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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT03225287
Registration number
NCT03225287
Ethics application status
Date submitted
17/07/2017
Date registered
21/07/2017
Titles & IDs
Public title
Extension Study of RA101495 for Patients With PNH Who Have Completed a Zilucoplan (RA101495) Clinical Study
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Scientific title
A Multicenter, Open-label, Uncontrolled, Extension Study of RA101495 in Subjects With Paroxysmal Nocturnal Hemoglobinuria Who Have Completed a RA101495 Clinical Study
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Secondary ID [1]
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2016-003523-34
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Secondary ID [2]
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RA101495-01.202
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Paroxysmal Nocturnal Hemoglobinuria (PNH)
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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 - Zilucoplan (RA101495)
Experimental: Zilucoplan (RA101495) - Subjects will continue to receive the final maintenance dose they were receiving in the qualifying study
Treatment: Drugs: Zilucoplan (RA101495)
Subjects will continue to receive the final maintenance dose they were receiving in the qualifying study.
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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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Percentage of Participants With Treatment Emergent Adverse Events (TEAEs)
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Assessment method [1]
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TEAEs were defined as an AE that occurs after a participant's initial treatment zilucoplan start for this study (RA101495-01.202) that was not present at the time of treatment start, or an AE that increases in severity after treatment start in this study, if the event was present at the time of treatment start.
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Timepoint [1]
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From Day 1 until the Final Study Visit (up to Month 49)
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Primary outcome [2]
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Percentage of Participants With Serious TEAEs
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Assessment method [2]
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Serious Adverse event (SAE) was defined as any untoward medical occurrence that:• results in death, • is life-threatening threatening (note that this refers to an event in which the participant was at risk of death at the time of the event; it does not refer to an event that hypothetically might have caused death if it were more severe), • requires hospitalization or prolongation of existing hospitalization, • results in persistent or significant disability/incapacity, and • results in a congenital anomaly/birth defect.
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Timepoint [2]
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From Day 1 until the Final Study Visit (up to Month 49)
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Secondary outcome [1]
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Number of Participants With Anti-drug Antibodies (ADA)
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Assessment method [1]
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Blood samples collection were planned to analyze for the presence/absence of ADAs to zilucoplan for immunogenicity assessments.
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Timepoint [1]
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At Day 1, Month 1, 2, 3, 6, 9, and 12
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Secondary outcome [2]
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Change From Baseline in Serum Lactate Dehydrogenase (LDH) Levels at Each Time Point
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Assessment method [2]
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Serum LDH levels were measure of intravascular hemolysis. As high level of LDH in the blood was indicative of hemolysis in participants with PNH.
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Timepoint [2]
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Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49)
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Secondary outcome [3]
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Change From Baseline in Total Bilirubin Values at Each Time Point
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Assessment method [3]
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Total Bilirubin was monitored for signs and symptoms of hepatic or biliary dysfunction.
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Timepoint [3]
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Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49)
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Secondary outcome [4]
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Change From Baseline in Total Hemoglobin Values at Each Time Point
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Assessment method [4]
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Total Hemoglobin Values were analyzed for hematology assessments.
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Timepoint [4]
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Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49)
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Secondary outcome [5]
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Change From Baseline in Free Hemoglobin Values at Each Time Point
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Assessment method [5]
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Free Hemoglobin Values were analyzed for hematology assessments.
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Timepoint [5]
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Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49)
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Secondary outcome [6]
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Change From Baseline in Haptoglobin Values at Each Time Point
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Assessment method [6]
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Haptoglobin values were analyzed for hematology assessments.
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Timepoint [6]
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Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49)
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Secondary outcome [7]
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Change From Baseline in Reticulocytes at Each Time Point
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Assessment method [7]
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Reticulocytes values were analyzed for hematology assessments.
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Timepoint [7]
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Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49)
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Secondary outcome [8]
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Change From Baseline in Hemoglobinuria Values at Each Time Point
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Assessment method [8]
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Hemoglobinuria was assessed using a urine colorimetric scoring system with a score of 1 through 10 where 1 represents no hemoglobinuria and 10 represents maximum hemoglobinuria.
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Timepoint [8]
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Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, 48 and Final Study Visit (Month 49)
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Secondary outcome [9]
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Plasma Concentrations of RA101495 and Its Major Metabolite(s)
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Assessment method [9]
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Blood samples of RA101495 (zilucoplan) and its metabolites (RA102758 and RA103488) were collected for Plasma concentration analysis.
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Timepoint [9]
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Predose: At Day 1 (Screening), Month 1, 2, 3, 6, 9, 12, and Final Study Visit (Month 49)
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Secondary outcome [10]
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Maximum Plasma Concentration (Cmax) of RA101495
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Assessment method [10]
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Cmax is the maximum plasma concentration.
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Timepoint [10]
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At Day 1, Month 1, 2, 3, 6, 9, and 12
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Secondary outcome [11]
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Time Corresponding to Cmax (Tmax) of RA101495
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Assessment method [11]
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tmax is the time to corresponding Cmax.
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Timepoint [11]
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At Day 1, Month 1, 2, 3, 6, 9, and 12
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Secondary outcome [12]
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Area Under the Drug Concentration-time Curve (AUC0-t) of RA101495
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Assessment method [12]
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AUC0-t is area under the drug concentration-time curves.
