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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03225287




Registration number
NCT03225287
Ethics application status
Date submitted
17/07/2017
Date registered
21/07/2017
Date last updated
19/06/2019

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
Adverse Events and Serious Adverse Events Profile - Adverse Events and Serious Adverse Events Profile
Timepoint [1] 0 0
Safety events will be assessed at each study visit through the completion of the study (approximately 2.5 years)

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
Gender
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
N/A
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
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Investigative site - Gosford
Recruitment hospital [2] 0 0
Investigative site - 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
Illinois
Country [3] 0 0
United States of America
State/province [3] 0 0
New York
Country [4] 0 0
United States of America
State/province [4] 0 0
North Carolina
Country [5] 0 0
United States of America
State/province [5] 0 0
Texas
Country [6] 0 0
Denmark
State/province [6] 0 0
Copenhagen
Country [7] 0 0
Finland
State/province [7] 0 0
Helsinki
Country [8] 0 0
Germany
State/province [8] 0 0
Essen
Country [9] 0 0
Germany
State/province [9] 0 0
Ulm
Country [10] 0 0
Hungary
State/province [10] 0 0
Budapest
Country [11] 0 0
New Zealand
State/province [11] 0 0
Christchurch
Country [12] 0 0
New Zealand
State/province [12] 0 0
Hamilton
Country [13] 0 0
United Kingdom
State/province [13] 0 0
Leeds
Country [14] 0 0
United Kingdom
State/province [14] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Ra Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

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.
Trial website
https://clinicaltrials.gov/show/NCT03225287
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

Summary results
Other publications