Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
LOGIN
CREATE ACCOUNT
MY TRIALS
LOGIN
CREATE ACCOUNT
MY TRIALS
REGISTER TRIAL
FAQs
HINTS AND TIPS
DEFINITIONS
Register a trial
The ANZCTR website will be unavailable from 1pm until 3pm (AEDT) on Wednesday the 30th of October for website maintenance. Please be sure to log out of the system in order to avoid any loss of data.
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT03078582
Registration number
NCT03078582
Ethics application status
Date submitted
8/03/2017
Date registered
13/03/2017
Titles & IDs
Public title
Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients
Query!
Scientific title
Phase 2 Multicenter, Open-Label, Uncontrolled Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of RA101495 in Subjects With Paroxysmal Nocturnal Hemoglobinuria
Query!
Secondary ID [1]
0
0
2016-003522-16
Query!
Secondary ID [2]
0
0
RA101495-01.201
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
Paroxysmal Nocturnal Hemoglobinuria (PNH)
0
0
Query!
Condition category
Condition code
Blood
0
0
0
0
Query!
Haematological diseases
Query!
Intervention/exposure
Study type
Interventional
Query!
Description of intervention(s) / exposure
Treatment: Drugs - Zilucoplan (RA101495)
Experimental: Zilucoplan (RA101495) treatment naive - 0.3mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC (treatment naïve)
Experimental: Zilucoplan (RA101495) previously on eculizumab - 0.3mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC (previously on eculizumab)
Treatment: Drugs: Zilucoplan (RA101495)
0.3mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC
Query!
Intervention code [1]
0
0
Treatment: Drugs
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
Change-from-baseline in Serum Lactate Dehydrogenase (LDH) Level.
Query!
Assessment method [1]
0
0
The primary efficacy endpoint is the change-from-baseline in serum LDH levels during this period, defined as the mean LDH values of Weeks 6, 8, 10, and 12 minus the baseline value of LDH.
Query!
Timepoint [1]
0
0
Through Week 12 of the study
Query!
Secondary outcome [1]
0
0
Changes From Baseline in Bilirubin Values
Query!
Assessment method [1]
0
0
Changes from baseline at each of the scheduled post-baseline time-points
Query!
Timepoint [1]
0
0
Through Week 12 of the study
Query!
Secondary outcome [2]
0
0
Total Hemoglobin
Query!
Assessment method [2]
0
0
Changes from baseline at each of the scheduled post-baseline time-points
Query!
Timepoint [2]
0
0
Through Week 12 of the Study
Query!
Secondary outcome [3]
0
0
Changes From Baseline in Free Hemoglobin Values
Query!
Assessment method [3]
0
0
Changes from baseline at each of the scheduled post-baseline time-points
Query!
Timepoint [3]
0
0
Through Week 12 of the study
Query!
Secondary outcome [4]
0
0
Haptoglobin Values
Query!
Assessment method [4]
0
0
Changes from baseline at each of the scheduled post-baseline time-points
Query!
Timepoint [4]
0
0
Through Week 12 of the Study
Query!
Secondary outcome [5]
0
0
Reticulocyte Values
Query!
Assessment method [5]
0
0
Changes from baseline at each of the scheduled post-baseline time-points
Query!
Timepoint [5]
0
0
Through Week 12 of the Study
Query!
Secondary outcome [6]
0
0
Hemoglobinuria Values
Query!
Assessment method [6]
0
0
Changes from baseline at each of the scheduled post-baseline time-points; 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.
Query!
Timepoint [6]
0
0
Through Week 12 of the Study
Query!
Eligibility
Key inclusion criteria
* Diagnosis of PNH by flow cytometry
* For treatment naive patients: subjects must not have received treatment with eculizumab prior to or during the Screening Period and must have a lactate dehydrogenase (LDH) level =2 times the upper limit of normal (xULN) during Screening
* For patients who previously received eculizumab: subjects must have received treatment with eculizumab for at least 6 months prior to Screening
Query!
Minimum age
18
Years
Query!
Query!
Maximum age
No limit
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
No
Query!
Key exclusion criteria
* History of meningococcal disease
* Current systemic infection or suspicion of active bacterial infection
Query!
Study design
Purpose of the study
Treatment
Query!
Allocation to intervention
Randomised controlled trial
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Open (masking not used)
Query!
Who is / are masked / blinded?
Query!
Query!
Query!
Query!
Intervention assignment
Parallel
Query!
Other design features
Query!
Phase
Phase 2
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Completed
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
8/03/2017
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
Query!
Actual
28/03/2018
Query!
Sample size
Target
Query!
Accrual to date
Query!
Final
26
Query!
Recruitment in Australia
Recruitment state(s)
NSW
Query!
Recruitment hospital [1]
0
0
Investigative Site - Gosford
Query!
Recruitment hospital [2]
0
0
Investigative Site - Parkville
Query!
Recruitment postcode(s) [1]
0
0
- Gosford
Query!
Recruitment postcode(s) [2]
0
0
- Parkville
Query!
Recruitment outside Australia
Country [1]
0
0
Canada
Query!
State/province [1]
0
0
Ontario
Query!
Country [2]
0
0
Denmark
Query!
State/province [2]
0
0
Copenhagen
Query!
Country [3]
0
0
Finland
Query!
State/province [3]
0
0
Helsinki
Query!
Country [4]
0
0
Germany
Query!
State/province [4]
0
0
Essen
Query!
Country [5]
0
0
Germany
Query!
State/province [5]
0
0
Ulm
Query!
Country [6]
0
0
Hungary
Query!
State/province [6]
0
0
Budapest
Query!
Country [7]
0
0
New Zealand
Query!
State/province [7]
0
0
Christchurch
Query!
Country [8]
0
0
New Zealand
Query!
State/province [8]
0
0
Hamilton
Query!
Country [9]
0
0
United Kingdom
Query!
State/province [9]
0
0
Leeds
Query!
Country [10]
0
0
United Kingdom
Query!
State/province [10]
0
0
London
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Query!
Name
Ra Pharmaceuticals
Query!
Address
Query!
Country
Query!
Ethics approval
Ethics application status
Query!
Summary
Brief summary
The purpose of the study is to evaluate the safety and efficacy of zilucoplan (RA101495) in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). There will be two groups of patients in the study: the first group will include patients who have never received eculizumab for treatment of PNH. The second group will include patients who have received treatment with eculizumab for at least 6 months prior to the study. Patients will be treated with RA101495 for 12 weeks.
Query!
Trial website
https://clinicaltrials.gov/study/NCT03078582
Query!
Trial related presentations / publications
Query!
Public notes
Query!
Contacts
Principal investigator
Name
0
0
Dr. Anita Hill
Query!
Address
0
0
St James' Institute of Oncology
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
Query!
No/undecided IPD sharing reason/comment
Query!
What supporting documents are/will be available?
No Supporting Document Provided
Type
Other Details
Attachment
Study protocol
https://cdn.clinicaltrials.gov/large-docs/82/NCT03078582/Prot_000.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/82/NCT03078582/SAP_001.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT03078582