A Phase 2 interventional study of Pegcetacoplan in Paroxysmal Nocturnal Hemoglobinuria (PNH) and Paroxysmal Hemoglobinuria, sponsored by Apellis Pharmaceuticals, Inc.. Recruiting at 12 sites in 9 countries. Open to participants aged 12 Years to 17 Years. Per ClinicalTrials.gov, last updated 2026-06-26.
Sponsored by Apellis Pharmaceuticals, Inc. (part of Biogen) · Phase 2, Interventional, and Treatment
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).
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.
191 studies on the registry are indexed under Hemoglobinuria, Paroxysmal; 49 are open to participants now.
This study's planned enrollment of 12 is below the median of 33 across 150 interventional studies indexed under Hemoglobinuria, Paroxysmal.
Browse Hemoglobinuria, Paroxysmal studies →Apellis Pharmaceuticals, Inc. is the lead sponsor of 28 studies on the registry; 3 are open to participants now.
Of its 18 completed or terminated interventional studies of FDA-regulated products, 16 (89%) have results posted.
First submitted before Apellis Pharmaceuticals, Inc. became part of Biogen.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
EITHER:
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Absolute neutrophil count (ANC)
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Drug: Pegcetacoplan
Complement (C3) inhibitor
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
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
Not enrolling at this site.
Not enrolling at this site.
Not enrolling at this site.
Not enrolling at this site.
Started Feb 2021 (actual). Primary completion is estimated for Dec 2028, 94 months after the start.
Of 26 industry-sponsored phase 2 interventional studies under Hemoglobinuria, Paroxysmal, half reached primary completion within 16 months of starting (the median), counting those still running; a quarter within 11 and three quarters within 25. This study's planned 94 months is above that.
Counting every sponsor, half of 34 studies reached it within 24 months; a quarter within 12 and three quarters within 80.
How these figures are worked out, and which studies are left out of them.
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
Contact study teamGet an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Studies of the drug tested here: Pegcetacoplan
Apellis Pharmaceuticals, Inc. →