PNHPhase 3NCT03531255
Pegcetacoplan Long Term Safety and Efficacy Extension Study
A Phase 3 study testing Pegcetacoplan in people with PNH, run by Apellis Pharmaceuticals, Inc. at 62 sites in 21 countries.
Trial summary
This is an Open-label, Non-Randomized, Multi-Center Extension Study. Eligible subjects will have previously completed a pegcetacoplan study. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- PNH
- What is being tested?
- Pegcetacoplan
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 137 participants
- Where
- 62 sitesin 21 countries
- Study dates
- 27 Aug 2018to 19 May 2026
- Phase
- Phase 3
- Are there results?
- None posted in the registry
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
- Vaccination against Neisseria meningitidis types A, C, W, Y and B, Streptococcus pneumoniae and Haemophilus influenzae Type B (Hib) either within 2 years prior to Day 1 dosing of this study, or within 14 days after starting treatment with pegcetacoplan. Vaccination is mandatory unless documented evidence exists that subjects are nonresponders to vaccination as evidenced by titers or display titer levels within acceptable local limits. Immunization status checks will be performed to determine whether subjects require primary or booster vaccinations.
- Willing and able to give written informed consent.
- Willing and able to self-administer pegcetacoplan (administration by caregiver will be allowed)
- Women of childbearing potential (WOCBP) defined as any females who have experienced menarche and who are NOT permanently sterile or postmenopausal must have a negative pregnancy test and must agree to continue to use an approved method of contraception for the duration of the study and 90 days after their last dose of study drug. Note: Postmenopausal is defined as 12 consecutive months with no menses without an alternative medical cause.
- Males must agree to continue to use an approved method of contraception and must agree to refrain from donating sperm for the duration of the study and 90 days after their last dose of study drug.
Who cannot join
- Subjects who have withdrawn from a pegcetacoplan clinical study.
- Any condition that could increase the subject's risk by participating in the study.
- Any comorbidity or condition (such as malignancy) that, in the opinion of the investigator, could put the subject at increased risk or potentially confound study data.
- History or presence of hypersensitivity or idiosyncratic reaction to compounds related to the investigational product or SC administration.
- Known infection with hepatitis B, C, or HIV.
- Hereditary complement deficiency.
Show all 11Show fewer
- History of bone marrow transplant.
- Concurrent severe aplastic anemia (SAA), defined as currently receiving immunosuppressive therapy for SAA including but not limited to cyclosporin A, tacrolimus, mycophenolate mofetil or anti-thymocyte globulin.
- History of meningococcal disease.
- Concomitant treatment with any complement inhibitor (eg, eculizumab, ravulizumab).
- Pregnancy, breastfeeding, or positive pregnancy test.
Trial locations and contacts
62 sites in 21 countries.
62 sites in 21 countries
United States9 sites
Northwestern University
Chicago, Illinois, 60611
Cleveland Clinic
Cleveland, Ohio, 44195
Denver Health Medical Center
Denver, Colorado, 80204
Duke University Medical Center
Durham, North Carolina, 27710
Investigative Clinical Research of Indiana
Indianapolis, Indiana, 46260
Cancer Specialists of North Florida
Jacksonville, Florida, 32257
University of Southern California
Los Angeles, California, 90033
Baptist Cancer Center
Memphis, Tennessee, 38120
Lakes Research
Miami, Florida, 33014
Thailand7 sites
Phramongkutklao Hospital
Bangkok, 10400
Ramathibodi Hospital
Bangkok, 10400
Siriraj Hospital
Bangkok, 10400
Maharaj Nakorn Chiang Mai Hospital
Chiang Mai, 50200
Srinagarind Hospital
Khon Kaen, 40002
Thammasat University Hospital
Pathum Thani, 12120
Songklanagarind Hospital
Songkhla, 90110
France6 sites
Hospital Center Chalon Sur Saone William Morey
Chalon-sur-Saône, 71100
CHRU de Lille
Lille, 59037
Hopital Saint-Louis
Paris, 75010
Lyon Sud Hospital Center
Pierre-Bénite, 69495
Centre Hospitalier Annecy Genevois - Site Annecy
Pringy, 74374
Centre Hospitalier de Saint-Quentin
Saint-Quentin, 02321
Japan6 sites
Juntendo University Hospital
Bunkyō-Ku, Tokyo, 113-8421
Shinshu University Hospital
Matsumoto, Nagano, 390-8621
Okayama University Hospital
Okayama, 700-8558
Japanese Red Cross Nagoya Daini Hospital
Showa-ku, Aichi-ken, 486-8650
