Status:
COMPLETED
A Phase I Study to Assess the Safety of Pegcetacoplan (APL-2) as an Add-On to Standard of Care in Subjects With PNH
Lead Sponsor:
Apellis Pharmaceuticals, Inc.
Conditions:
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Eligibility:
All Genders
18+ years
Phase:
PHASE1
Brief Summary
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 pegc...
Eligibility Criteria
Inclusion
- 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
Exclusion
- 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. 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.
Key Trial Info
Start Date :
February 23 2015
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 22 2018
Estimated Enrollment :
9 Patients enrolled
Trial Details
Trial ID
NCT02264639
Start Date
February 23 2015
End Date
October 22 2018
Last Update
January 8 2021
Active Locations (7)
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1
University of Southern California Norris Comprehensive Cancer Center
Los Angeles, California, United States, 90033
2
Lakes Research
Miami Lakes, Florida, United States, 33014
3
University of Lousiville
Louisville, Kentucky, United States, 40202
4
John Hopkins Hospital
Baltimore, Maryland, United States, 21231