A Phase 3 interventional study of Pegcetacoplan in PNH, sponsored by Apellis Pharmaceuticals, Inc.. Completed at 62 sites in 21 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-11-10.
Sponsored by Apellis Pharmaceuticals, Inc. (part of Biogen) · Phase 3, Interventional, and Treatment
This is an Open-label, Non-Randomized, Multi-Center Extension Study. Eligible subjects will have previously completed a pegcetacoplan study.
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.
Subjects at least 18 years of age with PNH who have participated in a pegcetacoplan clinical trial. Subjects who received treatment with pegcetacoplan must have experienced clinical benefit and adequate tolerability in the opinion of the investigator.
Note: Subjects with PNH who completed a pegcetacoplan clinical trial without receiving pegcetacoplan (or without receiving pegcetacoplan for long enough to demonstrate clinical benefit) may be enrolled in this study if, in the opinion of the Investigator, the subject is expected to demonstrate clinical benefit upon the initiation or continuation of pegcetacoplan therapy.
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.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Postmenopausal
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
1,080mg pegcetacoplan administered subcutaneously twice weekly or every three days.
Drug: Pegcetacoplan
Complement (C3) Inhibitor
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
An adverse event (AE) is any untoward medical occurrence associated with use of a drug in humans, whether or not considered drug related. An AE can therefore be any unfavorable and unintended sign, including a clinically significant abnormal laboratory finding, symptom, or disease temporally associated with the use of a study drug, whether or not considered related to the study drug. TEAEs were defined as those AEs that occurred or worsened in severity after the first dose of pegcetacoplan.
Time frame: From first dose of study drug up to 8 weeks from last dose (up to approximately 4 years or until pegcetacoplan development in participants with PNH was terminated)
Number of Participants With Thromboembolic Events
Time frame: From first dose of study drug up to 8 weeks from last dose (up to approximately 4 years or until pegcetacoplan development in participants with PNH was terminated)
Change From Baseline in Hematology Parameters (Erythrocytes)
Time frame: Baseline, Weeks 168 and 264
Change From Baseline in Hematology Parameters (Leukocytes, Platelets and Reticulocytes)
Time frame: Baseline, Weeks 168 and 264
Change From Baseline in Hematology Parameters (Hematocrit)
Time frame: Baseline, Weeks 168 and 264
Change From Baseline in Hematology Parameters (Hemoglobin)
Time frame: Baseline, Weeks 168 and 264
Change From Baseline in Serum Chemistry Parameters [Alanine Aminotransferase, Alkaline Phosphatase, Aspartate Aminotransferase, Lactate Dehydrogenase (LDH)]
Time frame: Baseline, Weeks 168 and 264
Change From Baseline in Serum Chemistry Parameters (Bilirubin)
Time frame: Baseline, Weeks 168 and 264
Number of Participants With Post-Baseline Potentially Clinically Significant Abnormal Coagulation Parameters
Coagulation parameters included Activated Partial Thromboplastin Time (APTT), D-Dimer, Fibrinogen, Prothrombin Intl. Normalized Ratio and Prothrombin Time.
Time frame: Baseline, Week 264
Number of Participants With Post-Baseline Potentially Clinically Significant Abnormal Urinalysis Parameters
Urinalysis parameters included ketones, leukocyte esterase, nitrite, occult blood, pH, protein, specific gravity and urobilinogen.
Time frame: Baseline, Week 264
Number of Participants With Shift From Baseline in Clinically Significant Abnormalities in Electrocardiograms (ECG)
ECG measurements included heart rate, PR, QT, QRS, QTcB and QTcF.
Time frame: Baseline, Week 324
Change From Baseline in Hemoglobin Concentration
Time frame: Baseline, Week 324
Change From Baseline in Lactate Dehydrogenase (LDH) Concentration
Time frame: Baseline, Week 324
Change From Baseline in Indirect Bilirubin Level
Time frame: Baseline, Week 324
Change From Baseline in Absolute Reticulocyte Count (ARC)
Time frame: Baseline, Week 324
Number of Participants Who Received Packed Red Blood Cell Transfusions (PRBCs)
Transfusion included whole blood, PRBCs, leukocyte-depleted red blood cells (LDPRCs), and all types of red blood cell (RBC) transfusions.
Time frame: From first dose of study drug up to 8 weeks from last dose (up to approximately 4 years or until pegcetacoplan development in participants with PNH was terminated)
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score
The FACIT-Fatigue scale consists of 13 questions that assess fatigue and its impact on daily activities over the past 7 days. Each question is scored on a 5-point scale from 0 ("not at all") to 4 ("very much"). For items where a higher response indicates greater fatigue, the score is reversed so that all items are scored in the same direction. The scores from the 13 items are then added together to calculate the FACIT-Fatigue total score. The total score ranges from 0 to 52, with higher scores indicating less fatigue and better functioning.