Kinan Hospital
Tanabe, Wakayama, 646-8588
NTT Medical Center Tokyo
Tokyo, 141-0022
Philippines5 sites
Perpetual Succour Hospital
Cebu, National Capital Region, 6000
Mary Mediatrix Medical Centre
Lipa City, National Capital Region, 4217
Makati Medical Centre
Makati City, National Capital Region, 1229
The Medical City
Pasig, National Capital Region, 1604
St. Lukes Medical Centre
Quezon City, National Capital Region, 1112
Germany4 sites
Uniklinik RWTH Aachen
Aachen, North Rhine-Westphalia, 52074
Essen University Hospital Department of Hematology
Essen, North Rhine-Westphalia, 45147
University Medical Center Hamburg-Eppendorf
Hamburg, 20246
Universitatsklinikum Ulm
Ulm, Baden-Wurttemberg, 89081
Canada3 sites
University of Calgary
Calgary, Alberta, T2N 4Z6
Alberta Health Services
Edmonton, Alberta, T6G 2V2
Toronto General Hospital
Toronto, Ontario, M5G 2C4
Peru3 sites
Hospital Edgardo Rebagliatti
Jesús María, Lima, 15072
Hospital Nacional Dos De Mayo - Centro de Investigación
Lima Cercado, Lima region, 15003
Centro de Investigacion en Hematologia Clinica del Centro Medico Corpac
San Isidro, Lima region, 15047
Belgium2 sites
Cliniques Universitaires Saint-luc
Brussels, 1200
AZ Delta
Roeselare, 8800
Bulgaria2 sites
Acibadem City Clinic Tokuda Hospital
Sofia, 1407
National Specialized Hospital for Active Treatment of Haematologic Diseases
Sofia, 1756
Hong Kong2 sites
Prince of Wales Hospital
Hong Kong
Queen Mary Hospital
Hong Kong
Malaysia2 sites
Hospital Ampang
Ampang, Selangor, 68000
University Malaya Medical Centre
Lembah Pantai, Kuala Lumpur, 59100
Russia2 sites
Pavlov First Saint Petersburg State Medical University
Saint Petersburg, 197022
Therapeutic hospital of regional clinical hospital #1
Tyumen, 625023
Spain2 sites
Hospital Univ. de Gran Canaria Dr.Negrin
Las Palmas de Gran Canaria, Canary Islands, 35019
Hospital Universitario Politecnico La Fe
Valencia, 46026
Colombia1 site
IPS Centro Medico Julian Coronel SAS
Cali, Valle del Cauca Department, 760035
Mexico1 site
Hospital Universitario "José Eleuterio González", UANL
Monterrey, 64460
South Korea1 site
Chungnam National University Hospital
Junggu, Daejeon, 35015
About this study
Official title: An Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegcetacoplan in the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH)
This is an Open-label, Non-Randomized, Multi-Center Extension Study. Eligible subjects will have previously completed a pegcetacoplan study.
- Conditions
- PNH
- Being tested
- Pegcetacoplan
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 |
|---|---|---|
| 1,080 mg pegcetacoplan administered subcutaneously | Experimental | 1,080mg pegcetacoplan administered subcutaneously twice weekly or every three days. Receives: Drug: Pegcetacoplan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and severity of treatment-emergent adverse events
Measured: Baseline to 2 Years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 May 2018
- Study start27 Aug 2018
- Primary completion4 Sep 2025
- Study completion19 May 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for PNHStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 137 participants (actual)
Sponsor and investigators
- Lead sponsor
- Apellis Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03531255
- Sponsor's protocol ID
- APL2-307
- Status last verified
- Mar 2026
- First submitted
- 9 May 2018
- Last updated
- 5 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
de Castro C, Kelly RJ, Griffin M, Patriquin CJ, Mulherin B, Hochsmann B, Selvaratnam V, Wong RSM, Hillmen P, Horneff R, Uchendu UO, Zhang Y, Surova E, Szamosi J, de Latour RP. Efficacy and Safety Maintained up to 3 Years in Adults with Paroxysmal Nocturnal Hemoglobinuria Receiving Pegcetacoplan. Adv Ther. 2025 Sep;42(9):4641-4658. doi: 10.1007/s12325-025-03310-8. Epub 2025 Jul 28.
PubMed 40720060Patriquin CJ, Bogdanovic A, Griffin M, Kelly RJ, Maciejewski JP, Mulherin B, Peffault de Latour R, Roth A, Selvaratnam V, Szer J, Al-Adhami M, Horneff R, Tan L, Yeh M, Panse J. Safety and Efficacy of Pegcetacoplan in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria over 48 Weeks: 307 Open-Label Extension Study. Adv Ther. 2024 May;41(5):2050-2069. doi: 10.1007/s12325-024-02827-8. Epub 2024 Apr 4.
PubMed 38573482Griffin M, Kelly RJ, Panse J, de Castro C, Szer J, Horneff R, Tan L, Yeh M, Peffault de Latour R. Management of acute breakthrough hemolysis with intensive pegcetacoplan dosing in patients with PNH. Blood Adv. 2024 Apr 9;8(7):1776-1786. doi: 10.1182/bloodadvances.2023011691.
