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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/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.
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)
Secondary ID [1] 0 0
APL2-CP-PNH-204
Universal Trial Number (UTN)
Trial acronym
PADDOCK
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Paroxysmal Nocturnal Hemoglobinuria 0 0
Condition category
Condition code
Blood 0 0 0 0
Haematological diseases

Intervention/exposure
Study type
Interventional
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
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity
Timepoint [1] 0 0
From first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days.
Primary outcome [2] 0 0
Mean Change From Baseline in Lactate Dehydrogenase (LDH) at Day 365
Timepoint [2] 0 0
Baseline (Day 1) and Day 365.
Primary outcome [3] 0 0
Mean Percentage Change From Baseline in LDH at Day 365
Timepoint [3] 0 0
Baseline (Day 1) and Day 365.
Primary outcome [4] 0 0
Mean Change From Baseline in Haptoglobin at Day 365
Timepoint [4] 0 0
Baseline (Day 1) and Day 365.
Primary outcome [5] 0 0
Mean Percentage Change From Baseline in Haptoglobin at Day 365
Timepoint [5] 0 0
Baseline (Day 1) and Day 365.
Primary outcome [6] 0 0
Mean Change From Baseline in Hemoglobin at Day 365
Timepoint [6] 0 0
Baseline (Day 1) and Day 365.
Primary outcome [7] 0 0
Mean Percentage Change From Baseline in Hemoglobin at Day 365
Timepoint [7] 0 0
Baseline (Day 1) and Day 365.
Secondary outcome [1] 0 0
Mean Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at Day 365
Timepoint [1] 0 0
Baseline (Day 1) and Day 365.
Secondary outcome [2] 0 0
Mean Change From Baseline in Absolute Reticulocyte Count (ARC) at Day 365
Timepoint [2] 0 0
Baseline (Day 1) and Day 365.
Secondary outcome [3] 0 0
Mean Percentage Change From Baseline in ARC at Day 365
Timepoint [3] 0 0
Baseline (Day 1) and Day 365.
Secondary outcome [4] 0 0
Mean Change From Baseline in Total Bilirubin at Day 365
Timepoint [4] 0 0
Baseline (Day 1) and Day 365.
Secondary outcome [5] 0 0
Mean Percentage Change From Baseline in Total Bilirubin at Day 365
Timepoint [5] 0 0
Baseline (Day 1) and Day 365.
Secondary outcome [6] 0 0
Number of Subjects Receiving Red Blood Cell (RBC) Transfusions
Timepoint [6] 0 0
From Day 1 up to Day 533.

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
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/12/2015
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
26/08/2019
Sample size
Target
Accrual to date
Final
23
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
Hong Kong
State/province [1] 0 0
Hong Kong
Country [2] 0 0
Malaysia
State/province [2] 0 0
Selangor
Country [3] 0 0
New Zealand
State/province [3] 0 0
Waikato
Country [4] 0 0
Thailand
State/province [4] 0 0
Bangkok

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Apellis Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Federico Grossi, MD, PhD
Address 0 0
Apellis Pharmaceuticals, Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results are available at https://clinicaltrials.gov/study/NCT02588833