Pegcetacoplan Long Term Safety and Efficacy Extension Study

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.

Study Overview

• Status: Active, not recruiting
• Conditions: PNH
• Intervention / Treatment: Drug: Pegcetacoplan

• Study Type: Interventional
• Enrollment (Estimated): 160
• Phase: Phase 3

Contacts and Locations

Study Locations

• Australia
  • Victoria
    • Melbourne, Victoria, Australia, 3000
      • Royal Melbourne Hospital
• Belgium
  • Brussels, Belgium, 1200
    • Cliniques Universitaires Saint-Luc
  • Roeselare, Belgium, 8800
    • AZ Delta
• Bulgaria
  • Sofia, Bulgaria, 1407
    • Acibadem City Clinic Tokuda Hospital
  • Sofia, Bulgaria, 1756
    • National Specialized Hospital for Active Treatment of Haematologic Diseases
• Canada
  • Alberta
    • Calgary, Alberta, Canada, T2N 4Z6
      • University of Calgary
    • Edmonton, Alberta, Canada, T6G 2V2
      • Alberta Health services
  • Ontario
    • Toronto, Ontario, Canada, M5G 2C4
      • Toronto General Hospital
• Colombia
  • Valle Del Cauca
    • Cali, Valle Del Cauca, Colombia, 760035
      • IPS Centro Medico Julian Coronel SAS
• France
  • Chalon-sur-Saône, France, 71100
    • Hospital Center Chalon Sur Saone William Morey
  • Lille, France, 59037
    • CHRU de Lille
  • Paris, France, 75010
    • Hôpital Saint-Louis
  • Pierre-Bénite, France, 69495
    • Lyon Sud Hospital Center
  • Pringy, France, 74374
    • Centre Hospitalier Annecy Genevois - Site Annecy
  • Saint-Quentin, France, 02321
    • Centre Hospitalier de Saint-Quentin
• Germany
  • Hamburg, Germany, 20246
    • University Medical Center Hamburg-Eppendorf
  • BW
    • Ulm, BW, Germany, 89081
      • Universitätsklinikum Ulm
  • NRW
    • Aachen, NRW, Germany, 52074
      • Uniklinik RWTH Aachen
    • Essen, NRW, Germany, 45147
      • Essen University Hospital Department of Hematology
• Hong Kong
  • Hong Kong, Hong Kong
    • Queen Mary Hospital
  • Hong Kong, Hong Kong
    • Prince of Wales Hospital
• Japan
  • Okayama, Japan, 700-8558
    • Okayama University Hospital
  • Tokyo, Japan, 141-0022
    • NTT Medical Center Tokyo
  • Aichi
    • Showa-ku, Aichi, Japan, 486-8650
      • Japanese Red Cross Nagoya Daini Hospital
  • Nagano
    • Matsumoto, Nagano, Japan, 390-8621
      • Shinshu University Hospital
  • Tokyo
    • Bunkyō-Ku, Tokyo, Japan, 113-8421
      • Juntendo University Hospital
  • Wakayama
    • Tanabe, Wakayama, Japan, 646-8588
      • Kinan Hospital
• Korea, Republic of
  • Daejeon
    • Junggu, Daejeon, Korea, Republic of, 35015
      • Chungnam National University Hospital
• Malaysia
  • Kuala Lumpur
    • Lembah Pantai, Kuala Lumpur, Malaysia, 59100
      • University Malaya Medical Centre
  • Selangor
    • Ampang, Selangor, Malaysia, 68000
      • Hospital Ampang
• Mexico
  • Monterrey, Mexico, 64460
    • Hospital Universitario "José Eleuterio González", UANL
• Peru
  • Lima
    • Jesús María, Lima, Peru, 15072
      • Hospital Edgardo Rebagliatti
    • Lima Cercado, Lima, Peru, 15003
      • Hospital Nacional Dos De Mayo - Centro de Investigación
    • San Isidro, Lima, Peru, 15047
      • Centro de Investigacion en Hematologia Clinica del Centro Medico Corpac
• Philippines
  • Metro Manila
    • Cebu, Metro Manila, Philippines, 6000
      • Perpetual Succour Hospital
    • Lipa City, Metro Manila, Philippines, 4217
      • Mary Mediatrix Medical Centre
    • Makati City, Metro Manila, Philippines, 1229
      • Makati Medical Centre
