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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
Ra Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Paroxysmal Nocturnal Hemoglobinuria (PNH) focused on measuring PNH

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

First Posted
January 20, 2017
Last Updated
July 26, 2022
Sponsor
Ra Pharmaceuticals
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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

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

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