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Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients

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: NCT03078582
Recruitment Status : Completed
First Posted : March 13, 2017
Results First Posted : March 10, 2020
Last Update Posted : March 10, 2020
Sponsor:
Information provided by (Responsible Party):
Ra Pharmaceuticals

Study Description
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Brief Summary:
The purpose of the study is to evaluate the safety and efficacy of zilucoplan (RA101495) in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). There will be two groups of patients in the study: the first group will include patients who have never received eculizumab for treatment of PNH. The second group will include patients who have received treatment with eculizumab for at least 6 months prior to the study. Patients will be treated with RA101495 for 12 weeks.

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

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 26 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase 2 Multicenter, Open-Label, Uncontrolled Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of RA101495 in Subjects With Paroxysmal Nocturnal Hemoglobinuria
Actual Study Start Date : March 8, 2017
Actual Primary Completion Date : March 28, 2018
Actual Study Completion Date : March 28, 2018

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Zilucoplan (RA101495) treatment naive
0.3mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC (treatment naïve)
 Drug: Zilucoplan (RA101495)
0.3mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC
Experimental: Zilucoplan (RA101495) previously on eculizumab
0.3mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC (previously on eculizumab)
 Drug: Zilucoplan (RA101495)
0.3mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC


Outcome Measures
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Primary Outcome Measures :
  1. Change-from-baseline in Serum Lactate Dehydrogenase (LDH) Level. [ Time Frame: Through Week 12 of the study ]
    The primary efficacy endpoint is the change-from-baseline in serum LDH levels during this period, defined as the mean LDH values of Weeks 6, 8, 10, and 12 minus the baseline value of LDH.


Secondary Outcome Measures :
  1. Changes From Baseline in Bilirubin Values [ Time Frame: Through Week 12 of the study ]
    Changes from baseline at each of the scheduled post-baseline time-points

  2. Total Hemoglobin [ Time Frame: Through Week 12 of the Study ]
    Changes from baseline at each of the scheduled post-baseline time-points

  3. Changes From Baseline in Free Hemoglobin Values [ Time Frame: Through Week 12 of the study ]
    Changes from baseline at each of the scheduled post-baseline time-points

  4. Haptoglobin Values [ Time Frame: Through Week 12 of the Study ]
    Changes from baseline at each of the scheduled post-baseline time-points

  5. Reticulocyte Values [ Time Frame: Through Week 12 of the Study ]
    Changes from baseline at each of the scheduled post-baseline time-points

  6. Hemoglobinuria Values [ Time Frame: Through Week 12 of the Study ]
    Changes from baseline at each of the scheduled post-baseline 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.


Eligibility Criteria
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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:

  • Diagnosis of PNH by flow cytometry
  • For treatment naive patients: subjects must not have received treatment with eculizumab prior to or during the Screening Period and must have a lactate dehydrogenase (LDH) level ≥2 times the upper limit of normal (xULN) during Screening
  • For patients who previously received eculizumab: subjects must have received treatment with eculizumab for at least 6 months prior to Screening

Exclusion Criteria:

  • History of meningococcal disease
  • Current systemic infection or suspicion of active bacterial infection
Contacts and Locations
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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): NCT03078582


Locations
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Australia, New South Wales
Investigative Site
Gosford, New South Wales, Australia
Australia
Investigative Site
Parkville, Australia
Canada, Ontario
Investigative Site
Toronto, Ontario, Canada
Denmark
Investigative Site
Copenhagen, Denmark
Finland
Investigative Site
Helsinki, Finland
Germany
Investigative Site
Essen, Germany
Investigative Site
Ulm, Germany
Hungary
Investigative Site
Budapest, Hungary
New Zealand
Investigative Site
Christchurch, New Zealand
Investigative Site
Hamilton, New Zealand
United Kingdom
Investigative Site
Leeds, United Kingdom
Investigative Site
London, United Kingdom
Sponsors and Collaborators
Ra Pharmaceuticals
Investigators
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Principal Investigator: Dr. Anita Hill St James' Institute of Oncology
  Study Documents (Full-Text)

Documents provided by Ra Pharmaceuticals:
Study Protocol  [PDF] October 20, 2016
Statistical Analysis Plan  [PDF] March 22, 2018

More Information
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Additional Information:

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Responsible Party: Ra Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03078582    
Other Study ID Numbers: RA101495-01.201
2016-003522-16 ( EudraCT Number )
First Posted: March 13, 2017    Key Record Dates
Results First Posted: March 10, 2020
Last Update Posted: March 10, 2020
Last Verified: September 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Ra Pharmaceuticals:
PNH
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