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Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients Who Have an Inadequate Response to Eculizumab

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: NCT03030183
Recruitment Status : Completed
First Posted : January 24, 2017
Results First Posted : February 26, 2020
Last Update Posted : February 26, 2020
Sponsor:
Information provided by (Responsible Party):
Ra Pharmaceuticals

Tracking Information
First Submitted Date  ICMJE January 20, 2017
First Posted Date  ICMJE January 24, 2017
Results First Submitted Date  ICMJE August 13, 2019
Results First Posted Date  ICMJE February 26, 2020
Last Update Posted Date February 26, 2020
Actual Study Start Date  ICMJE April 17, 2017
Actual Primary Completion Date March 28, 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 19, 2019)
Change-from-baseline in Serum Lactate Dehydrogenase (LDH) Levels. [ Time Frame: Through Week 12 of the study ]
Change-from-baseline through Week 12 in serum lactate dehydrogenase (LDH) levels
Original Primary Outcome Measures  ICMJE
 (submitted: January 20, 2017)
Change-from-baseline in Serum Lactate Dehydrogenase (LDH) Levels. [ Time Frame: Through Week 12 of the study ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: February 25, 2020)
  • Change-from-baseline Bilirubin Values [ Time Frame: Through week 12 ]
    Changes from baseline at each of the scheduled postbaseline time-points
  • Change-from-baseline Total Hemoglobin Values [ Time Frame: Through week 12 ]
    Changes from baseline at each of the scheduled postbaseline time-points
  • Change-from-baseline Free Hemoglobin Values [ Time Frame: Through week 12 ]
    Changes from baseline at each of the scheduled postbaseline time-points
  • Change-from-baseline Haptoglobin Values [ Time Frame: Through week 12 ]
    Changes from baseline at each of the scheduled postbaseline time-points
  • Change-from-baseline Reticulocyte Values [ Time Frame: Through week 12 ]
    Changes from baseline at each of the scheduled postbaseline time-points
  • Change-from-baseline Hemoglobinuria Values [ Time Frame: Through week 12 ]
    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.
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 Who Have an Inadequate Response to Eculizumab
Official Title  ICMJE 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
Brief Summary 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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Paroxysmal Nocturnal Hemoglobinuria (PNH)
Intervention  ICMJE Drug: Zilucoplan (RA101495)
0.3 mg/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)
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
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)
3
Original Estimated Enrollment  ICMJE
 (submitted: January 20, 2017)
8
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
  • 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
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 United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03030183
Other Study ID Numbers  ICMJE RA101495-01.203
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Ra Pharmaceuticals
Study Sponsor  ICMJE Ra Pharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Chair: 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