Pegcetacoplan Long Term Safety and Efficacy Extension Study

Phase 3

Active, not recruiting

• Conditions: PNH
• Interventions: Drug: Pegcetacoplan

• Registration Number: NCT03531255
• Lead Sponsor: Apellis Pharmaceuticals, Inc.

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2018-05-09
• Study First Submitted QC: 2018-05-09
• Study First Posted: 2018-05-21
• Study Start: 2018-08-27
• Status Verified: 2025-03
• Last Update Submitted: 2025-03-27
• Last Update Posted: 2025-03-28
• Primary Completion: 2025-06
• Study Completion: 2025-06

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 160

Inclusion Criteria

Not provided

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 Subjects who have withdrawn from a pegcetacoplan clinical study.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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
1,080 mg pegcetacoplan administered subcutaneously	Pegcetacoplan	1,080mg pegcetacoplan administered subcutaneously twice weekly or every three days.

Primary Outcome Measures

Name	Time	Method
Incidence and severity of treatment-emergent adverse events	Baseline to 2 Years

Trial Locations

Locations (62)

Acibadem City Clinic Tokuda Hospital; 🇧🇬 Sofia, Bulgaria

Baptist Cancer Center; 🇺🇸 Memphis, Tennessee, United States

Japanese Red Cross Nagoya Daini Hospital; 🇯🇵 Showa-ku, Aichi, Japan

Lakes Research; 🇺🇸 Miami, Florida, United States

Cancer Specialists of North Florida; 🇺🇸 Jacksonville, Florida, United States

Investigative Clinical Research of Indiana; 🇺🇸 Indianapolis, Indiana, United States

Duke University Medical Center; 🇺🇸 Durham, North Carolina, United States

National Specialized Hospital for Active Treatment of Haematologic Diseases; 🇧🇬 Sofia, Bulgaria

CHRU de Lille; 🇫🇷 Lille, France

Royal Melbourne Hospital; 🇦🇺 Melbourne, Victoria, Australia

Cliniques Universitaires Saint-luc; 🇧🇪 Brussels, Belgium

Okayama University Hospital; 🇯🇵 Okayama, Japan

Denver Health Medical Center; 🇺🇸 Denver, Colorado, United States

University of Calgary; 🇨🇦 Calgary, Alberta, Canada

Centre Hospitalier Annecy Genevois - Site Annecy; 🇫🇷 Pringy, France

Chungnam National University Hospital; 🇰🇷 Junggu, Daejeon, Korea, Republic of

Hopital Saint-Louis; 🇫🇷 Paris, France

University Medical Center Hamburg-Eppendorf; 🇩🇪 Hamburg, Germany

Hospital Universitario Politecnico La Fe; 🇪🇸 Valencia, Spain

Hospital Ampang; 🇲🇾 Ampang, Selangor, Malaysia

Essen University Hospital Department of Hematology; 🇩🇪 Essen, NRW, Germany

Toronto General Hospital; 🇨🇦 Toronto, Ontario, Canada

Makati Medical Centre; 🇵🇭 Makati City, Metro Manila, Philippines

Leeds Teaching Hospitals NHS Trust; 🇬🇧 Leeds, United Kingdom

NTT Medical Center Tokyo; 🇯🇵 Tokyo, Japan

Siriraj Hospital; 🇹🇭 Bangkok, Thailand

Hospital Univ. de Gran Canaria Dr.Negrin; 🇪🇸 Las Palmas De Gran Canaria, Islas Canarias, Spain

Srinagarind Hospital; 🇹🇭 Khon Kaen, Thailand

Juntendo University Hospital; 🇯🇵 Bunkyō-Ku, Tokyo, Japan

University Malaya Medical Centre; 🇲🇾 Lembah Pantai, Kuala Lumpur, Malaysia

Hospital Universitario "José Eleuterio González", UANL; 🇲🇽 Monterrey, Mexico

Singapore General Hospital; 🇸🇬 Singapore, Singapore

Songklanagarind Hospital; 🇹🇭 Songkhla, Thailand

IPS Centro Medico Julian Coronel SAS; 🇨🇴 Cali, Valle Del Cauca, Colombia

Queen Mary Hospital; 🇭🇰 Hong Kong, Hong Kong

Hospital Edgardo Rebagliatti; 🇵🇪 Jesús María, Lima, Peru

Perpetual Succour Hospital; 🇵🇭 Cebu, Metro Manila, Philippines

Phramongkutklao Hospital; 🇹🇭 Bangkok, Thailand

Prince of Wales Hospital; 🇭🇰 Hong Kong, Hong Kong

Thammasat University Hospital; 🇹🇭 Pathum Thani, Thailand

Hospital Nacional Dos De Mayo - Centro de Investigación; 🇵🇪 Lima Cercado, Lima, Peru

The Medical City; 🇵🇭 Pasig City, Metro Manila, Philippines

Ramathibodi Hospital; 🇹🇭 Bangkok, Thailand

Centre Hospitalier de Saint-Quentin; 🇫🇷 Saint-Quentin, France

University of Southern California; 🇺🇸 Los Angeles, California, United States

Northwestern University; 🇺🇸 Chicago, Illinois, United States

Alberta Health Services; 🇨🇦 Edmonton, Alberta, Canada

Universitatsklinikum Ulm; 🇩🇪 Ulm, BW, Germany

Lyon Sud Hospital Center; 🇫🇷 Pierre-Bénite, France

Kinan Hospital; 🇯🇵 Tanabe, Wakayama, Japan

Shinshu University Hospital; 🇯🇵 Matsumoto, Nagano, Japan

Centro de Investigacion en Hematologia Clinica del Centro Medico Corpac; 🇵🇪 San Isidro, Lima, Peru

Mary Mediatrix Medical Centre; 🇵🇭 Lipa City, Metro Manila, Philippines

Pavlov First Saint Petersburg State Medical University; 🇷🇺 Saint Petersburg, Russian Federation

Clinical Center of Serbia; 🇷🇸 Belgrade, Serbia

St. Lukes Medical Centre; 🇵🇭 Quezon City, Metro Manila, Philippines

Therapeutic hospital of regional clinical hospital #1; 🇷🇺 Tyumen, Russian Federation

Maharaj Nakorn Chiang Mai Hospital; 🇹🇭 Chiang Mai, Thailand

Hospital Center Chalon Sur Saone William Morey; 🇫🇷 Chalon-sur-Saône, France

Uniklinik RWTH Aachen; 🇩🇪 Aachen, NRW, Germany

AZ Delta; 🇧🇪 Roeselare, Belgium

Cleveland Clinic; 🇺🇸 Cleveland, Ohio, United States