A Phase I Study to Assess the Safety APL-2 as an Add-On to Standard of Care in Subjects with PNH | Aplastic Anemia and MDS International Foundation

ClinicalTrials.gov Identifier:



Apellis Pharmaceuticals

Contact Info

Candace Depp
(502) 241-4114


Start: November 2014

Official Title

A Phase I Study to Assess the Safety APL-2 as an Add-On to Standard of Care in Subjects with PNH


The purpose of the study is to assess the safety, tolerability and pharmacokinetics of APL-2 in subjects with paroxysmal nocturnal hemoglobinuria (PNH) who are still anemic after treatment with eculizumab (Soliris®).


If you are interested in learning more about your possible participation in this clinical trial, please complete the form. Your information will be forwarded directly to the sponsoring company.

Phase 1
Age Group: 
18 years and older
Accepts Healthy Volunteers: 
Inclusion Criteria: 
  • Male or Female

  • At least 18 years of age

  • Weigh >55 kg and have a body mass index (BMI) <38.0 kg/m2

  • 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

  • Males must agree to use protocol defined methods of contraception and agree to refrain from donating sperm for the duration of the study

  • Willing and able to give informed consent

Exclusion Criteria: 
  • 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
  • Breast-feeding women
  • History of meningococcal disease
  • No vaccination against N. meningitides 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 dosing.

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