This is a Phase IIa, open-label, multiple dose, study in patients with PNH who have not received eculizumab (Soliris ®) in the past. A single cohort of subjects is planned for evaluation.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
4
Complement (C3) Inhibitor
Acibadem City Clinic MHAT Tokuda EAD Sofia
Sofia, Bulgaria
Specialized Hospital for Active Treatment of Hematologic Diseases EAD, Sofia
Sofia, Bulgaria
Klinički centar Srbije
Belgrade, Serbia
Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity
TEAEs were defined as adverse events (AE) that occurred after dosing on Day 1 and up to 30 days after the last dose of study drug. A treatment-related TEAE was defined as a TEAE with a relationship to study drug of possible, probable, or definite. TEAEs were graded according to the Common Terminology Criteria for Adverse Events (v4.03) based on: Grade 1: Mild, Grade 2: Moderate, Grade 3: Severe, Grade 4: Life-threatening, Grade 5: Death related to AE.
Time frame: From Day 1 to 30 days after the last dose (approximately 56 weeks)
Mean Change From Baseline in Lactate Dehydrogenase (LDH) Level
Serum chemistry assessments of LDH were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the treatment period.
Time frame: Baseline and Day 365
Mean Change From Baseline in Haptoglobin Level
Serum chemistry assessments of haptoglobin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the treatment period.
Time frame: Baseline and Day 365
Mean Change From Baseline in Hemoglobin (Hb) Level
Hematology assessments of Hb were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the treatment period.
Time frame: Baseline and Day 365
Mean Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Score
The FACIT-Fatigue scale is a 13 item Likert scaled instrument where the subject was presented with 13 statements and asked to indicate their response as it applied to the past 7 days. The 5 possible responses were 'Not at all' (0), 'A little bit (1), 'Somewhat' (2), 'Quite a bit' (3) and 'Very much' (4). With 13 statements the total score had a range of 0 to 52. Higher score corresponds to a higher quality of life (QoL).
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Time frame: Baseline and Day 365
Mean Change From Baseline in Absolute Reticulocyte Count (ARC) Level
Hematology assessments of ARC were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the treatment period.
Time frame: Baseline and Day 365
Mean Change From Baseline in Total Bilirubin Level
Serum chemistry assessments of total bilirubin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the treatment period.
Time frame: Baseline and Day 365
Mean Number of Red Blood Cell (RBC) Transfusions Per Month
The number of on-study RBC transfusions was monitored throughout the treatment period.
Time frame: From Day 1 to Day 364
Mean Change From Baseline in Linear Analog Scale Assessment (LASA) Score for QoL
The LASA consists of 3 items, where the respondents were asked to rate their perceived level of functioning. Specific domains included activity level, ability to carry out daily activities, and an item for overall QoL. Their level of functioning was reported on a 0 to 100 scale with 0 indicates "As low as could be" and 100 indicates "As high as could be". The combined score ranged from 0 to 300, with higher scores corresponding to a higher QoL.
Time frame: Baseline and Day 365
Mean Serum Concentrations of Pegcetacoplan
Serum concentrations of pegcetacoplan at Day 365 are presented.
Time frame: Day 365
Mean Area Under the Serum Concentration Versus Time Curve From Time 0 to the Last Measurable Concentration at the End of the Study (AUCtotal)
The AUCtotal of pegcetacoplan was estimated using a non-compartmental approach.
Time frame: Blood samples were collected predose and at least 2.5 hours post dose on Day 1 and predose on Days 2 up to Day 365.
Mean Maximum Observed Predose Serum Concentration During the Study (Ctrough,Max,Total)
The Ctrough,max,total of pegcetacoplan was estimated using a non-compartmental approach.
Time frame: Blood samples were collected predose and at least 2.5 hours post dose on Day 1 and predose on Days 2 up to Day 365.