PubMed 38315872Sharma V, Koprivnikar J, Drago K, Savage J, Bachelor A. Injection Site Reactions with Long-Term Pegcetacoplan Use in Patients with Paroxysmal Nocturnal Hemoglobinuria: A Brief Report. Adv Ther. 2023 Nov;40(11):5115-5129. doi: 10.1007/s12325-023-02653-4. Epub 2023 Sep 14.
PubMed 37707673
Study at a glance
From the registry record, last updated by the sponsor on 5 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegcetacoplan in the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH)
- ClinicalTrials.gov ID
- NCT03531255
- Acronym / sponsor ID
- APL2-307
- Conditions
- PNH
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 137 participants (actual)
- Lead sponsor
- Apellis Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 27 Aug 2018 (actual)
- Primary completion
- 4 Sep 2025 (actual)
- Study completion
- 19 May 2026 (actual)
- First posted
- 21 May 2018 (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.
This is an Open-label, Non-Randomized, Multi-Center Extension Study. Eligible subjects will have previously completed a pegcetacoplan study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1,080 mg pegcetacoplan administered subcutaneously | Experimental | Drug: Pegcetacoplan1,080mg pegcetacoplan administered subcutaneously twice weekly or every three days. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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.
- Vaccination against Neisseria meningitidis types A, C, W, Y and B, Streptococcus pneumoniae and Haemophilus influenzae Type B (Hib) either within 2 years prior to Day 1 dosing of this study, or within 14 days after starting treatment with pegcetacoplan. Vaccination is mandatory unless documented evidence exists that subjects are nonresponders to vaccination as evidenced by titers or display titer levels within acceptable local limits. Immunization status checks will be performed to determine whether subjects require primary or booster vaccinations.
- Willing and able to give written informed consent.
- Willing and able to self-administer pegcetacoplan (administration by caregiver will be allowed)
Key exclusion criteria (11 listed)
- Subjects who have withdrawn from a pegcetacoplan clinical study.
- Any condition that could increase the subject's risk by participating in the study.
- Any comorbidity or condition (such as malignancy) that, in the opinion of the investigator, could put the subject at increased risk or potentially confound study data.
- History or presence of hypersensitivity or idiosyncratic reaction to compounds related to the investigational product or SC administration.
- Known infection with hepatitis B, C, or HIV.
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 | 1,080 mg pegcetacoplan administered subcutaneously |
Primary outcome
Incidence and severity of treatment-emergent adverse events
Time frame: Baseline to 2 Years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Jun 2026.
- Derived from this study: PubMed 40720060de Castro C, Kelly RJ, Griffin M, Patriquin CJ, Mulherin B, Hochsmann B, Selvaratnam V, Wong RSM, Hillmen P, Horneff R, Uchendu UO, Zhang Y, Surova E, Szamosi J, de Latour RP. Efficacy and Safety Maintained up to 3 Years in Adults with Paroxysmal Nocturnal Hemoglobinuria Receiving Pegcetacoplan. Adv Ther. 2025 Sep;42(9):4641-4658. doi: 10.1007/s12325-025-03310-8. Epub 2025 Jul 28.
- Derived from this study: PubMed 38573482Patriquin CJ, Bogdanovic A, Griffin M, Kelly RJ, Maciejewski JP, Mulherin B, Peffault de Latour R, Roth A, Selvaratnam V, Szer J, Al-Adhami M, Horneff R, Tan L, Yeh M, Panse J. Safety and Efficacy of Pegcetacoplan in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria over 48 Weeks: 307 Open-Label Extension Study. Adv Ther. 2024 May;41(5):2050-2069. doi: 10.1007/s12325-024-02827-8. Epub 2024 Apr 4.
- Derived from this study: PubMed 38315872Griffin M, Kelly RJ, Panse J, de Castro C, Szer J, Horneff R, Tan L, Yeh M, Peffault de Latour R. Management of acute breakthrough hemolysis with intensive pegcetacoplan dosing in patients with PNH. Blood Adv. 2024 Apr 9;8(7):1776-1786. doi: 10.1182/bloodadvances.2023011691.
- Derived from this study: PubMed 37707673Sharma V, Koprivnikar J, Drago K, Savage J, Bachelor A. Injection Site Reactions with Long-Term Pegcetacoplan Use in Patients with Paroxysmal Nocturnal Hemoglobinuria: A Brief Report. Adv Ther. 2023 Nov;40(11):5115-5129. doi: 10.1007/s12325-023-02653-4. Epub 2023 Sep 14.
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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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
62 sites in 21 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Pegcetacoplan in people with PNH, run by Apellis Pharmaceuticals, Inc. at 62 sites in 21 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 11 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: finished, as of the sponsor's last update on 5 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 62 sites in United States, Thailand, France, Japan, Philippines and 16 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence and severity of treatment-emergent adverse events (time frame: Baseline to 2 Years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03531255, which also lists 4 linked publications.
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 5 Jun 2026.