Myelodysplastic Syndrome Clinical Trial
A Phase I Study to Assess the Safety of Pegcetacoplan (APL-2) as an Add-On to Standard of Care in Subjects With PNH
This study will be the initial exploration of pegcetacoplan in patients with PNH. The assessments of the safety, tolerability, PK, and PD following administration of single and multiples doses of pegcetacoplan will guide decisions to further develop the drug.
Male or Female
At least 18 years of age
Weigh >55 kg
Diagnosed with PNH
On treatment with eculizumab (Soliris®) for at least 3 months
Hb < 10 g/dL at screening OR have received at least one transfusion within 12 months prior to screening
Platelet count of >30,000/mm3
Absolute neutrophil count > 500/mm3
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 (see below)
Males with female partners of child bearing potential must agree to use protocol defined methods of contraception (see below) and agree to refrain from donating sperm for the duration of the study
Willing and able to give informed consent
Active bacterial infection
Known infection with hepatitis B, C or HIV
Hereditary complement deficiency
History of bone marrow transplantation
Participation in any other investigational drug trial or exposure to other 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
Creatinine clearance (CrCl) < 50 mL/min (Cockcroft-Gault formula) at screening
History of meningococcal disease
No vaccination against N. meningitidis types A, C, W, Y and B (administered as two separate vaccinations), Pneumococcal conjugate vaccine or Pneumococcal polysaccharide vaccine 23 (PCV13 or PPSV23, respectively) and Haemophilus influenzae Type B (Hib) vaccination within 2 years prior to Day 1 (Visit 2) dosing.
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There are 7 Locations for this study
Los Angeles California, 90033, United States
Miami Lakes Florida, 33014, United States
Louisville Kentucky, 40202, United States
Baltimore Maryland, 21231, United States
Las Vegas Nevada, 89135, United States
Durham North Carolina, 27710, United States
Cleveland Ohio, 44195, United States
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