Pegcetacoplan Długoterminowe badanie przedłużające bezpieczeństwo i skuteczność
Otwarte, nierandomizowane, wieloośrodkowe rozszerzenie badania w celu oceny długoterminowego bezpieczeństwa i skuteczności pegcetakoplanu w leczeniu napadowej nocnej hemoglobinurii (PNH)
This is an Open-label, Non-Randomized, Multi-Center Extension Study. Eligible subjects will have previously completed a pegcetacoplan study.
Przegląd badań
Typ studiów
Zapisy (Rzeczywisty)
Faza
- Faza 3
Kontakty i lokalizacje
Lokalizacje studiów
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Victoria
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Melbourne, Victoria, Australia, 3000
- Royal Melbourne Hospital
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Brussels, Belgia, 1200
- Cliniques Universitaires Saint-luc
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Roeselare, Belgia, 8800
- AZ Delta
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Sofia, Bułgaria, 1407
- Acibadem City Clinic Tokuda Hospital
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Sofia, Bułgaria, 1756
- National Specialized Hospital for Active Treatment of Haematologic Diseases
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National Capital Region
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Cebu, National Capital Region, Filipiny, 6000
- Perpetual Succour Hospital
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Lipa City, National Capital Region, Filipiny, 4217
- Mary Mediatrix Medical Centre
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Makati City, National Capital Region, Filipiny, 1229
- Makati Medical Centre
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Pasig, National Capital Region, Filipiny, 1604
- The Medical City
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Quezon City, National Capital Region, Filipiny, 1112
- St. Lukes Medical Centre
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Chalon-sur-Saône, Francja, 71100
- Hospital Center Chalon Sur Saone William Morey
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Lille, Francja, 59037
- CHRU de Lille
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Paris, Francja, 75010
- Hopital Saint-Louis
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Pierre-Bénite, Francja, 69495
- Lyon Sud Hospital Center
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Pringy, Francja, 74374
- Centre Hospitalier Annecy Genevois - Site Annecy
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Saint-Quentin, Francja, 02321
- Centre Hospitalier de Saint-Quentin
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Valencia, Hiszpania, 46026
- Hospital Universitario Politecnico La Fe
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Canary Islands
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Las Palmas de Gran Canaria, Canary Islands, Hiszpania, 35019
- Hospital Univ. de Gran Canaria Dr.Negrin
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Hong Kong, Hongkong
- Queen Mary Hospital
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Hong Kong, Hongkong
- Prince of Wales Hospital
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Okayama, Japonia, 700-8558
- Okayama University Hospital
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Tokyo, Japonia, 141-0022
- NTT Medical Center Tokyo
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Aichi-ken
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Showa-ku, Aichi-ken, Japonia, 486-8650
- Japanese Red Cross Nagoya Daini Hospital
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Nagano
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Matsumoto, Nagano, Japonia, 390-8621
- Shinshu University Hospital
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Tokyo
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Bunkyō-Ku, Tokyo, Japonia, 113-8421
- Juntendo University Hospital
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Wakayama
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Tanabe, Wakayama, Japonia, 646-8588
- Kinan Hospital
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Alberta
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Calgary, Alberta, Kanada, T2N 4Z6
- University of Calgary
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Edmonton, Alberta, Kanada, T6G 2V2
- Alberta Health Services
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Ontario
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Toronto, Ontario, Kanada, M5G 2C4
- Toronto General Hospital
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Valle del Cauca Department
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Cali, Valle del Cauca Department, Kolumbia, 760035
- IPS Centro Medico Julian Coronel SAS
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Daejeon
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Junggu, Daejeon, Korea Południowa, 35015
- Chungnam National University Hospital
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Kuala Lumpur
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Lembah Pantai, Kuala Lumpur, Malezja, 59100
- University Malaya Medical Centre
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Selangor
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Ampang, Selangor, Malezja, 68000
- Hospital Ampang
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Monterrey, Meksyk, 64460
- Hospital Universitario "José Eleuterio González", UANL
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Hamburg, Niemcy, 20246
- University Medical Center Hamburg-Eppendorf
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Baden-Wurttemberg
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Ulm, Baden-Wurttemberg, Niemcy, 89081
- Universitatsklinikum Ulm
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North Rhine-Westphalia
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Aachen, North Rhine-Westphalia, Niemcy, 52074
- Uniklinik RWTH Aachen
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Essen, North Rhine-Westphalia, Niemcy, 45147
- Essen University Hospital Department of Hematology
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Lima
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Jesús María, Lima, Peru, 15072
- Hospital Edgardo Rebagliatti
