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

July 26, 2022 updated by: Ra Pharmaceuticals

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

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.

Study Overview

Status

Completed

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

3

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

    • California
      • Duarte, California, United States, 91010
        • Investigative Site
      • Los Angeles, California, United States, 90033
        • Investigative Site
    • Illinois
      • Chicago, Illinois, United States, 60612
        • Investigative Site
    • New York
      • Manhasset, New York, United States, 11030
        • Investigative Site
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Investigative Site
    • Texas
      • Dallas, Texas, United States, 75390
        • Investigative Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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
0.3 mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
Through Week 12 of the study

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change-from-baseline Bilirubin Values
Time Frame: Through week 12
Changes from baseline at each of the scheduled postbaseline time-points
Through week 12
Change-from-baseline Total Hemoglobin Values
Time Frame: Through week 12
Changes from baseline at each of the scheduled postbaseline time-points
Through week 12
Change-from-baseline Free Hemoglobin Values
Time Frame: Through week 12
Changes from baseline at each of the scheduled postbaseline time-points
Through week 12
Change-from-baseline Haptoglobin Values
Time Frame: Through week 12
Changes from baseline at each of the scheduled postbaseline time-points
Through week 12
Change-from-baseline Reticulocyte Values
Time Frame: Through week 12
Changes from baseline at each of the scheduled postbaseline time-points
Through week 12
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.
Through week 12

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

April 17, 2017

Primary Completion (ACTUAL)

March 28, 2018

Study Completion (ACTUAL)

March 28, 2018

Study Registration Dates

First Submitted

January 20, 2017

First Submitted That Met QC Criteria

January 20, 2017

First Posted (ESTIMATE)

January 24, 2017

Study Record Updates

Last Update Posted (ACTUAL)

July 27, 2022

Last Update Submitted That Met QC Criteria

July 26, 2022

Last Verified

July 1, 2022

More Information

Terms related to this study

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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