Time frame: Baseline, Week 324
Participants took part in the study at multiple investigative sites from 27 August 2018 to 19 May 2026.
| Milestone | Pegcetacoplan |
|---|---|
| Started | 137 |
| Completed | 1 |
| Not completed | 136 |
| Withdrew: Adverse event | 6 |
| Withdrew: Withdrew consent | 5 |
| Withdrew: Lost to follow-up | 2 |
| Withdrew: Physician decision | 5 |
| Withdrew: Sponsor request | 1 |
| Withdrew: Entered the substudy | 27 |
| Withdrew: Death | 5 |
| Withdrew: Commercial study drug use | 77 |
| Withdrew: Reason not specified | 1 |
| Withdrew: Ongoing in study | 7 |
An adverse event (AE) is any untoward medical occurrence associated with use of a drug in humans, whether or not considered drug related. An AE can therefore be any unfavorable and unintended sign, including a clinically significant abnormal laboratory finding, symptom, or disease temporally associated with the use of a study drug, whether or not considered related to the study drug. TEAEs were defined as those AEs that occurred or worsened in severity after the first dose of pegcetacoplan.
| Participants | Pegcetacoplan |
|---|---|
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | 135 |
| Participants | Pegcetacoplan |
|---|---|
| Number of Participants With Thromboembolic Events | 2 |
| 10^12 cells/liter | Pegcetacoplan |
|---|---|
| Baseline | 3.484 ± 0.7648 |
| Change at Week 168 | -0.214 ± 0.6687 |
| Change at Week 264 | -0.579 ± 1.0062 |
| 10^9 cells/liter | Pegcetacoplan |
|---|---|
| Leukocytes (Baseline) | 4.15 ± 1.665 |
| Leukocytes (Change at Week 168) | -0.22 ± 1.333 |
| Leukocytes (Change at Week 264) | -0.03 ± 0.955 |
| Platelets (Baseline) | 134.6 ± 61.60 |
| Platelets (Change at Week 168) | 13.9 ± 36.06 |
| Platelets (Change at Week 264) | 8.2 ± 35.47 |
| Reticulocytes (Baseline) | 87.4 ± 38.98 |
| Reticulocytes (Change at Week 168) | -11.1 ± 41.35 |
| Reticulocytes (Change at Week 264) | 6.6 ± 53.29 |
| liter/liter | Pegcetacoplan |
|---|---|
| Baseline | 0.359 ± 0.0653 |
| Change at Week 168 | -0.005 ± 0.0587 |
| Change at Week 264 | -0.059 ± 0.0885 |
| grams/deciliter (g/dl) | Pegcetacoplan |
|---|---|
| Baseline | 11.64 ± 2.153 |
| Change at Week 168 | -0.49 ± 2.000 |
| Change at Week 264 | -1.59 ± 2.870 |
| units per liter (U/L) | Pegcetacoplan |
|---|---|
| Alanine aminotransferase (Baseline) | 22.9 ± 24.12 |
| Alanine aminotransferase (Change at Week 168) | -0.5 ± 12.36 |
| Alanine aminotransferase (Change at Week 264) | -1.6 ± 13.36 |
| Alkaline phosphatase (Baseline) | 72.8 ± 23.85 |
| Alkaline phosphatase (Change at Week 168) | -11.8 ± 21.73 |
| Alkaline phosphatase (Change at Week 264) | -6.1 ± 21.72 |
| Aspartate aminotransferase (Baseline) | 21.0 ± 24.83 |
| Aspartate aminotransferase (Change at Week 168) | 1.8 ± 15.21 |
| Aspartate aminotransferase (Change at Week 264) | 1.4 ± 23.12 |
| LDH (Baseline) | 300.3 ± 435.30 |
| LDH (Change at Week 168) | -1.7 ± 401.35 |
| LDH (Change at Week 264) | 168.5 ± 941.22 |
| micromole/liter (μmol/L) | Pegcetacoplan |
|---|---|
| Baseline | 14.665 ± 8.8433 |
| Change at Week 168 | -1.399 ± 6.7758 |
| Change at Week 264 | 0.529 ± 13.3418 |
Coagulation parameters included Activated Partial Thromboplastin Time (APTT), D-Dimer, Fibrinogen, Prothrombin Intl. Normalized Ratio and Prothrombin Time.
| Participants | Pegcetacoplan |
|---|---|
| APTT (Shift to Low) | 0 |
| APTT (Shift to High) | 3 |
| D-Dimer (Shift to Low) | 0 |
| D-Dimer (Shift to High) | 3 |
| Fibrinogen (Shift to Low) | 2 |
| Fibrinogen (Shift to High) | 3 |
| Prothrombin Intl. Normalized Ratio (Shift to Low) | 0 |
| Prothrombin Intl. Normalized Ratio (Shift to High) | 2 |
| Prothrombin Time (Shift to Low) | 0 |
| Prothrombin Time (Shift to High) | 1 |
Urinalysis parameters included ketones, leukocyte esterase, nitrite, occult blood, pH, protein, specific gravity and urobilinogen.
