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:

NCT02264639

Company

Apellis Pharmaceuticals

Contact Info

Candace Depp
(502) 241-4114
pnh@apellis.com

Dates

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

Purpose

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®).

Instructions

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.

Status: 
Recruiting
Phase: 
Phase 1
Gender: 
Female
Male
Age Group: 
18 years and older
Accepts Healthy Volunteers: 
No
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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