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Extension Study of RA101495 for Patients With PNH Who Have Completed a Zilucoplan (RA101495) Clinical Study

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 evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03225287
Recruitment Status : Terminated (After careful consideration, UCB has decided to no longer pursue PNH as a potential indication for zilucoplan.)
First Posted : July 21, 2017
Last Update Posted : April 19, 2022
Sponsor:
Information provided by (Responsible Party):
Ra Pharmaceuticals

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.

Condition or disease Intervention/treatment Phase
Paroxysmal Nocturnal Hemoglobinuria (PNH) Drug: Zilucoplan (RA101495) Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 28 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multicenter, Open-label, Uncontrolled, Extension Study of RA101495 in Subjects With Paroxysmal Nocturnal Hemoglobinuria Who Have Completed a RA101495 Clinical Study
Actual Study Start Date : July 17, 2017
Actual Primary Completion Date : September 7, 2021
Actual Study Completion Date : October 26, 2021


Arm Intervention/treatment
Experimental: Zilucoplan (RA101495)
Subjects will continue to receive the final maintenance dose they were receiving in the qualifying study
Drug: Zilucoplan (RA101495)
Subjects will continue to receive the final maintenance dose they were receiving in the qualifying study.




Primary Outcome Measures :
  1. Adverse Events and Serious Adverse Events Profile [ Time Frame: Safety events will be assessed at each study visit through the completion of the study (approximately 2.5 years) ]
    Adverse Events and Serious Adverse Events Profile



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Completion of a qualifying Ra Pharmaceuticals sponsored zilucoplan (RA101495) PNH study
  • Evidence of ongoing clinical benefit in the opinion of the Investigator

Exclusion criteria:

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03225287


Locations
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United States, California
Investigative Site 4
Los Angeles, California, United States, 90033
United States, Texas
Investigative Site 19
Dallas, Texas, United States, 75390
Australia
Investigative Site 3
Gosford, Australia
Investigative Site 5
Melbourne, Australia
Canada
Investigative Site 10
Toronto, Canada
Finland
Investigative Site 14
Helsinki, Finland
Germany
Investigative Site 9
Ulm, Germany
Hungary
Investigative Site 17
Budapest, Hungary
New Zealand
Investigative Site 13
Christchurch, New Zealand
Investigative Site 12
Hamilton, New Zealand
United Kingdom
Investigative Site 6
Leeds, United Kingdom
Investigative Site 7
London, United Kingdom
Sponsors and Collaborators
Ra Pharmaceuticals
Investigators
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Study Chair: Dr. Anita Hill St James' Institute of Oncology
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Responsible Party: Ra Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03225287    
Other Study ID Numbers: RA101495-01.202
2016-003523-34 ( EudraCT Number )
First Posted: July 21, 2017    Key Record Dates
Last Update Posted: April 19, 2022
Last Verified: April 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: 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 Materials: Study Protocol
Statistical Analysis Plan (SAP)
Clinical Study Report (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.
URL: http://www.Vivli.org

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Hemoglobinuria
Hemoglobinuria, Paroxysmal
Proteinuria
Urination Disorders
Urologic Diseases
Urological Manifestations
Anemia, Hemolytic
Anemia
Hematologic Diseases
Myelodysplastic Syndromes
Bone Marrow Diseases