| Participants | Pegcetacoplan |
|---|---|
| Ketones (Shift to Low) | 0 |
| Ketones (Shift to High) | 0 |
| Leukocyte Esterase (Shift to Low) | 0 |
| Leukocyte Esterase (Shift to High) | 0 |
| Nitrite (Shift to Low) | 0 |
| Nitrite (Shift to High) | 2 |
| Occult Blood (Shift to Low) | 0 |
| Occult Blood (Shift to High) | 0 |
| pH (Shift to Low) | 0 |
| pH (Shift to High) | 0 |
| Protein (Shift to Low) | 0 |
| Protein (Shift to High) | 0 |
| Specific Gravity (Shift to Low) | 0 |
| Specific Gravity (Shift to High) | 0 |
| Urobilinogen (Shift to Low) | 0 |
| Urobilinogen (Shift to High) | 0 |
ECG measurements included heart rate, PR, QT, QRS, QTcB and QTcF.
| Participants | Pegcetacoplan |
|---|---|
| Number of Participants With Shift From Baseline in Clinically Significant Abnormalities in Electrocardiograms (ECG) | 0 |
| grams per deciliter (g/dL) | Pegcetacoplan |
|---|---|
| Change From Baseline in Hemoglobin Concentration | 0.20 ± NA |
| units per liter (U/L) | Pegcetacoplan |
|---|---|
| Change From Baseline in Lactate Dehydrogenase (LDH) Concentration | 303.0 ± NA |
| μmol/L | Pegcetacoplan |
|---|---|
| Change From Baseline in Indirect Bilirubin Level | 0.400 ± NA |
| 10^9 cells/liter | Pegcetacoplan |
|---|---|
| Change From Baseline in Absolute Reticulocyte Count (ARC) | 40.0 ± NA |
Transfusion included whole blood, PRBCs, leukocyte-depleted red blood cells (LDPRCs), and all types of red blood cell (RBC) transfusions.
| Participants | Pegcetacoplan |
|---|---|
| Number of Participants Who Received Packed Red Blood Cell Transfusions (PRBCs) | 78 |
The FACIT-Fatigue scale consists of 13 questions that assess fatigue and its impact on daily activities over the past 7 days. Each question is scored on a 5-point scale from 0 ("not at all") to 4 ("very much"). For items where a higher response indicates greater fatigue, the score is reversed so that all items are scored in the same direction. The scores from the 13 items are then added together to calculate the FACIT-Fatigue total score. The total score ranges from 0 to 52, with higher scores indicating less fatigue and better functioning.
| score on a scale | Pegcetacoplan |
|---|---|
| Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score | 0.00 ± NA |
Collected over From first dose of study drug up to 8 weeks from last dose (up to approximately 4 years or until pegcetacoplan development in participants with PNH was terminated). Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Pegcetacoplan | 5/137 (3.6%) | 72/137 (52.6%) | 119/137 (86.9%) |
| Event | Pegcetacoplan |
|---|---|
| HaemolysisBlood and lymphatic system disorders | 44/137 |
| COVID-19Infections and infestations | 11/137 |
| PneumoniaInfections and infestations | 5/137 |
| AnaemiaBlood and lymphatic system disorders | 4/137 |
| Dengue feverInfections and infestations | 4/137 |
| Urinary tract infectionInfections and infestations | 4/137 |
| Acute kidney injuryRenal and urinary disorders | 3/137 |
| Haemolytic anaemiaBlood and lymphatic system disorders | 2/137 |
| Intravascular haemolysisBlood and lymphatic system disorders | 2/137 |
| ThrombocytopeniaBlood and lymphatic system disorders | 2/137 |
| Event | Pegcetacoplan |
|---|---|
| HaemolysisBlood and lymphatic system disorders | 60/137 |
| COVID-19Infections and infestations | 40/137 |
| Upper respiratory tract infectionInfections and infestations | 22/137 |
| NasopharyngitisInfections and infestations | 15/137 |
| Urinary tract infectionInfections and infestations | 15/137 |
| HeadacheNervous system disorders | 14/137 |
| DiarrhoeaGastrointestinal disorders | 13/137 |
| HypertensionVascular disorders | 12/137 |
| AnaemiaBlood and lymphatic system disorders | 11/137 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 11/137 |
Safety set included all participants who received at least 1 dose of pegcetacoplan.
| Age, Continuous(years) | Pegcetacoplan |
|---|---|
| Mean | 46.5 ± 14.60 |
| Sex: Female, Male(Participants) | Pegcetacoplan |
|---|---|
| Female | 76 |
| Male | 61 |
| Race/Ethnicity, Customized(Participants) | Pegcetacoplan |
|---|---|
| Race — American Indian or Alaska Native | 5 |
| Race — Asian | 63 |
| Race — Black or African American | 3 |
| Race — White | 48 |
| Race — Other | 8 |
| Race — Missing | 10 |
| Race/Ethnicity, Customized(Participants) | Pegcetacoplan |
|---|---|
| Ethnicity — Hispanic or Latino | 16 |
| Ethnicity — Not Hispanic or Latino | 111 |
| Ethnicity — Missing | 10 |
PDFs hosted by the registry, ClinicalTrials.gov.
Ran from Aug 2018 to primary completion in Sep 2025, 84 months.
Results were first posted in Nov 2026, 1 year 2 months after primary completion.
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
This study is completed, as verified in Oct 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Pegcetacoplan
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