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

• ClinicalTrials.gov Identifier: NCT03225287
• Recruitment Status: Terminated
• First Posted: July 21, 2017
• Last Update Posted: April 19, 2022
• Sponsor: Ra Pharmaceuticals
• Information provided by (Responsible Party): Ra Pharmaceuticals

Study Description

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

Study Design

• 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

Arms and Interventions

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.

Outcome Measures

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

Eligibility Criteria

• 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

Contacts and Locations

Locations

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

• Study Chair: Dr. Anita Hill; St James' Institute of Oncology

More Information

• 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
• 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

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Hemoglobinuria; Hemoglobinuria, Paroxysmal; Proteinuria; Urination Disorders; Urologic Diseases; Urological Manifestations; Anemia, Hemolytic; Anemia; Hematologic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases