH >1.5 times the upper limit of normal (ULN); OR
- Currently receiving treatment with an approved complement inhibitor (eculizumab or ravulizumab) AND have evidence of ongoing anemia. Ongoing anemia is defined as Hb < LLN and ARC > ULN
- Have a platelet count >75,000/mm3 and an absolute neutrophil count >1000/mm3
Who cannot join
- Are an adult, 18 years of age or older, with PNH
- Known or suspected hereditary fructose intolerance (HFI)
- History of hereditary complement deficiency, bone marrow transplant, or meningococcal disease (meningitis, bacteremia or septicemia)
- Females who are pregnant or breastfeeding
Trial locations and contacts
12 sites in 9 countries. Contact the study team or a site near you to ask about taking part.
Apellis Clinical Trial Information Line contact
12 sites in 9 countries
Netherlands2 sites, 1 recruiting
- Completed
Radboud University Hospital Nijmegen
Nijmegen, 6525 GA
- Recruiting
University Medical Center Utrecht
Utrecht, 3508 GA
Spain2 sites, 2 recruiting
- Recruiting
University Hospital Vall d'Hebron
Barcelona, E-08035
- Recruiting
University Hospital 12 de Octubre
Madrid, E-28041
Thailand2 sites, 1 recruiting
- Completed
Phramongkutklao Hospital and College of Medicine
Bangkok, 10400
- Recruiting
Maharaj Nakorn Chiang Mai hospital
Chiang Mai, 50200
United States1 site, 1 recruiting
- Recruiting
Children's Hospital of Atlanta
Atlanta, Georgia, 30329
France1 site, 1 recruiting
- Not yet recruiting
Robert-Debré Hospital Paris
Paris, 75009
Serbia1 site, 1 recruiting
- Recruiting
University Children's Hospital
Belgrade, 11000
About this study
Official title: An Open Label, Single-Arm, Phase 2 Study to Evaluate the Safety, Pharmacokinetics, and Biologic Activity of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria
The purpose of this study is to evaluate the safety, effectiveness, and biological activity (how the investigational medication is processed by the body) of pegcetacoplan in 12-17 year-olds (adolescents) who have paroxysmal nocturnal hemoglobinuria (PNH).
Read the detailed description
This is an open-label study to evaluate pegcetacoplan in people with PNH who are 12-17 years old. The study will consist of a 4-week screening period followed by a 16-week treatment period. Participants switching from a C5 inhibitor will have an additional 4 week run-in period between the screening and treatment periods. At the completion of the study treatment period, participants will either enter a long-term extension period or a 2-month follow-up period.
All eligible study participants will receive pegcetacoplan, administered via subcutaneous infusion twice a week at home. The subcutaneous infusion requires two small needles to be inserted into the fatty layer of tissue under the skin and the investigational medication will flow into the body. Study participants and/or caregivers will be trained on home administration of pegcetacoplan.
- Being tested
- Pegcetacoplan
- Keywords
- Paroxysmal Nocturnal Hemoglobinuria, PNH, Pediatric, Adolescent, Apellis, Anemia, Hemolytic, Pegcetacoplan, APL-2
- Medical subject headings
- Hemoglobinuria, Paroxysmal, Anemia, Hemolytic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PegcetacoplanDrug
Complement (C3) inhibitor
| Study group | Role | What they receive |
|---|---|---|
| Pegcetacoplan | Experimental | Not described Receives: Drug: Pegcetacoplan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Pegcetacoplan serum concentrations over the course of the 16-week treatment period
Measured: 16 weeks
Change from baseline to Wk 16 in hemoglobin (Hb)
Measured: 16 weeks
Incidence and severity of treatment-emergent adverse events (TEAEs) over the course of the 16-week treatment period, including monitoring bacterial infections
Measured: 16 weeks
Change from baseline to wk 16 lactate dehydrogenase (LDH)
Measured: 16 weeks
Change from baseline to wk 16 absolute reticulocyte count (ARC)
Measured: 16 weeks
Secondary outcomes (9)
Change from baseline from week 16 to week 52 of C3 deposition on RBC cells
Measured: Week 16-52
Incidence of thromboembolic events (major adverse vascular events [MAVE]) over the course of the 16-week treatment period and over 52 weeks of treatment with pegcetacoplan
Measured: 52 weeks
Occurrence of breakthrough hemolysis over 16 and 52 weeks of treatment with pegcetacoplan
Measured: Week 16-52
Change from baseline to Week 52, and from Week 16 to Week 52 , in Hb
Measured: Week 16-52
Change from baseline to Week 16 and to Week 52 in Health-Related Quality of Life (HRQOL) assessments
Measured: Week 16-52
Number of packed red blood cell (PRBC) units
Measured: Week 16-52
Total units (mL/kg) transfused over 16 and 52 weeks of treatment with pegcetacoplan
Measured: Week 16-52
Change from baseline to Week 52, and from Week 16 to Week 52, in LDH
Measured: Week 16-52
Change from baseline to Week 52, and from Week 16 to Week 52 ARC
Measured: Week 16-52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 May 2021
- Study start4 Feb 2021
- Primary completionDec 2028 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Paroxysmal Nocturnal Hemoglobinuria (PNH)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Apellis Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04901936
- Sponsor's protocol ID
- APL2-PNH-209
- Status last verified
- Jun 2026
- First submitted
- 8 Mar 2021
- Last updated
- 26 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label, Single-Arm, Phase 2 Study to Evaluate the Safety, Pharmacokinetics, and Biologic Activity of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria
- ClinicalTrials.gov ID
- NCT04901936
- Acronym / sponsor ID
- APL2-PNH-209
- Keywords
- Paroxysmal Nocturnal Hemoglobinuria, PNH, Pediatric, Adolescent, Apellis, Anemia, Hemolytic, Pegcetacoplan, APL-2
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Apellis Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 4 Feb 2021 (actual)
- Primary completion
- Dec 2028 (estimated)
- Study completion
- Dec 2028 (estimated)
- First posted
- 26 May 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to evaluate the safety, effectiveness, and biological activity (how the investigational medication is processed by the body) of pegcetacoplan in 12-17 year-olds (adolescents) who have paroxysmal nocturnal hemoglobinuria (PNH).