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Timepoint [12]
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At Day 1, Month 1, 2, 3, 6, 9, and 12
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Secondary outcome [13]
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Total Complement (CH50) Levels
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Assessment method [13]
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Blood samples collection were planned to assess complement (CH50) levels. The planned analysis of CH50 was not performed because the CH50 assay was not able to be validated due to lack of reproducibility of the manufacturer's kits.
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Timepoint [13]
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At Day 1, Month 1, 2, 3, 6, 9, and 12
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Secondary outcome [14]
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Change From Baseline in Sheep Red Blood Cell (sRBC) Values at Each Time Point
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Assessment method [14]
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Blood samples were collected for measurement of sRBC lysis for the Classical Complement Pathways.
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Timepoint [14]
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Baseline, Month 1, 2, 3, 6, 9, 12 and Final Study Visit (Month 49)
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Secondary outcome [15]
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Change From Baseline in Wieslab Enzyme-linked Immunosorbent Assay (ELISA) Values for Alternative Complement Pathway at Each Time Point
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Assessment method [15]
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Blood samples were collected for measurement of membrane attack complex (MAC) by Wieslab ELISA for alternative complement pathway.
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Timepoint [15]
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Baseline, Month 1, 2, 3, 6, 9, 12 and Final Study Visit (Month 49)
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Secondary outcome [16]
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Change From Baseline in Complement Component 5 (C5) Values at Each Time Point
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Assessment method [16]
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Blood samples were collected for measurement of Complement component 5 (C5) levels.
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Timepoint [16]
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Baseline, Month 1, 2, 3, 6, 9, 12 and Final Study Visit (Month 49)
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Eligibility
Key inclusion criteria
* Completion of a qualifying Ra Pharmaceuticals sponsored zilucoplan (RA101495) PNH study
* Evidence of ongoing clinical benefit in the opinion of the Investigator
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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
Exclusion criteria:
* History of meningococcal disease
* Current systemic infection or suspicion of active bacterial infection
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Study design
Purpose of the study
Treatment
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Allocation to intervention
NA
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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 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Stopped early
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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
17/07/2017
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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
26/10/2021
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Sample size
Target
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Accrual to date
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Final
19
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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Investigative Site 3 - Gosford
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Recruitment hospital [2]
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Investigative Site 5 - Melbourne
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Recruitment postcode(s) [1]
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- Gosford
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Recruitment postcode(s) [2]
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- Melbourne
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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California
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Country [2]
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United States of America
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State/province [2]
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Texas
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Country [3]
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Canada
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State/province [3]
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Toronto
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Country [4]
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Finland
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State/province [4]
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Helsinki
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Country [5]
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Germany
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State/province [5]
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Ulm
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Country [6]
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Hungary
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State/province [6]
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Budapest
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Country [7]
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New Zealand
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State/province [7]
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Christchurch
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Country [8]
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New Zealand
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State/province [8]
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Hamilton
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Country [9]
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United Kingdom
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State/province [9]
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Leeds
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Country [10]
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United Kingdom
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State/province [10]
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London
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Ra 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 purpose of this study is to enable continued access to zilucoplan (RA101495) for patients with paroxysmal nocturnal hemoglobinuria (PNH) after they complete a zilucoplan clinical study.
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Trial website
https://clinicaltrials.gov/study/NCT03225287
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Trial related presentations / publications
Kulasekararaj AG, Lehtinen AE, Forsyth C, Gandhi S, Griffin M, Korper S, Mikala G, Muus P, Overgaard U, Patriquin CJ, Pullon H, Shen YM, Spearing R, Szer J, De la Borderie G, Duda PW, Farzaneh-Far R, Ragunathan S, Sayegh CE, Vadysirisack DD, Schrezenmeier H. Phase II trials of zilucoplan in paroxysmal nocturnal hemoglobinuria. Haematologica. 2024 Mar 1;109(3):929-935. doi: 10.3324/haematol.2022.281780. No abstract available.
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Public notes
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Contacts
Principal investigator
Name
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Dr. Anita Hill
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Address
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St James' Institute of Oncology
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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
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
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What data in particular will be shared?
Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a prespecified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.
Supporting document/s available: Study protocol, Statistical analysis plan (SAP), Clinical study report (CSR)
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When will data be available (start and end dates)?
Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe or global development is discontinued, and 18 months after trial completion.
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Available to whom?
Qualified researchers may request access to anonymized IPD and redacted study documents which may include: raw datasets, analysis-ready datasets, study protocol, blank case report form, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal.
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Available for what types of analyses?
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How or where can data be obtained?
IPD available at link: http://www.Vivli.org
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What supporting documents are/will be available?
No Supporting Document Provided
Type
Other Details
Attachment
Study protocol
https://cdn.clinicaltrials.gov/large-docs/87/NCT03225287/Prot_000.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/87/NCT03225287/SAP_001.pdf
Results publications and other study-related documents
Type
Citations or Other Details
Journal
Kulasekararaj AG, Lehtinen AE, Forsyth C, Gandhi S...
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More Details
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Results are available at
https://clinicaltrials.gov/study/NCT03225287