A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH
NCT ID: NCT04085601
Last Updated: 2022-10-21
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
53 participants
INTERVENTIONAL
2019-08-27
2021-06-23
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Standard of Care (SOC) excluding complement inhibitors
No interventions assigned to this group
1,080mg APL-2 administered subcutaneously twice weekly
APL-2
Complement (C3) Inhibitor
Interventions
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APL-2
Complement (C3) Inhibitor
Eligibility Criteria
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Inclusion Criteria
* Have LDH ≥1.5 x ULN at the screening visit.
* Have PNH diagnosis, confirmed by high sensitivity flow cytometry (granulocyte or monocyte clone \>10%).
* Have Hb less than the lower limit of normal (LLN) at the screening visit.
* Have ferritin greater than/equal to the LLN, or total iron binding capacity (TIBC) less than/equal to ULN at the screening visit, based on central laboratory reference ranges. If a subject is receiving iron supplements at screening, the Investigator must ensure that the subject's dose has been stable for 4 weeks prior to screening, and it must be maintained throughout the study. Subjects not receiving iron at screening must not start iron supplementation during the course of the study.
* Body mass index (BMI) ≤ 35 kg/m2 at the screening visit.
* Have a platelet count of \>50,000/mm3 at the screening visit.
* Have an absolute neutrophil count \>500/mm3 at the screening visit.
Exclusion Criteria
* Hereditary complement deficiency.
* History of bone marrow transplantation.
* Concomitant use of any of the following medications is prohibited if not on a stable regimen for the time period indicated below prior to screening:
* Erythropoietin or immunosuppressants for at least 8 weeks
* Systemic corticosteroids for at least 4 weeks
* Vitamin K antagonists (eg, warfarin) with a stable international normalized ratio (INR) for at least 4 weeks
* Iron supplements, vitamin B12, or folic acid for at least 4 weeks
* Low-molecular-weight heparin for at least 4 weeks
18 Years
ALL
No
Sponsors
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Apellis Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Locations
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Julian Coronel Medical Center
Cali, , Colombia
Research Center of the Colombian Clinical Life Cancer Foundation
Medellín, , Colombia
Queen Mary Hospital
Hong Kong, , Hong Kong
Princess Margaret Hospital
Kwai Chung, , Hong Kong
Prince and Wales Hospital
Shatin, , Hong Kong
Hospital Ampang
Ampang, Selangor, Malaysia
University Malaya Medical Centre
Kuala Lumpur, , Malaysia
Hospital Universitario Dr.Jose Eleuterio Gonzalez
Monterrey, , Mexico
Hospital Cayetano Heredia
San Martín de Porres, Lima region, Peru
Hospital Cayetano Heredia
Jesús María, Lima, Peru
Hospital Nacional Dos de Mayo
Lima, , Peru
Baguio General Hospital
Benguet, , Philippines
Perpetual Succour Hospital
Cebu City, , Philippines
Mary Mediatrix Medical Center
Lipa City, , Philippines
Makati Medical Centre
Makati City, , Philippines
The Medical City
Pasig, , Philippines
St. Lukes Medical Centre
Quezon City, , Philippines
Independent Public Clinical Hospital
Lubin, , Poland
Institute of Hematology and Transfusiology
Warsaw, , Poland
EMC Medical Institute
Wroclaw, , Poland
Clinical Centre of Serbia
Belgrade, , Serbia
Singapore General Hospital (SGH)
Singapore, , Singapore
Ramathibodi Hospital
Bangkok, , Thailand
Siriraj Hospital
Bangkok, , Thailand
Hospital for Tropical disease
Bangkok, , Thailand
Phramongkutklao Hospital
Bangkok, , Thailand
Maharaj Nakorn Chiang Mai Hospital
Chiang Mai, , Thailand
Srinagaring Hospital
Khon Kaen, , Thailand
Thammasat University Hospital
Pathum Thani, , Thailand
Songklanagaring Hospital
Songkhla, , Thailand
Countries
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References
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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.
Panse J, Daguindau N, Okuyama S, Peffault de Latour R, Schafhausen P, Straetmans N, Al-Adhami M, Persson E, Wong RSM. Improvements in hematologic markers and decreases in fatigue with pegcetacoplan for patients with paroxysmal nocturnal hemoglobinuria and mild or moderate anemia (hemoglobin >/=10 g/dL) who had received eculizumab or were naive to complement inhibitors. PLoS One. 2024 Jul 29;19(7):e0306407. doi: 10.1371/journal.pone.0306407. eCollection 2024.
Mulherin BP, Yeh M, Al-Adhami M, Dingli D. Normalization of Hemoglobin, Lactate Dehydrogenase, and Fatigue in Patients with Paroxysmal Nocturnal Hemoglobinuria Treated with Pegcetacoplan. Drugs R D. 2024 Jun;24(2):169-177. doi: 10.1007/s40268-024-00463-9. Epub 2024 May 10.
Fishman J, Wilson K, Drzewiecka A, Pochopien M, Dingli D. The cost-effectiveness of pegcetacoplan in complement treatment-naive adults with paroxysmal nocturnal hemoglobinuria in the USA. J Comp Eff Res. 2023 Oct;12(10):e230055. doi: 10.57264/cer-2023-0055. Epub 2023 Sep 1.
Wojciechowski P, Wdowiak M, Hakimi Z, Wilson K, Fishman J, Nazir J, Toumi M. Mapping the EORTC QLQ-C30 onto the EQ-5D-5L index for patients with paroxysmal nocturnal hemoglobinuria in France. J Comp Eff Res. 2023 May;12(5):e220178. doi: 10.57264/cer-2022-0178. Epub 2023 Apr 13.
Wong RSM, Navarro-Cabrera JR, Comia NS, Goh YT, Idrobo H, Kongkabpan D, Gomez-Almaguer D, Al-Adhami M, Ajayi T, Alvarenga P, Savage J, Deschatelets P, Francois C, Grossi F, Dumagay T. Pegcetacoplan controls hemolysis in complement inhibitor-naive patients with paroxysmal nocturnal hemoglobinuria. Blood Adv. 2023 Jun 13;7(11):2468-2478. doi: 10.1182/bloodadvances.2022009129.
Wong R, Fishman J, Wilson K, Yeh M, Al-Adhami M, Zion A, Yee CW, Huynh L, Duh MS. Comparative Effectiveness of Pegcetacoplan Versus Ravulizumab and Eculizumab in Complement Inhibitor-Naive Patients with Paroxysmal Nocturnal Hemoglobinuria: A Matching-Adjusted Indirect Comparison. Adv Ther. 2023 Apr;40(4):1571-1589. doi: 10.1007/s12325-023-02438-9. Epub 2023 Feb 7.
Del Pozo Martin Y. 2021 ASH annual meeting. Lancet Haematol. 2022 Feb;9(2):e92-e93. doi: 10.1016/S2352-3026(21)00384-7. Epub 2021 Dec 16. No abstract available.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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APL2-308
Identifier Type: -
Identifier Source: org_study_id
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