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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. Identifier: NCT03030183
Recruitment Status : Completed
First Posted : January 24, 2017
Last Update Posted : June 20, 2019
Information provided by (Responsible Party):
Ra Pharmaceuticals

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.

Condition or disease Intervention/treatment Phase
Paroxysmal Nocturnal Hemoglobinuria (PNH) Drug: Zilucoplan (RA101495) Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 3 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : April 17, 2017
Actual Primary Completion Date : March 28, 2018
Actual Study Completion Date : March 28, 2018

Arm Intervention/treatment
Experimental: Zilucoplan (RA101495)
Subjects will receive RA101495 at the dose of 0.3mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC
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

Primary Outcome Measures :
  1. 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

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03030183

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United States, California
Investigative Site
Duarte, California, United States, 91010
Investigative Site
Los Angeles, California, United States, 90033
United States, Illinois
Investigative Site
Chicago, Illinois, United States, 60612
United States, New York
Investigative Site
Manhasset, New York, United States, 11030
United States, North Carolina
Investigative Site
Durham, North Carolina, United States, 27710
United States, Texas
Investigative Site
Dallas, Texas, United States, 75390
Sponsors and Collaborators
Ra Pharmaceuticals
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Study Chair: Dr. Anita Hill St James' Institute of Oncology

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Responsible Party: Ra Pharmaceuticals Identifier: NCT03030183     History of Changes
Other Study ID Numbers: RA101495-01.203
First Posted: January 24, 2017    Key Record Dates
Last Update Posted: June 20, 2019
Last Verified: June 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Ra Pharmaceuticals:
Additional relevant MeSH terms:
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Hemoglobinuria, Paroxysmal
Urination Disorders
Urologic Diseases
Urological Manifestations
Signs and Symptoms
Anemia, Hemolytic
Hematologic Diseases
Myelodysplastic Syndromes
Bone Marrow Diseases