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New hope for PNH patients: Long-Term drug safety confirmed

NCT ID NCT03531255

Disease control Sponsor: Apellis Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested the long-term safety of a drug called pegcetacoplan in people with PNH, a rare blood disease that destroys red blood cells. All 137 participants had already completed a prior pegcetacoplan study and continued treatment. The main goal was to track side effects over time.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

137 people

The number who actually took part.

Started

Aug 2018

Finished

May 2026

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

paroxysmal nocturnal hemoglobinuria

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

PNH

Contacts and locations

Locations

  • AZ Delta

    Roeselare, 8800, Belgium

  • Acibadem City Clinic Tokuda Hospital

    Sofia, 1407, Bulgaria

  • Alberta Health Services

    Edmonton, Alberta, T6G 2V2, Canada

  • Baptist Cancer Center

    Memphis, Tennessee, 38120, United States

  • CHRU de Lille

    Lille, 59037, France

  • Cancer Specialists of North Florida

    Jacksonville, Florida, 32257, United States

  • Centre Hospitalier Annecy Genevois - Site Annecy

    Pringy, 74374, France

  • Centre Hospitalier de Saint-Quentin

    Saint-Quentin, 02321, France

  • Centro de Investigacion en Hematologia Clinica del Centro Medico Corpac

    San Isidro, Lima region, 15047, Peru

  • Chungnam National University Hospital

    Junggu, Daejeon, 35015, South Korea

  • Cleveland Clinic

    Cleveland, Ohio, 44195, United States

  • Clinical Center of Serbia

    Belgrade, 11000, Serbia

  • Cliniques Universitaires Saint-luc

    Brussels, 1200, Belgium

  • Denver Health Medical Center

    Denver, Colorado, 80204, United States

  • Duke University Medical Center

    Durham, North Carolina, 27710, United States

  • Essen University Hospital Department of Hematology

    Essen, North Rhine-Westphalia, 45147, Germany

  • Hopital Saint-Louis

    Paris, 75010, France

  • Hospital Ampang

    Ampang, Selangor, 68000, Malaysia

  • Hospital Center Chalon Sur Saone William Morey

    Chalon-sur-Saône, 71100, France

  • Hospital Edgardo Rebagliatti

    Jesús María, Lima, 15072, Peru

  • Hospital Nacional Dos De Mayo - Centro de Investigación

    Lima Cercado, Lima region, 15003, Peru

  • Hospital Univ. de Gran Canaria Dr.Negrin

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

  • Hospital Universitario "José Eleuterio González", UANL

    Monterrey, 64460, Mexico

  • Hospital Universitario Politecnico La Fe

    Valencia, 46026, Spain

  • IPS Centro Medico Julian Coronel SAS

    Cali, Valle del Cauca Department, 760035, Colombia

  • Investigative Clinical Research of Indiana

    Indianapolis, Indiana, 46260, United States

  • Japanese Red Cross Nagoya Daini Hospital

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

  • Juntendo University Hospital

    Bunkyō-Ku, Tokyo, 113-8421, Japan

  • Kinan Hospital

    Tanabe, Wakayama, 646-8588, Japan

  • Lakes Research

    Miami, Florida, 33014, United States

  • Leeds Teaching Hospitals NHS Trust

    Leeds, LS9 7TF, United Kingdom

  • Lyon Sud Hospital Center

    Pierre-Bénite, 69495, France

  • Maharaj Nakorn Chiang Mai Hospital

    Chiang Mai, 50200, Thailand

  • Makati Medical Centre

    Makati City, National Capital Region, 1229, Philippines

  • Mary Mediatrix Medical Centre

    Lipa City, National Capital Region, 4217, Philippines

  • NTT Medical Center Tokyo

    Tokyo, 141-0022, Japan

  • National Specialized Hospital for Active Treatment of Haematologic Diseases

    Sofia, 1756, Bulgaria

  • Northwestern University

    Chicago, Illinois, 60611, United States

  • Okayama University Hospital

    Okayama, 700-8558, Japan

  • Pavlov First Saint Petersburg State Medical University

    Saint Petersburg, 197022, Russia

  • Perpetual Succour Hospital

    Cebu, National Capital Region, 6000, Philippines

  • Phramongkutklao Hospital

    Bangkok, 10400, Thailand

  • Prince of Wales Hospital

    Hong Kong, Hong Kong

  • Queen Mary Hospital

    Hong Kong, Hong Kong

  • Ramathibodi Hospital

    Bangkok, 10400, Thailand

  • Royal Melbourne Hospital

    Melbourne, Victoria, 3000, Australia

  • Shinshu University Hospital

    Matsumoto, Nagano, 390-8621, Japan

  • Singapore General Hospital

    Singapore, 169608, Singapore

  • Siriraj Hospital

    Bangkok, 10400, Thailand

  • Songklanagarind Hospital

    Songkhla, 90110, Thailand

  • Srinagarind Hospital

    Khon Kaen, 40002, Thailand

  • St. Lukes Medical Centre

    Quezon City, National Capital Region, 1112, Philippines

  • Thammasat University Hospital

    Pathum Thani, 12120, Thailand

  • The Medical City

    Pasig, National Capital Region, 1604, Philippines

  • Therapeutic hospital of regional clinical hospital #1

    Tyumen, 625023, Russia

  • Toronto General Hospital

    Toronto, Ontario, M5G 2C4, Canada

  • Uniklinik RWTH Aachen

    Aachen, North Rhine-Westphalia, 52074, Germany

  • Universitatsklinikum Ulm

    Ulm, Baden-Wurttemberg, 89081, Germany

  • University Malaya Medical Centre

    Lembah Pantai, Kuala Lumpur, 59100, Malaysia

  • University Medical Center Hamburg-Eppendorf

    Hamburg, 20246, Germany

  • University of Calgary

    Calgary, Alberta, T2N 4Z6, Canada

  • University of Southern California

    Los Angeles, California, 90033, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.