Pegcetacoplan Long Term Safety and Efficacy Extension Study
NCT ID: NCT03531255
Last Updated: 2025-03-28
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE3
160 participants
INTERVENTIONAL
2018-08-27
2025-06-30
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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1,080 mg pegcetacoplan administered subcutaneously
1,080mg pegcetacoplan administered subcutaneously twice weekly or every three days.
Pegcetacoplan
Complement (C3) Inhibitor
Interventions
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Pegcetacoplan
Complement (C3) Inhibitor
Eligibility Criteria
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Inclusion Criteria
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.
2. 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.
3. Willing and able to give written informed consent.
4. Willing and able to self-administer pegcetacoplan (administration by caregiver will be allowed)
5. 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.
6. 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.
Exclusion Criteria
2. Any condition that could increase the subject's risk by participating in the study.
3. 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.
4. History or presence of hypersensitivity or idiosyncratic reaction to compounds related to the investigational product or SC administration.
5. Known infection with hepatitis B, C, or HIV.
6. Hereditary complement deficiency.
7. History of bone marrow transplant.
8. 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.
9. History of meningococcal disease.
10. Concomitant treatment with any complement inhibitor (eg, eculizumab, ravulizumab).
11. Pregnancy, breastfeeding, or positive pregnancy test.
18 Years
ALL
No
Sponsors
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Apellis Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Locations
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University of Southern California
Los Angeles, California, United States
Denver Health Medical Center
Denver, Colorado, United States
Cancer Specialists of North Florida
Jacksonville, Florida, United States
Lakes Research
Miami, Florida, United States
Northwestern University
Chicago, Illinois, United States
Investigative Clinical Research of Indiana
Indianapolis, Indiana, United States
Duke University Medical Center
Durham, North Carolina, United States
Cleveland Clinic
Cleveland, Ohio, United States
Baptist Cancer Center
Memphis, Tennessee, United States
Royal Melbourne Hospital
Melbourne, Victoria, Australia
Cliniques Universitaires Saint-luc
Brussels, , Belgium
AZ Delta
Roeselare, , Belgium
Acibadem City Clinic Tokuda Hospital
Sofia, , Bulgaria
National Specialized Hospital for Active Treatment of Haematologic Diseases
Sofia, , Bulgaria
University of Calgary
Calgary, Alberta, Canada
Alberta Health Services
Edmonton, Alberta, Canada
Toronto General Hospital
Toronto, Ontario, Canada
IPS Centro Medico Julian Coronel SAS
Cali, Valle del Cauca Department, Colombia
Hospital Center Chalon Sur Saone William Morey
Chalon-sur-Saône, , France
CHRU de Lille
Lille, , France
Hopital Saint-Louis
Paris, , France
Lyon Sud Hospital Center
Pierre-Bénite, , France
Centre Hospitalier Annecy Genevois - Site Annecy
Pringy, , France
Centre Hospitalier de Saint-Quentin
Saint-Quentin, , France
Universitatsklinikum Ulm
Ulm, Baden-Wurttemberg, Germany
Uniklinik RWTH Aachen
Aachen, North Rhine-Westphalia, Germany
Essen University Hospital Department of Hematology
Essen, North Rhine-Westphalia, Germany
University Medical Center Hamburg-Eppendorf
Hamburg, , Germany
Prince of Wales Hospital
Hong Kong, , Hong Kong
Queen Mary Hospital
Hong Kong, , Hong Kong
Japanese Red Cross Nagoya Daini Hospital
Showa-ku, Aichi-ken, Japan
Shinshu University Hospital
Matsumoto, Nagano, Japan
Juntendo University Hospital
Bunkyō-Ku, Tokyo, Japan
Kinan Hospital
Tanabe, Wakayama, Japan
Okayama University Hospital
Okayama, , Japan
NTT Medical Center Tokyo
Tokyo, , Japan
University Malaya Medical Centre
Lembah Pantai, Kuala Lumpur, Malaysia
Hospital Ampang
Ampang, Selangor, Malaysia
Hospital Universitario "José Eleuterio González", UANL
Monterrey, , Mexico
Hospital Nacional Dos De Mayo - Centro de Investigación
Lima Cercado, Lima region, Peru
Centro de Investigacion en Hematologia Clinica del Centro Medico Corpac
San Isidro, Lima region, Peru
Hospital Edgardo Rebagliatti
Jesús María, Lima, Peru
Perpetual Succour Hospital
Cebu, National Capital Region, Philippines
Mary Mediatrix Medical Centre
Lipa City, National Capital Region, Philippines
Makati Medical Centre
Makati City, National Capital Region, Philippines
The Medical City
Pasig, National Capital Region, Philippines
St. Lukes Medical Centre
Quezon City, National Capital Region, Philippines
Pavlov First Saint Petersburg State Medical University
Saint Petersburg, , Russia
Therapeutic hospital of regional clinical hospital #1
Tyumen, , Russia
Clinical Center of Serbia
Belgrade, , Serbia
Singapore General Hospital
Singapore, , Singapore
Chungnam National University Hospital
Junggu, Daejeon, South Korea
Hospital Univ. de Gran Canaria Dr.Negrin
Las Palmas de Gran Canaria, Canary Islands, Spain
Hospital Universitario Politecnico La Fe
Valencia, , Spain
Phramongkutklao Hospital
Bangkok, , Thailand
Ramathibodi Hospital
Bangkok, , Thailand
Siriraj Hospital
Bangkok, , Thailand
Maharaj Nakorn Chiang Mai Hospital
Chiang Mai, , Thailand
Srinagarind Hospital
Khon Kaen, , Thailand
Thammasat University Hospital
Pathum Thani, , Thailand
Songklanagarind Hospital
Songkhla, , Thailand
Leeds Teaching Hospitals NHS Trust
Leeds, , United Kingdom
Countries
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References
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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.
Patriquin 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.
Griffin 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.
Sharma 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.
Other Identifiers
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APL2-307
Identifier Type: -
Identifier Source: org_study_id
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