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Lima region
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Lima Cercado, Lima region, Peru, 15003
- Hospital Nacional Dos De Mayo - Centro de Investigación
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San Isidro, Lima region, Peru, 15047
- Centro de Investigacion en Hematologia Clinica del Centro Medico Corpac
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Saint Petersburg, Rosja, 197022
- Pavlov First Saint Petersburg State Medical University
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Tyumen, Rosja, 625023
- Therapeutic hospital of regional clinical hospital #1
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Belgrade, Serbia, 11000
- Clinical Center of Serbia
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Singapore, Singapur, 169608
- Singapore General Hospital
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California
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Los Angeles, California, Stany Zjednoczone, 90033
- University of Southern California
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Colorado
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Denver, Colorado, Stany Zjednoczone, 80204
- Denver Health Medical Center
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Florida
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Jacksonville, Florida, Stany Zjednoczone, 32257
- Cancer Specialists of North Florida
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Miami, Florida, Stany Zjednoczone, 33014
- Lakes Research
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Illinois
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Chicago, Illinois, Stany Zjednoczone, 60611
- Northwestern University
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Indiana
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Indianapolis, Indiana, Stany Zjednoczone, 46260
- Investigative Clinical Research of Indiana
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North Carolina
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Durham, North Carolina, Stany Zjednoczone, 27710
- Duke University Medical Center
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Ohio
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Cleveland, Ohio, Stany Zjednoczone, 44195
- Cleveland Clinic
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Tennessee
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Memphis, Tennessee, Stany Zjednoczone, 38120
- Baptist Cancer Center
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Bangkok, Tajlandia, 10400
- Phramongkutklao Hospital
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Bangkok, Tajlandia, 10400
- Ramathibodi Hospital
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Bangkok, Tajlandia, 10400
- Siriraj Hospital
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Chiang Mai, Tajlandia, 50200
- Maharaj Nakorn Chiang Mai Hospital
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Khon Kaen, Tajlandia, 40002
- Srinagarind Hospital
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Pathum Thani, Tajlandia, 12120
- Thammasat University Hospital
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Songkhla, Tajlandia, 90110
- Songklanagarind Hospital
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Leeds, Zjednoczone Królestwo, LS9 7TF
- Leeds Teaching Hospitals NHS Trust
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Kryteria uczestnictwa
Kryteria kwalifikacji
Wiek uprawniający do nauki
Akceptuje zdrowych ochotników
Płeć kwalifikująca się do nauki
Opis
Inclusion criteria:
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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.
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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.
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Willing and able to give written informed consent.
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Willing and able to self-administer pegcetacoplan (administration by caregiver will be allowed)
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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.
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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:
- 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.
Plan studiów
Jak projektuje się badanie?
Szczegóły projektu
- Główny cel: Leczenie
- Przydział: Nie dotyczy
- Model interwencyjny: Zadanie dla jednej grupy
- Maskowanie: Brak (otwarta etykieta)
Broń i interwencje
Grupa uczestników / Arm |
Interwencja / Leczenie |
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Eksperymentalny: 1,080 mg pegcetacoplan administered subcutaneously
1,080mg pegcetacoplan administered subcutaneously twice weekly or every three days. |
Complement (C3) Inhibitor |
Co mierzy badanie?
Podstawowe miary wyniku
Miara wyniku |
Opis środka |
Ramy czasowe |
|---|---|---|
|
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Ramy czasowe: From first dose of study drug up to 8 weeks from last dose (up to approximately 4 years or until pegcetacoplan development in participants with PNH was terminated)
|
An adverse event (AE) is any untoward medical occurrence associated with use of a drug in humans, whether or not considered drug related. An AE can therefore be any unfavorable and unintended sign, including a clinically significant abnormal laboratory finding, symptom, or disease temporally associated with the use of a study drug, whether or not considered related to the study drug. TEAEs were defined as those AEs that occurred or worsened in severity after the first dose of pegcetacoplan. |
From first dose of study drug up to 8 weeks from last dose (up to approximately 4 years or until pegcetacoplan development in participants with PNH was terminated)
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Number of Participants With Thromboembolic Events
Ramy czasowe: From first dose of study drug up to 8 weeks from last dose (up to approximately 4 years or until pegcetacoplan development in participants with PNH was terminated)
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From first dose of study drug up to 8 weeks from last dose (up to approximately 4 years or until pegcetacoplan development in participants with PNH was terminated)
