A Phase IIa Study to Assess the Safety, Efficacy, and Pharmacokinetics of Subcutaneously Administered Pegcetacoplan (APL-2) in Subjects With PNH
NCT ID: NCT03593200
Last Updated: 2020-12-22
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE2
4 participants
INTERVENTIONAL
2018-08-16
2019-10-22
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Experimental: Cohort 1
270 mg/day (up to 360 mg/day from Day 29) from Day 1 to Day 364\*
Pegcetacoplan
Complement (C3) Inhibitor
Interventions
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Pegcetacoplan
Complement (C3) Inhibitor
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Diagnosed with PNH (white blood cell (WBC) clone \>10%)
* Lactose dehydrogenase (LD) ≥2 times the upper limit of normal
* Screening Ferritin ≥ normal and Total Iron Binding Capacity (TIBC) \< LLN based on central lab reference ranges. If a subject is receiving iron supplements at screening, the investigator must ensure that his/her dose has been stable for 8 weeks prior to enrolment and must be maintained throughout the study
* Last transfusion within 12 months prior to screening
* Platelet count of \>30,000/mm3 at the screening visit
* Absolute neutrophil count \>500/ mm3 at the screening visit
* Women of child-bearing potential (WOCBP) must have a negative pregnancy test at screening and must agree to use protocol defined methods of contraception for the duration of the study
* Males must agree to use protocol defined methods of contraception and agree to refrain from donating sperm for the duration of the study
* Vaccination against Neisseria meningitides types A, C, W, Y and B, Streptococcus pneumoniae and Haemophilus influenzae Type B (Hib) either within 2 years prior to Day 1 dosing, or within 14 days after starting treatment with pegcetacoplan. Unless documented evidence exists that subjects are non-responders to vaccination as evidenced by titers or display titer levels within acceptable local limits
* Willing and able to give informed consent
Exclusion Criteria
* Active bacterial infection
* Hereditary complement deficiency
* History of bone marrow transplantation
* 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
* Participation in any other investigational drug trial or exposure to another investigational agent, device or procedure within 30 days
* Evidence of QTcF prolongation defined as \>450 ms for males and \>470 ms for females at screening
* Breast-feeding women
* History of meningococcal disease
18 Years
ALL
No
Sponsors
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Apellis Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Federico Grossi, MD, PhD
Role: STUDY_DIRECTOR
Study Director
Locations
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Acibadem City Clinic MHAT Tokuda EAD Sofia
Sofia, , Bulgaria
Specialized Hospital for Active Treatment of Hematologic Diseases EAD, Sofia
Sofia, , Bulgaria
Klinički centar Srbije
Belgrade, , Serbia
Countries
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References
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Wong RSM, Pullon HWH, Amine I, Bogdanovic A, Deschatelets P, Francois CG, Ignatova K, Issaragrisil S, Niparuck P, Numbenjapon T, Roman E, Sathar J, Xu R, Al-Adhami M, Tan L, Tse E, Grossi FV. Inhibition of C3 with pegcetacoplan results in normalization of hemolysis markers in paroxysmal nocturnal hemoglobinuria. Ann Hematol. 2022 Sep;101(9):1971-1986. doi: 10.1007/s00277-022-04903-x. Epub 2022 Jul 22.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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APL2-202
Identifier Type: -
Identifier Source: org_study_id