Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients Who Have an Inadequate Response to Eculizumab
Primary Purpose
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Zilucoplan (RA101495)
Sponsored by
About this trial
This is an interventional treatment trial for Paroxysmal Nocturnal Hemoglobinuria (PNH) focused on measuring PNH
Eligibility Criteria
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
Sites / Locations
- Investigative Site
- Investigative Site
- Investigative Site
- Investigative Site
- Investigative Site
- Investigative Site
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Zilucoplan (RA101495)
Arm Description
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
Outcomes
Primary Outcome Measures
Change-from-baseline in Serum Lactate Dehydrogenase (LDH) Levels.
Change-from-baseline through Week 12 in serum lactate dehydrogenase (LDH) levels
Secondary Outcome Measures
Change-from-baseline Bilirubin Values
Changes from baseline at each of the scheduled postbaseline time-points
Change-from-baseline Total Hemoglobin Values
Changes from baseline at each of the scheduled postbaseline time-points
Change-from-baseline Free Hemoglobin Values
Changes from baseline at each of the scheduled postbaseline time-points
Change-from-baseline Haptoglobin Values
Changes from baseline at each of the scheduled postbaseline time-points
Change-from-baseline Reticulocyte Values
Changes from baseline at each of the scheduled postbaseline time-points
Change-from-baseline Hemoglobinuria Values
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.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03030183
Brief Title
Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients Who Have an Inadequate Response to Eculizumab
Official Title
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
Study Type
Interventional
2. Study Status
Record Verification Date
July 2022
Overall Recruitment Status
Completed
Study Start Date
April 17, 2017 (Actual)
Primary Completion Date
March 28, 2018 (Actual)
Study Completion Date
March 28, 2018 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Ra Pharmaceuticals
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Keywords
PNH
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
3 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Zilucoplan (RA101495)
Arm Type
Experimental
Arm Description
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 Type
Drug
Intervention Name(s)
Zilucoplan (RA101495)
Intervention Description
0.3 mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC
Primary Outcome Measure Information:
Title
Change-from-baseline in Serum Lactate Dehydrogenase (LDH) Levels.
Description
Change-from-baseline through Week 12 in serum lactate dehydrogenase (LDH) levels
Time Frame
Through Week 12 of the study
Secondary Outcome Measure Information:
Title
Change-from-baseline Bilirubin Values
Description
Changes from baseline at each of the scheduled postbaseline time-points
Time Frame
Through week 12
Title
Change-from-baseline Total Hemoglobin Values
Description
Changes from baseline at each of the scheduled postbaseline time-points
Time Frame
Through week 12
Title
Change-from-baseline Free Hemoglobin Values
Description
Changes from baseline at each of the scheduled postbaseline time-points
Time Frame
Through week 12
Title
Change-from-baseline Haptoglobin Values
Description
Changes from baseline at each of the scheduled postbaseline time-points
Time Frame
Through week 12
Title
Change-from-baseline Reticulocyte Values
Description
Changes from baseline at each of the scheduled postbaseline time-points
Time Frame
Through week 12
Title
Change-from-baseline Hemoglobinuria Values
Description
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.
Time Frame
Through week 12
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Dr. Anita Hill
Organizational Affiliation
St James' Institute of Oncology
Official's Role
Study Chair
Facility Information:
Facility Name
Investigative Site
City
Duarte
State/Province
California
ZIP/Postal Code
91010
Country
United States
Facility Name
Investigative Site
City
Los Angeles
State/Province
California
ZIP/Postal Code
90033
Country
United States
Facility Name
Investigative Site
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Facility Name
Investigative Site
City
Manhasset
State/Province
New York
ZIP/Postal Code
11030
Country
United States
Facility Name
Investigative Site
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
Investigative Site
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390
Country
United States
12. IPD Sharing Statement
Links:
URL
https://learningcenter.ehaweb.org/eha/2018/stockholm/214782/anita.hill.ra101495.a.subcutaneously-administered.peptide.inhibitor.of.html?f=menu=14*media=3*speaker=664274
Description
Related Info
Learn more about this trial
Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients Who Have an Inadequate Response to Eculizumab
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