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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

Tracking Information
First Submitted Date  ICMJE March 8, 2017
First Posted Date  ICMJE March 13, 2017
Results First Submitted Date  ICMJE June 19, 2019
Results First Posted Date  ICMJE March 10, 2020
Last Update Posted Date March 10, 2020
Actual Study Start Date  ICMJE March 8, 2017
Actual Primary Completion Date March 28, 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 25, 2020)
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.
Original Primary Outcome Measures  ICMJE
 (submitted: March 8, 2017)
Change-from-baseline in serum lactate dehydrogenase (LDH) level. [ Time Frame: Through Week 12 of the study ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: February 25, 2020)
  • 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
  • Total Hemoglobin [ Time Frame: Through Week 12 of the Study ]
    Changes from baseline at each of the scheduled post-baseline time-points
  • 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
  • Haptoglobin Values [ Time Frame: Through Week 12 of the Study ]
    Changes from baseline at each of the scheduled post-baseline time-points
  • Reticulocyte Values [ Time Frame: Through Week 12 of the Study ]
    Changes from baseline at each of the scheduled post-baseline time-points
  • 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.
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients
Official Title  ICMJE Phase 2 Multicenter, Open-Label, Uncontrolled Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of RA101495 in Subjects With Paroxysmal Nocturnal Hemoglobinuria
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Paroxysmal Nocturnal Hemoglobinuria (PNH)
Intervention  ICMJE 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
Study Arms  ICMJE
  • 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)
    Intervention: Drug: Zilucoplan (RA101495)
  • 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)
    Intervention: Drug: Zilucoplan (RA101495)
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: September 5, 2018)
26
Original Estimated Enrollment  ICMJE
 (submitted: March 8, 2017)
20
Actual Study Completion Date  ICMJE March 28, 2018
Actual Primary Completion Date March 28, 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Australia,   Canada,   Denmark,   Finland,   Germany,   Hungary,   New Zealand,   United Kingdom
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03078582
Other Study ID Numbers  ICMJE RA101495-01.201
2016-003522-16 ( EudraCT Number )
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Ra Pharmaceuticals
Study Sponsor  ICMJE Ra Pharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Dr. Anita Hill St James' Institute of Oncology
PRS Account Ra Pharmaceuticals
Verification Date September 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP