Details

Details

Title An Open Label, Single and Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of APL-2 as an Add-On to Standard of Care in Subjects with Paroxysmal Nocturnal Hemoglobinuria (PNH).

IRB APLS1Z14

CC 14-1275

Hospital Main Campus

Disease Paroxysmal Nocturnal Hemoglobinuria (PNH)

Drug APL-2

Description

Description

Primary Objectives
  • To assess the safety, tolerability and pharmacokinetics of single and multiple subcutaneous (SC) doses of APL-2 in subjects with paroxysmal nocturnal hemoglobinuria (PNH) who are still anemic (pre-transfusion Hb < 10 g/dL) after treatment with eculizumab (Soliris®).
Exploratory Objective
  • To assess the pharmacodynamics (PD) of single and multiple SC doses of APL-2 when administered to PNH patients.
Inclusion Criteria

Inclusion Criteria

  1. Male or Female
  2. At least 18 years of age
  3. Weigh >55kg
  4. Diagnosed with PNH
  5. On treatment with eculizumab (Soliris®) for at least 3 months
  6. Hb < 10 g/dL at screening or have received at least one transfusion within 12 months prior to screening
  7. Platelet count of >30,000/mm3
  8. Absolute neutrophil count >cells/500 μL
  9. 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)
  10. 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
  11. Willing and able to give informed consent
Exclusion Criteria

Exclusion Criteria

  1. Active bacterial infection
  2. Known infection with hepatitis B, C or HIV
  3. Hereditary complement deficiency
  4. History of bone marrow transplantation
  5. Participation in any other investigational drug trial or exposure to other investigational agent, device or procedure within 30 days
  6. Evidence of QTcF prolongation defined as > 450 ms for males and > 470 ms for females at screening
  7. Creatinine clearance (CrCl) < 50 mL/min (Cockcroft-Gault formula) at screening
  8. Breast-feeding women
  9. History of meningococcal disease
  10. No vaccination against N. meningitidis