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Change From Baseline in Hematology Parameters (Erythrocytes)
Ramy czasowe: Baseline, Weeks 168 and 264
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Baseline, Weeks 168 and 264
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Change From Baseline in Hematology Parameters (Leukocytes, Platelets and Reticulocytes)
Ramy czasowe: Baseline, Weeks 168 and 264
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Baseline, Weeks 168 and 264
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Change From Baseline in Hematology Parameters (Hematocrit)
Ramy czasowe: Baseline, Weeks 168 and 264
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Baseline, Weeks 168 and 264
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Change From Baseline in Hematology Parameters (Hemoglobin)
Ramy czasowe: Baseline, Weeks 168 and 264
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Baseline, Weeks 168 and 264
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Change From Baseline in Serum Chemistry Parameters [Alanine Aminotransferase, Alkaline Phosphatase, Aspartate Aminotransferase, Lactate Dehydrogenase (LDH)]
Ramy czasowe: Baseline, Weeks 168 and 264
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Baseline, Weeks 168 and 264
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Change From Baseline in Serum Chemistry Parameters (Bilirubin)
Ramy czasowe: Baseline, Weeks 168 and 264
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Baseline, Weeks 168 and 264
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Number of Participants With Post-Baseline Potentially Clinically Significant Abnormal Coagulation Parameters
Ramy czasowe: Baseline, Week 264
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Coagulation parameters included Activated Partial Thromboplastin Time (APTT), D-Dimer, Fibrinogen, Prothrombin Intl. Normalized Ratio and Prothrombin Time. |
Baseline, Week 264
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Number of Participants With Post-Baseline Potentially Clinically Significant Abnormal Urinalysis Parameters
Ramy czasowe: Baseline, Week 264
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Urinalysis parameters included ketones, leukocyte esterase, nitrite, occult blood, pH, protein, specific gravity and urobilinogen. |
Baseline, Week 264
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Number of Participants With Shift From Baseline in Clinically Significant Abnormalities in Electrocardiograms (ECG)
Ramy czasowe: Baseline, Week 324
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ECG measurements included heart rate, PR, QT, QRS, QTcB and QTcF. |
Baseline, Week 324
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Change From Baseline in Hemoglobin Concentration
Ramy czasowe: Baseline, Week 324
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Baseline, Week 324
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Change From Baseline in Lactate Dehydrogenase (LDH) Concentration
Ramy czasowe: Baseline, Week 324
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Baseline, Week 324
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Change From Baseline in Indirect Bilirubin Level
Ramy czasowe: Baseline, Week 324
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Baseline, Week 324
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Change From Baseline in Absolute Reticulocyte Count (ARC)
Ramy czasowe: Baseline, Week 324
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Baseline, Week 324
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Number of Participants Who Received Packed Red Blood Cell Transfusions (PRBCs)
Ramy czasowe: From first dose of study drug up to 8 weeks from last dose (up to approximately 4 years or until pegcetacoplan development in participants with PNH was terminated)
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Transfusion included whole blood, PRBCs, leukocyte-depleted red blood cells (LDPRCs), and all types of red blood cell (RBC) transfusions. |
From first dose of study drug up to 8 weeks from last dose (up to approximately 4 years or until pegcetacoplan development in participants with PNH was terminated)
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Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score
Ramy czasowe: Baseline, Week 324
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The FACIT-Fatigue scale consists of 13 questions that assess fatigue and its impact on daily activities over the past 7 days. Each question is scored on a 5-point scale from 0 ("not at all") to 4 ("very much"). For items where a higher response indicates greater fatigue, the score is reversed so that all items are scored in the same direction. The scores from the 13 items are then added together to calculate the FACIT-Fatigue total score. The total score ranges from 0 to 52, with higher scores indicating less fatigue and better functioning. |
Baseline, Week 324
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Współpracownicy i badacze
Sponsor
Publikacje i pomocne linki
Publikacje ogólne
- 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.
- 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.
- 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.
- 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.
Daty zapisu na studia
Główne daty studiów
Rozpoczęcie studiów (Rzeczywisty)
Zakończenie podstawowe (Rzeczywisty)
Ukończenie studiów (Rzeczywisty)
Daty rejestracji na studia
Pierwszy przesłany
Pierwszy przesłany, który spełnia kryteria kontroli jakości
Pierwszy wysłany (Rzeczywisty)
Aktualizacje rekordów badań
Ostatnia wysłana aktualizacja (Rzeczywisty)
Ostatnia przesłana aktualizacja, która spełniała kryteria kontroli jakości
Ostatnia weryfikacja
Więcej informacji
Terminy związane z tym badaniem
Dodatkowe istotne warunki MeSH
Inne numery identyfikacyjne badania
- APL2-307
Informacje o lekach i urządzeniach, dokumenty badawcze
Bada produkt leczniczy regulowany przez amerykańską FDA
Bada produkt urządzenia regulowany przez amerykańską FDA
produkt wyprodukowany i wyeksportowany z USA
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