- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03030183
Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients Who Have an Inadequate Response to Eculizumab
July 26, 2022 updated by: Ra Pharmaceuticals
A Phase 2 Multicenter, Open-Label, Uncontrolled Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of RA101495 in Subjects With Paroxysmal Nocturnal Hemoglobinuria Who Have an Inadequate Response to Eculizumab
The purpose of the study is to evaluate the safety and efficacy of RA101495 in patients with paroxysmal nocturnal hemoglobinuria (PNH) who have an inadequate response to eculizumab.
Patients will be treated with RA101495 for 12 weeks.
Study Overview
Status
Completed
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Actual)
3
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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California
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Duarte, California, United States, 91010
- Investigative Site
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Los Angeles, California, United States, 90033
- Investigative Site
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Illinois
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Chicago, Illinois, United States, 60612
- Investigative Site
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New York
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Manhasset, New York, United States, 11030
- Investigative Site
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North Carolina
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Durham, North Carolina, United States, 27710
- Investigative Site
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Texas
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Dallas, Texas, United States, 75390
- Investigative Site
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years and older (ADULT, OLDER_ADULT)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Inclusion Criteria:
- Diagnosis of PNH by flow cytometry
- Inadequate response to eculizumab defined as having received eculizumab for at least 6 months plus a documented LDH level ≥ 1.5 x the upper limit of normal (ULN) and/or the presence of a known C5 mutation conferring resistance to eculizumab
Exclusion Criteria:
- History of meningococcal disease
- Current systemic infection or suspicion of active bacterial infection
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
- Allocation: NA
- Interventional Model: SINGLE_GROUP
- Masking: NONE
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
EXPERIMENTAL: Zilucoplan (RA101495)
Subjects will receive RA101495 at the dose of 0.3 mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC
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0.3 mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change-from-baseline in Serum Lactate Dehydrogenase (LDH) Levels.
Time Frame: Through Week 12 of the study
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Change-from-baseline through Week 12 in serum lactate dehydrogenase (LDH) levels
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Through Week 12 of the study
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change-from-baseline Bilirubin Values
Time Frame: Through week 12
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Changes from baseline at each of the scheduled postbaseline time-points
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Through week 12
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Change-from-baseline Total Hemoglobin Values
Time Frame: Through week 12
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Changes from baseline at each of the scheduled postbaseline time-points
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Through week 12
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Change-from-baseline Free Hemoglobin Values
Time Frame: Through week 12
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Changes from baseline at each of the scheduled postbaseline time-points
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Through week 12
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Change-from-baseline Haptoglobin Values
Time Frame: Through week 12
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Changes from baseline at each of the scheduled postbaseline time-points
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Through week 12
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Change-from-baseline Reticulocyte Values
Time Frame: Through week 12
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Changes from baseline at each of the scheduled postbaseline time-points
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Through week 12
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Change-from-baseline Hemoglobinuria Values
Time Frame: Through week 12
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Changes from baseline at each of the scheduled postbaseline time-points Hemoglobinuria was assessed using a urine colorimetric scoring system with a score of 1 through 10.
Where 1 represents no hemoglobinuria and 10 represents maximum hemoglobinuria.
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Through week 12
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Helpful Links
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (ACTUAL)
April 17, 2017
Primary Completion (ACTUAL)
March 28, 2018
Study Completion (ACTUAL)
March 28, 2018
Study Registration Dates
First Submitted
January 20, 2017
First Submitted That Met QC Criteria
January 20, 2017
First Posted (ESTIMATE)
January 24, 2017
Study Record Updates
Last Update Posted (ACTUAL)
July 27, 2022
Last Update Submitted That Met QC Criteria
July 26, 2022
Last Verified
July 1, 2022
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- RA101495-01.203
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Paroxysmal Nocturnal Hemoglobinuria (PNH)
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Novartis PharmaceuticalsCompletedParoxysmal Nocturnal Hemoglobinuria PNHLithuania, Japan, Czechia
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Alexion PharmaceuticalsAchillion, a wholly owned subsidiary of AlexionCompletedParoxysmal Nocturnal Hemoglobinuria (PNH)United Kingdom, New Zealand, Korea, Republic of, Italy
-
AKARI TherapeuticsCompletedParoxysmal Nocturnal Hemoglobinuria (PNH)Kazakhstan, Lithuania, Sri Lanka
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AlexionActive, not recruitingParoxysmal Nocturnal Hemoglobinuria (PNH)United Kingdom, Italy, Canada, Korea, Republic of, New Zealand, Spain, Turkey
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AlexionCompletedParoxysmal Nocturnal Hemoglobinuria (PNH)Belgium, France, Italy, Japan, Spain, Taiwan, United Kingdom, United States, Canada, Czechia, Germany, Sweden, Singapore, Korea, Republic of, Russian Federation, Austria, Poland, Argentina, Australia, Brazil, Estonia, Malaysia, Mexico, Thaila... and more
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Apellis Pharmaceuticals, Inc.CompletedParoxysmal Nocturnal Hemoglobinuria (PNH)United States
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AlexionCompletedParoxysmal Nocturnal Hemoglobinuria (PNH)United States, Korea, Republic of, Canada, France, Germany, Spain, United Kingdom, Japan, Australia, Italy, Netherlands
-
Ra PharmaceuticalsCompletedParoxysmal Nocturnal Hemoglobinuria (PNH)United Kingdom, Australia, Canada, Denmark, Finland, Germany, Hungary, New Zealand
Clinical Trials on Zilucoplan (RA101495)
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Ra Pharmaceuticals, Inc.TerminatedParoxysmal Nocturnal Hemoglobinuria (PNH)United States, Australia, Canada, Finland, Germany, Hungary, New Zealand, United Kingdom
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Ra Pharmaceuticals, Inc.Active, not recruitingGeneralized Myasthenia GravisUnited States, Canada, France, Germany, Italy, Japan, Norway, Poland, Spain, United Kingdom
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Ra PharmaceuticalsCompletedParoxysmal Nocturnal Hemoglobinuria (PNH)United Kingdom, Australia, Canada, Denmark, Finland, Germany, Hungary, New Zealand
-
Ra Pharmaceuticals, Inc.CompletedMyasthenia Gravis, GeneralizedUnited States, Canada, France, Germany, Italy, Japan, Norway, Poland, Spain, United Kingdom
-
Ra PharmaceuticalsCompletedGeneralized Myasthenia GravisUnited States, Canada
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UCB Biopharma SRLActive, not recruiting
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UCB Biopharma SRLRecruitingGeneralized Myasthenia GravisItaly
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Merit E. Cudkowicz, MDRa PharmaceuticalsCompleted
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Ra PharmaceuticalsTerminatedImmune Mediated Necrotizing MyopathyUnited States, Netherlands, France, United Kingdom
-
University Hospital, GhentUCB PharmaCompleted