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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT02588833
Registration number
NCT02588833
Ethics application status
Date submitted
27/10/2015
Date registered
28/10/2015
Date last updated
11/01/2021
Titles & IDs
Public title
Pilot Study to Assess Safety, Preliminary Efficacy and Pharmacokinetics of S.C. Pegcetacoplan (APL-2) in PNH Subjects.
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Scientific title
A Phase Ib, Open Label, Multiple Ascending Dose, Pilot Study to Assess the Safety, Preliminary Efficacy and Pharmacokinetics of Subcutaneously Administered APL-2 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)
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Secondary ID [1]
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APL2-CP-PNH-204
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Universal Trial Number (UTN)
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Trial acronym
PADDOCK
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Paroxysmal Nocturnal Hemoglobinuria
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Condition category
Condition code
Blood
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Haematological diseases
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Pegcetacoplan
Experimental: Cohort 1 - 180 mg pegcetacoplan/day
Experimental: Cohort 2 - 270 mg pegcetacoplan/day
Treatment: Drugs: Pegcetacoplan
Complement (C3) Inhibitor
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity
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Assessment method [1]
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TEAEs were defined as adverse events (AEs) that occurred after dosing on Day 1 and up to 30 days after the last dose of study drug. A treatment-related TEAE is defined as a TEAE with a relationship to study drug of probably, possibly, unlikely, or unrelated. A serious adverse event (SAE) is defined as any AE that resulted in death; was life-threatening; required hospitalization or prolongation of existing hospitalization; resulted in persistent or significant incapacity or substantial disruption of ability to conduct normal life functions; was a congenital anomaly or birth defect. TEAEs were graded according to 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.
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Timepoint [1]
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From first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days.
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Primary outcome [2]
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Mean Change From Baseline in Lactate Dehydrogenase (LDH) at Day 365
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Assessment method [2]
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Serum chemistry assessments of LDH were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. LDH results were summarized for Cohort 2 only.
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Timepoint [2]
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Baseline (Day 1) and Day 365.
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Primary outcome [3]
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Mean Percentage Change From Baseline in LDH at Day 365
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Assessment method [3]
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Serum chemistry assessments of LDH were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. LDH results were summarized for Cohort 2 only.
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Timepoint [3]
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Baseline (Day 1) and Day 365.
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Primary outcome [4]
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Mean Change From Baseline in Haptoglobin at Day 365
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Assessment method [4]
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Serum chemistry assessments of haptoglobin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. Haptoglobin results were summarized for Cohort 2 only.
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Timepoint [4]
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Baseline (Day 1) and Day 365.
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Primary outcome [5]
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Mean Percentage Change From Baseline in Haptoglobin at Day 365
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Assessment method [5]
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Serum chemistry assessments of haptoglobin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. Haptoglobin results were summarized for Cohort 2 only.
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Timepoint [5]
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Baseline (Day 1) and Day 365.
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Primary outcome [6]
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Mean Change From Baseline in Hemoglobin at Day 365
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Assessment method [6]
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Hematology assessments of hemoglobin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. Hemoglobin results were summarized for Cohort 2 only.
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Timepoint [6]
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Baseline (Day 1) and Day 365.
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Primary outcome [7]
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Mean Percentage Change From Baseline in Hemoglobin at Day 365
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Assessment method [7]
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Hematology assessments of hemoglobin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. Hemoglobin results were summarized for Cohort 2 only.
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Timepoint [7]
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Baseline (Day 1) and Day 365.
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Secondary outcome [1]
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Mean Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at Day 365
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Assessment method [1]
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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). There are 13 statements with the total score ranging from 0 to 52 and higher scores indicating better quality of life. The FACIT-Fatigue Scale results were summarized for Cohort 2 only.
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Timepoint [1]
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Baseline (Day 1) and Day 365.
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Secondary outcome [2]
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Mean Change From Baseline in Absolute Reticulocyte Count (ARC) at Day 365
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Assessment method [2]
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Hematology assessments of ARC were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. ARC results were summarized for Cohort 2 only.
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Timepoint [2]
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Baseline (Day 1) and Day 365.
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Secondary outcome [3]
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Mean Percentage Change From Baseline in ARC at Day 365
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Assessment method [3]
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Hematology assessments of ARC were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. ARC results were summarized for Cohort 2 only.
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Timepoint [3]
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Baseline (Day 1) and Day 365.
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Secondary outcome [4]
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Mean Change From Baseline in Total Bilirubin at Day 365
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Assessment method [4]
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Serum chemistry assessments of total bilirubin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the 3 parts of the treatment period. Total bilirubin results were summarized for Cohort 2 only.
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Timepoint [4]
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Baseline (Day 1) and Day 365.
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Secondary outcome [5]
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Mean Percentage Change From Baseline in Total Bilirubin at Day 365
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Assessment method [5]
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Serum chemistry assessments of total bilirubin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the 3 parts of the treatment period. Total bilirubin results were summarized for Cohort 2 only.
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Timepoint [5]
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Baseline (Day 1) and Day 365.
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Secondary outcome [6]
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Number of Subjects Receiving Red Blood Cell (RBC) Transfusions
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Assessment method [6]
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The number of on-study RBC transfusions were monitored throughout the treatment period.
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Timepoint [6]
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From Day 1 up to Day 533.
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Eligibility
Key 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
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Minimum age
18
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key 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 aplastic anemia (SAA), defined as currently receiving
immunosuppressive therapy for SAA including but not limited to cyclosporin A,
tacrolimus, mycophenolate mofetil or anti-thymocyte globulin.
- Participation in any other investigational drug trial or exposure to another
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
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Non-randomised trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 1
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
1/12/2015
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
26/08/2019
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Sample size
Target
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Accrual to date
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Final
23
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Recruitment in Australia
Recruitment state(s)
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Recruitment outside Australia
Country [1]
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Hong Kong
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State/province [1]
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Hong Kong
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Country [2]
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Malaysia
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State/province [2]
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Selangor
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Country [3]
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New Zealand
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State/province [3]
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Waikato
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Country [4]
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Thailand
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State/province [4]
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Bangkok
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Apellis Pharmaceuticals, Inc.
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief 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.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT02588833
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Federico Grossi, MD, PhD
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Address
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Apellis Pharmaceuticals, Inc.
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT02588833
Download to PDF