Pilot Study to Assess Safety, Preliminary Efficacy and Pharmacokinetics of S.C. Pegcetacoplan (AP… (NCT02588833) | Clinical Trial Compass
CompletedPhase 1
Pilot Study to Assess Safety, Preliminary Efficacy and Pharmacokinetics of S.C. Pegcetacoplan (APL-2) in PNH Subjects.
Hong Kong, Malaysia, New Zealand23 participantsStarted 2015-12-01
Plain-language summary
The objectives of the study are to assess the safety, tolerability, preliminary efficacy and PK of multiple subcutaneous (SC) doses of pegcetacoplan in subjects with paroxysmal nocturnal hemoglobinuria (PNH) who have not received treatment with eculizumab in the past.
An exploratory objective of the study is to assess the pharmacodynamics (PD) of multiple SC doses of pegcetacoplan when administered to PNH patients.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Male or female
* At least 18 years old (inclusive)
* Weigh \>55 kg and have a body mass index (BMI) ≤38.0 kg/m2
* Diagnosed with PNH (white blood cell (WBC) clone \>10%)
* Lactose dehydrogenase (LD) ≥2 times the upper limit of normal
* Last transfusion within 12 months prior to screening
* Platelet count of \>30,000/mm3
* Absolute neutrophil count \>cells/500 µL
* 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
* Able to provide documentary evidence of Neisseria meningitidis, Pneumococcal conjugate vaccine (multivalent) or Pneumococcal polysaccharide vaccine 23 (PCV13 or PPSV23) and Haemophilus influenzae Type B (Hib) vaccination within 2 years prior to Day 1 dosing, OR willing to receive vaccinations against Neisseria meningitidis at least two weeks prior to dosing on Day 1 with a booster on Day 57, and PCV13 and Hib vaccines at least two weeks prior to dosing on Day 1.
* Willing and able to give informed consent
Exclusion Criteria:
* Prior eculizumab (Soliris)® treatment
* Active bacterial infection
* Known infection with hepatitis B, hepatitis C or human immunodeficiency virus (HIV)
* Hereditary complement deficiency
* History of bone marrow transplantation
* Concurrent severe a…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity
Timeframe: From first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days.
2
Mean Change From Baseline in Lactate Dehydrogenase (LDH) at Day 365
Timeframe: Baseline (Day 1) and Day 365.
3
Mean Percentage Change From Baseline in LDH at Day 365
Timeframe: Baseline (Day 1) and Day 365.
4
Mean Change From Baseline in Haptoglobin at Day 365
Timeframe: Baseline (Day 1) and Day 365.
5
Mean Percentage Change From Baseline in Haptoglobin at Day 365
Timeframe: Baseline (Day 1) and Day 365.
6
Mean Change From Baseline in Hemoglobin at Day 365
Timeframe: Baseline (Day 1) and Day 365.
7
Mean Percentage Change From Baseline in Hemoglobin at Day 365