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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
Scientific title
A Multicenter, Open-label, Uncontrolled, Extension Study of RA101495 in Subjects With Paroxysmal Nocturnal Hemoglobinuria Who Have Completed a RA101495 Clinical Study
Secondary ID [1] 0 0
2016-003523-34
Secondary ID [2] 0 0
RA101495-01.202
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Paroxysmal Nocturnal Hemoglobinuria (PNH) 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 - 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.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percentage of Participants With Treatment Emergent Adverse Events (TEAEs)
Timepoint [1] 0 0
From Day 1 until the Final Study Visit (up to Month 49)
Primary outcome [2] 0 0
Percentage of Participants With Serious TEAEs
Timepoint [2] 0 0
From Day 1 until the Final Study Visit (up to Month 49)
Secondary outcome [1] 0 0
Number of Participants With Anti-drug Antibodies (ADA)
Timepoint [1] 0 0
At Day 1, Month 1, 2, 3, 6, 9, and 12
Secondary outcome [2] 0 0
Change From Baseline in Serum Lactate Dehydrogenase (LDH) Levels at Each Time Point
Timepoint [2] 0 0
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)
Secondary outcome [3] 0 0
Change From Baseline in Total Bilirubin Values at Each Time Point
Timepoint [3] 0 0
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)
Secondary outcome [4] 0 0
Change From Baseline in Total Hemoglobin Values at Each Time Point
Timepoint [4] 0 0
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)
Secondary outcome [5] 0 0
Change From Baseline in Free Hemoglobin Values at Each Time Point
Timepoint [5] 0 0
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)
Secondary outcome [6] 0 0
Change From Baseline in Haptoglobin Values at Each Time Point
Timepoint [6] 0 0
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)
Secondary outcome [7] 0 0
Change From Baseline in Reticulocytes at Each Time Point
Timepoint [7] 0 0
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)
Secondary outcome [8] 0 0
Change From Baseline in Hemoglobinuria Values at Each Time Point
Timepoint [8] 0 0
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)
Secondary outcome [9] 0 0
Plasma Concentrations of RA101495 and Its Major Metabolite(s)
Timepoint [9] 0 0
Predose: At Day 1 (Screening), Month 1, 2, 3, 6, 9, 12, and Final Study Visit (Month 49)
Secondary outcome [10] 0 0
Maximum Plasma Concentration (Cmax) of RA101495
Timepoint [10] 0 0
At Day 1, Month 1, 2, 3, 6, 9, and 12
Secondary outcome [11] 0 0
Time Corresponding to Cmax (Tmax) of RA101495
Timepoint [11] 0 0
At Day 1, Month 1, 2, 3, 6, 9, and 12
Secondary outcome [12] 0 0
Area Under the Drug Concentration-time Curve (AUC0-t) of RA101495
Timepoint [12] 0 0
At Day 1, Month 1, 2, 3, 6, 9, and 12
Secondary outcome [13] 0 0
Total Complement (CH50) Levels
Timepoint [13] 0 0
At Day 1, Month 1, 2, 3, 6, 9, and 12
Secondary outcome [14] 0 0
Change From Baseline in Sheep Red Blood Cell (sRBC) Values at Each Time Point
Timepoint [14] 0 0
Baseline, Month 1, 2, 3, 6, 9, 12 and Final Study Visit (Month 49)
Secondary outcome [15] 0 0
Change From Baseline in Wieslab Enzyme-linked Immunosorbent Assay (ELISA) Values for Alternative Complement Pathway at Each Time Point
Timepoint [15] 0 0
Baseline, Month 1, 2, 3, 6, 9, 12 and Final Study Visit (Month 49)
Secondary outcome [16] 0 0
Change From Baseline in Complement Component 5 (C5) Values at Each Time Point
Timepoint [16] 0 0
Baseline, Month 1, 2, 3, 6, 9, 12 and Final Study Visit (Month 49)

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
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Exclusion criteria:

* History of meningococcal disease
* Current systemic infection or suspicion of active bacterial infection

Study design
Purpose of the study
Treatment
Allocation to intervention
NA
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 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
17/07/2017
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
26/10/2021
Sample size
Target
Accrual to date
Final
19
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Investigative Site 3 - Gosford
Recruitment hospital [2] 0 0
Investigative Site 5 - Melbourne
Recruitment postcode(s) [1] 0 0
- Gosford
Recruitment postcode(s) [2] 0 0
- Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Texas
Country [3] 0 0
Canada
State/province [3] 0 0
Toronto
Country [4] 0 0
Finland
State/province [4] 0 0
Helsinki
Country [5] 0 0
Germany
State/province [5] 0 0
Ulm
Country [6] 0 0
Hungary
State/province [6] 0 0
Budapest
Country [7] 0 0
New Zealand
State/province [7] 0 0
Christchurch
Country [8] 0 0
New Zealand
State/province [8] 0 0
Hamilton
Country [9] 0 0
United Kingdom
State/province [9] 0 0
Leeds
Country [10] 0 0
United Kingdom
State/province [10] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Ra 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
Dr. Anita Hill
Address 0 0
St James' Institute of Oncology
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

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
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)
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.
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.
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: http://www.Vivli.org


What supporting documents are/will be available?




Results publications and other study-related documents

TypeCitations or Other Details
Journal Kulasekararaj AG, Lehtinen AE, Forsyth C, Gandhi S... [More Details]


Results are available at https://clinicaltrials.gov/study/NCT03225287