Pegcetacoplan Long Term Safety and Efficacy Extension Study

NCT03531255 | Active Not Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: APL2-307
• Study Type: interventional
• Target: 160
• Locations: 21 countries
• Sites: 62

Brief Summary

This is an Open-label, Non-Randomized, Multi-Center Extension Study. Eligible subjects will have previously completed a pegcetacoplan study.

Conditions

PNH

Outcome Measures

Primary Outcomes (1)

• Incidence and severity of treatment-emergent adverse events

  Baseline to 2 Years

Study Arms (1)

1,080 mg pegcetacoplan administered subcutaneously

EXPERIMENTAL

1,080mg pegcetacoplan administered subcutaneously twice weekly or every three days.

Drug: Pegcetacoplan

Interventions

Pegcetacoplan DRUG

Complement (C3) Inhibitor

1,080 mg pegcetacoplan administered subcutaneously

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• Apellis Pharmaceuticals, Inc.: lead

Study Sites (62)

University of Southern California

Los Angeles, California, 90033, United States

Location

Denver Health Medical Center

Denver, Colorado, 80204, United States

Location

Cancer Specialists of North Florida

Jacksonville, Florida, 32257, United States

Location

Lakes Research

Miami, Florida, 33014, United States

Location

Northwestern University

Chicago, Illinois, 60611, United States

Location

Investigative Clinical Research of Indiana

Indianapolis, Indiana, 46260, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

Baptist Cancer Center

Memphis, Tennessee, 38120, United States

Location

Royal Melbourne Hospital

Melbourne, Victoria, 3000, Australia

Location

Cliniques Universitaires Saint-luc

Brussels, 1200, Belgium

Location

AZ Delta

Roeselare, 8800, Belgium

Location

Acibadem City Clinic Tokuda Hospital

Sofia, 1407, Bulgaria

Location

National Specialized Hospital for Active Treatment of Haematologic Diseases

Sofia, 1756, Bulgaria

Location

University of Calgary

Calgary, Alberta, T2N 4Z6, Canada

Location

Alberta Health Services

Edmonton, Alberta, T6G 2V2, Canada

Location

Toronto General Hospital

Toronto, Ontario, M5G 2C4, Canada

Location

IPS Centro Medico Julian Coronel SAS

Cali, Valle del Cauca Department, 760035, Colombia

Location

Hospital Center Chalon Sur Saone William Morey

Chalon-sur-Saône, 71100, France

Location

CHRU de Lille

Lille, 59037, France

Location

Hopital Saint-Louis

Paris, 75010, France

Location

Lyon Sud Hospital Center

Pierre-Bénite, 69495, France

Location

Centre Hospitalier Annecy Genevois - Site Annecy

Pringy, 74374, France

Location

Centre Hospitalier de Saint-Quentin

Saint-Quentin, 02321, France

Location

Universitatsklinikum Ulm

Ulm, Baden-Wurttemberg, 89081, Germany

Location

Uniklinik RWTH Aachen

Aachen, North Rhine-Westphalia, 52074, Germany

Location

Essen University Hospital Department of Hematology

Essen, North Rhine-Westphalia, 45147, Germany

Location

University Medical Center Hamburg-Eppendorf

Hamburg, 20246, Germany

Location

Prince of Wales Hospital

Hong Kong, Hong Kong

Location

Queen Mary Hospital

Hong Kong, Hong Kong

Location

Japanese Red Cross Nagoya Daini Hospital

Showa-ku, Aichi-ken, 486-8650, Japan

Location

Shinshu University Hospital

Matsumoto, Nagano, 390-8621, Japan

Location

Juntendo University Hospital

Bunkyō-Ku, Tokyo, 113-8421, Japan

Location

Kinan Hospital

Tanabe, Wakayama, 646-8588, Japan

Location

Okayama University Hospital

Okayama, 700-8558, Japan

Location

NTT Medical Center Tokyo

Tokyo, 141-0022, Japan

Location

University Malaya Medical Centre

Lembah Pantai, Kuala Lumpur, 59100, Malaysia

Location

Hospital Ampang

Ampang, Selangor, 68000, Malaysia

Location

Hospital Universitario "José Eleuterio González", UANL

Monterrey, 64460, Mexico

Location

Hospital Nacional Dos De Mayo - Centro de Investigación

Lima Cercado, Lima region, 15003, Peru

Location

Centro de Investigacion en Hematologia Clinica del Centro Medico Corpac

San Isidro, Lima region, 15047, Peru

Location

Hospital Edgardo Rebagliatti

Jesús María, Lima, 15072, Peru

Location

Perpetual Succour Hospital

Cebu, National Capital Region, 6000, Philippines

Location

Mary Mediatrix Medical Centre

Lipa City, National Capital Region, 4217, Philippines

Location

Makati Medical Centre

Makati City, National Capital Region, 1229, Philippines

Location

The Medical City

Pasig, National Capital Region, 1604, Philippines

Location

St. Lukes Medical Centre

Quezon City, National Capital Region, 1112, Philippines

Location

Pavlov First Saint Petersburg State Medical University

Saint Petersburg, 197022, Russia

Location

Therapeutic hospital of regional clinical hospital #1

Tyumen, 625023, Russia

Location

Clinical Center of Serbia

Belgrade, 11000, Serbia

Location

Singapore General Hospital

Singapore, 169608, Singapore

Location

Chungnam National University Hospital

Junggu, Daejeon, 35015, South Korea

Location

Hospital Univ. de Gran Canaria Dr.Negrin

Las Palmas de Gran Canaria, Canary Islands, 35019, Spain

Location

Hospital Universitario Politecnico La Fe

Valencia, 46026, Spain

Location

Phramongkutklao Hospital

Bangkok, 10400, Thailand

Location

Ramathibodi Hospital

Bangkok, 10400, Thailand

Location

Siriraj Hospital

Bangkok, 10400, Thailand

Location

Maharaj Nakorn Chiang Mai Hospital

Chiang Mai, 50200, Thailand

Location

Srinagarind Hospital

Khon Kaen, 40002, Thailand

Location

Thammasat University Hospital

Pathum Thani, 12120, Thailand

Location

Songklanagarind Hospital

Songkhla, 90110, Thailand

Location

Leeds Teaching Hospitals NHS Trust

Leeds, LS9 7TF, United Kingdom

Location

Related Publications (4)

• 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.

  PMID: 40720060 DERIVED

• 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.

  PMID: 38573482 DERIVED

• 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.

  PMID: 38315872 DERIVED

• 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.

  PMID: 37707673 DERIVED

MeSH Terms

Interventions

pegcetacoplan

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 9, 2018
• First Posted: May 21, 2018
• Study Start: August 27, 2018
• Primary Completion: June 1, 2025
• Study Completion: June 1, 2025
• Last Updated: March 28, 2025

Record last verified: 2025-03

Locations

SE (1); BE (2); BU (2); FR (6); JP (6); PH (5); ME (1); US (9); GB (1); CO (1); MY (2); TH (7); SG (1); AU (1); PE (3); RU (2); KR (1); ES (2); DE (4); HK (2); CA (3)