    • Pasig City, Metro Manila, Philippines, 1604
      • The Medical City
    • Quezon City, Metro Manila, Philippines, 1112
      • St. Lukes Medical Centre
• Russian Federation
  • Saint Petersburg, Russian Federation, 197022
    • Pavlov First Saint Petersburg State Medical University
  • Tyumen, Russian Federation, 625023
    • Therapeutic hospital of regional clinical hospital #1
• Serbia
  • Belgrade, Serbia, 11000
    • Clinical Center of Serbia
• Singapore
  • Singapore, Singapore, 169608
    • Singapore General Hospital
• Spain
  • Valencia, Spain, 46026
    • Hospital Universitario Politecnico La Fe
  • Islas Canarias
    • Las Palmas De Gran Canaria, Islas Canarias, Spain, 35019
      • Hospital Univ. de Gran Canaria Dr.Negrin
• Thailand
  • Bangkok, Thailand, 10400
    • Phramongkutklao Hospital
  • Bangkok, Thailand, 10400
    • Ramathibodi Hospital
  • Bangkok, Thailand, 10400
    • Siriraj Hospital
  • Chiang Mai, Thailand, 50200
    • Maharaj Nakorn Chiang Mai Hospital
  • Khon Kaen, Thailand, 40002
    • Srinagarind Hospital
  • Pathum Thani, Thailand, 12120
    • Thammasat University Hospital
  • Songkhla, Thailand, 90110
    • Songklanagarind Hospital
• United Kingdom
  • Leeds, United Kingdom, LS9 7TF
    • Leeds Teaching Hospitals NHS Trust
• United States
  • California
    • Los Angeles, California, United States, 90033
      • University of Southern California
  • Colorado
    • Denver, Colorado, United States, 80204
      • Denver Health Medical Center
  • Florida
    • Jacksonville, Florida, United States, 32257
      • Cancer Specialists of North Florida
    • Miami, Florida, United States, 33014
      • Lakes Research
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Northwestern University
  • Indiana
    • Indianapolis, Indiana, United States, 46260
      • Investigative Clinical Research of Indiana
  • North Carolina
    • Durham, North Carolina, United States, 27710
      • Duke University Medical Center
  • Ohio
    • Cleveland, Ohio, United States, 44195
      • Cleveland Clinic
  • Tennessee
    • Memphis, Tennessee, United States, 38120
      • Baptist Cancer Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion criteria:

1. 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.

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:

1. Subjects who have withdrawn from a pegcetacoplan clinical study.
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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 1,080 mg pegcetacoplan administered subcutaneously; 1,080mg pegcetacoplan administered subcutaneously twice weekly or every three days.	Drug: Pegcetacoplan; Complement (C3) Inhibitor

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Incidence and severity of treatment-emergent adverse events	Baseline to 2 Years

Collaborators and Investigators

• Sponsor: Apellis Pharmaceuticals, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): August 27, 2018
• Primary Completion (Estimated): June 1, 2025
• Study Completion (Estimated): June 1, 2025

Study Registration Dates

• First Submitted: May 9, 2018
• First Submitted That Met QC Criteria: May 9, 2018
• First Posted (Actual): May 21, 2018

Study Record Updates

• Last Update Posted (Actual): July 3, 2023
• Last Update Submitted That Met QC Criteria: June 29, 2023
• Last Verified: June 1, 2023

More Information

Terms related to this study

• Other Study ID Numbers: APL2-307

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No