Detailed description
This is an open-label study to evaluate pegcetacoplan in people with PNH who are 12-17 years old. The study will consist of a 4-week screening period followed by a 16-week treatment period. Participants switching from a C5 inhibitor will have an additional 4 week run-in period between the screening and treatment periods. At the completion of the study treatment period, participants will either enter a long-term extension period or a 2-month follow-up period.
All eligible study participants will receive pegcetacoplan, administered via subcutaneous infusion twice a week at home. The subcutaneous infusion requires two small needles to be inserted into the fatty layer of tissue under the skin and the investigational medication will flow into the body. Study participants and/or caregivers will be trained on home administration of pegcetacoplan.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Pegcetacoplan | Experimental | Drug: Pegcetacoplan |
Who may qualify
- Ages
- 12 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- Are 12-17 years old at the time of screening
- Weigh at least 20 kg (approx. 44 lbs)
- Have the diagnosis of PNH, confirmed by high-sensitivity flow cytometry (granulocyte or monocyte clone >10%)
- EITHER:
- Have a platelet count >75,000/mm3 and an absolute neutrophil count >1000/mm3
Key exclusion criteria (4 listed)
- Are an adult, 18 years of age or older, with PNH
- Known or suspected hereditary fructose intolerance (HFI)
- History of hereditary complement deficiency, bone marrow transplant, or meningococcal disease (meningitis, bacteremia or septicemia)
- Females who are pregnant or breastfeeding
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PegcetacoplanComplement (C3) inhibitor | Drug | Pegcetacoplan |
Primary outcome
Pegcetacoplan serum concentrations over the course of the 16-week treatment period
Time frame: 16 weeks
Change from baseline to Wk 16 in hemoglobin (Hb)
Time frame: 16 weeks
Incidence and severity of treatment-emergent adverse events (TEAEs) over the course of the 16-week treatment period, including monitoring bacterial infections
Time frame: 16 weeks
Change from baseline to wk 16 lactate dehydrogenase (LDH)
Time frame: 16 weeks
Change from baseline to wk 16 absolute reticulocyte count (ARC)
Time frame: 16 weeks
9 secondary outcomes
Change from baseline from week 16 to week 52 of C3 deposition on RBC cells
Time frame: Week 16-52
Incidence of thromboembolic events (major adverse vascular events [MAVE]) over the course of the 16-week treatment period and over 52 weeks of treatment with pegcetacoplan
Time frame: 52 weeks
Occurrence of breakthrough hemolysis over 16 and 52 weeks of treatment with pegcetacoplan
Time frame: Week 16-52
Change from baseline to Week 52, and from Week 16 to Week 52 , in Hb
Time frame: Week 16-52
Change from baseline to Week 16 and to Week 52 in Health-Related Quality of Life (HRQOL) assessments
Time frame: Week 16-52
Number of packed red blood cell (PRBC) units
Time frame: Week 16-52
Total units (mL/kg) transfused over 16 and 52 weeks of treatment with pegcetacoplan
Time frame: Week 16-52
Change from baseline to Week 52, and from Week 16 to Week 52, in LDH
Time frame: Week 16-52
Change from baseline to Week 52, and from Week 16 to Week 52 ARC
Time frame: Week 16-52
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Jun 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Apellis Clinical Trial Information Line contact
12 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Pegcetacoplan in people with Paroxysmal Nocturnal Hemoglobinuria (PNH) and Paroxysmal Hemoglobinuria, run by Apellis Pharmaceuticals, Inc. at 12 sites in 9 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 26 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in Netherlands, Spain, Thailand, United States, Czechia and 4 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Apellis Clinical Trial Information Line (1-833-284-6361 (833-CT Info-1), clinicaltrials@apellis.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Pegcetacoplan serum concentrations over the course of the 16-week treatment period (time frame: 16 weeks). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Jun 2026.