Study Stopped
After careful consideration, UCB has decided to no longer pursue PNH as a potential indication for zilucoplan.
Extension Study of RA101495 for Patients With PNH Who Have Completed a Zilucoplan (RA101495) Clinical Study
A Multicenter, Open-label, Uncontrolled, Extension Study of RA101495 in Subjects With Paroxysmal Nocturnal Hemoglobinuria Who Have Completed a RA101495 Clinical Study
2 other identifiers
interventional
19
8 countries
12
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 Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Jul 2017
Typical duration for phase_2
12 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
July 17, 2017
CompletedStudy Start
First participant enrolled
July 17, 2017
CompletedFirst Posted
Study publicly available on registry
July 21, 2017
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 7, 2021
CompletedStudy Completion
Last participant's last visit for all outcomes
October 26, 2021
CompletedResults Posted
Study results publicly available
October 27, 2022
CompletedSeptember 28, 2023
September 1, 2023
4.1 years
July 17, 2017
September 7, 2022
September 20, 2023
Conditions
Outcome Measures
Primary Outcomes (2)
Percentage of Participants With Treatment Emergent Adverse Events (TEAEs)
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.
From Day 1 until the Final Study Visit (up to Month 49)
Percentage of Participants With Serious TEAEs
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.
From Day 1 until the Final Study Visit (up to Month 49)
Secondary Outcomes (16)
Number of Participants With Anti-drug Antibodies (ADA)
At Day 1, Month 1, 2, 3, 6, 9, and 12
Change From Baseline in Serum Lactate Dehydrogenase (LDH) Levels at Each Time Point
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)
Change From Baseline in Total Bilirubin Values at Each Time Point
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)
Change From Baseline in Total Hemoglobin Values at Each Time Point
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)
Change From Baseline in Free Hemoglobin Values at Each Time Point
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)
- +11 more secondary outcomes
Study Arms (1)
Zilucoplan (RA101495)
EXPERIMENTALSubjects will continue to receive the final maintenance dose they were receiving in the qualifying study
Interventions
Subjects will continue to receive the final maintenance dose they were receiving in the qualifying study.
Eligibility Criteria
You may qualify if:
- Completion of a qualifying Ra Pharmaceuticals sponsored zilucoplan (RA101495) PNH study
- Evidence of ongoing clinical benefit in the opinion of the Investigator
You may not qualify if:
- History of meningococcal disease
- Current systemic infection or suspicion of active bacterial infection
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (12)
Investigative Site 4
Los Angeles, California, 90033, United States
Investigative Site 19
Dallas, Texas, 75390, United States
Investigative Site 3
Gosford, Australia
Investigative Site 5
Melbourne, Australia
Investigative Site 10
Toronto, Canada
Investigative Site 14
Helsinki, Finland
Investigative Site 9
Ulm, Germany
Investigative Site 17
Budapest, Hungary
Investigative Site 13
Christchurch, New Zealand
Investigative Site 12
Hamilton, New Zealand
Investigative Site 6
Leeds, United Kingdom
Investigative Site 7
London, United Kingdom
Related Publications (1)
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.
PMID: 37534517RESULT
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- UCB
- Organization
- Cares
Study Officials
- STUDY CHAIR
Dr. Anita Hill
St James' Institute of Oncology
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- GT60
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
July 17, 2017
First Posted
July 21, 2017
Study Start
July 17, 2017
Primary Completion
September 7, 2021
Study Completion
October 26, 2021
Last Updated
September 28, 2023
Results First Posted
October 27, 2022
Record last verified: 2023-09
Data Sharing
- IPD Sharing
- Will share
- Shared Documents
- STUDY PROTOCOL, SAP, CSR
- Time Frame
- 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.
- Access Criteria
- 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.
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.