PNH Clinical Trial
Official title:
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)
Verified date | June 2023 |
Source | Apellis Pharmaceuticals, Inc. |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
This is an Open-label, Non-Randomized, Multi-Center Extension Study. Eligible subjects will have previously completed a pegcetacoplan study.
Status | Active, not recruiting |
Enrollment | 160 |
Est. completion date | June 2025 |
Est. primary completion date | June 2025 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years and older |
Eligibility | 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. |
Country | Name | City | State |
---|---|---|---|
Australia | Royal Melbourne Hospital | Melbourne | Victoria |
Belgium | Cliniques Universitaires Saint-luc | Brussels | |
Belgium | AZ Delta | Roeselare | |
Bulgaria | Acibadem City Clinic Tokuda Hospital | Sofia | |
Bulgaria | National Specialized Hospital for Active Treatment of Haematologic Diseases | Sofia | |
Canada | University of Calgary | Calgary | Alberta |
Canada | Alberta Health Services | Edmonton | Alberta |
Canada | Toronto General Hospital | Toronto | Ontario |
Colombia | IPS Centro Medico Julian Coronel SAS | Cali | Valle Del Cauca |
France | 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 | |
Germany | Uniklinik RWTH Aachen | Aachen | NRW |
Germany | Essen University Hospital Department of Hematology | Essen | NRW |
Germany | University Medical Center Hamburg-Eppendorf | Hamburg | |
Germany | Universitatsklinikum Ulm | Ulm | BW |
Hong Kong | Prince of Wales Hospital | Hong Kong | |
Hong Kong | Queen Mary Hospital | Hong Kong | |
Japan | Juntendo University Hospital | Bunkyo-Ku | Tokyo |
Japan | Shinshu University Hospital | Matsumoto | Nagano |
Japan | Okayama University Hospital | Okayama | |
Japan | Japanese Red Cross Nagoya Daini Hospital | Showa-ku | Aichi |
Japan | Kinan Hospital | Tanabe | Wakayama |
Japan | NTT Medical Center Tokyo | Tokyo | |
Korea, Republic of | Chungnam National University Hospital | Junggu | Daejeon |
Malaysia | Hospital Ampang | Ampang | Selangor |
Malaysia | University Malaya Medical Centre | Lembah Pantai | Kuala Lumpur |
Mexico | Hospital Universitario "José Eleuterio González", UANL | Monterrey | |
Peru | Hospital Edgardo Rebagliatti | Jesús María | Lima |
Peru | Hospital Nacional Dos De Mayo - Centro de Investigación | Lima Cercado | Lima |
Peru | Centro de Investigacion en Hematologia Clinica del Centro Medico Corpac | San Isidro | Lima |
Philippines | Perpetual Succour Hospital | Cebu | Metro Manila |
Philippines | Mary Mediatrix Medical Centre | Lipa City | Metro Manila |
Philippines | Makati Medical Centre | Makati City | Metro Manila |
Philippines | The Medical City | Pasig City | Metro Manila |
Philippines | St. Lukes Medical Centre | Quezon City | Metro Manila |
Russian Federation | Pavlov First Saint Petersburg State Medical University | Saint Petersburg | |
Russian Federation | Therapeutic hospital of regional clinical hospital #1 | Tyumen | |
Serbia | Clinical Center of Serbia | Belgrade | |
Singapore | Singapore General Hospital | Singapore | |
Spain | Hospital Univ. de Gran Canaria Dr.Negrin | Las Palmas De Gran Canaria | Islas Canarias |
Spain | Hospital Universitario Politecnico La Fe | Valencia | |
Thailand | 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 | |
United Kingdom | Leeds Teaching Hospitals NHS Trust | Leeds | |
United States | Northwestern University | Chicago | Illinois |
United States | Cleveland Clinic | Cleveland | Ohio |
United States | Denver Health Medical Center | Denver | Colorado |
United States | Duke University Medical Center | Durham | North Carolina |
United States | Investigative Clinical Research of Indiana | Indianapolis | Indiana |
United States | Cancer Specialists of North Florida | Jacksonville | Florida |
United States | University of Southern California | Los Angeles | California |
United States | Baptist Cancer Center | Memphis | Tennessee |
United States | Lakes Research | Miami | Florida |
Lead Sponsor | Collaborator |
---|---|
Apellis Pharmaceuticals, Inc. |
United States, Australia, Belgium, Bulgaria, Canada, Colombia, France, Germany, Hong Kong, Japan, Korea, Republic of, Malaysia, Mexico, Peru, Philippines, Russian Federation, Serbia, Singapore, Spain, Thailand, United Kingdom,
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---|---|---|---|---|
Primary | Incidence and severity of treatment-emergent adverse events | Baseline to 2 Years |
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