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OpenTrials
Completed

NCT Number: NCT03030183

Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients 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.

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Key information

Conditions

Paroxysmal Nocturnal Hemoglobinuria (PNH) Anemia Anemia, Hemolytic Bone Marrow Diseases Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinuria, Paroxysmal Myelodysplastic Syndromes

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Investigative Site, Duarte, California, United States

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

zilucoplan (RA101495)

Drug

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 outcomes

  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

Secondary outcomes

  1. Change-from-baseline Bilirubin Values

    Time frame: Through week 12

    Changes from baseline at each of the scheduled postbaseline time-points

  2. Change-from-baseline Total Hemoglobin Values

    Time frame: Through week 12

    Changes from baseline at each of the scheduled postbaseline time-points

  3. Change-from-baseline Free Hemoglobin Values

    Time frame: Through week 12

    Changes from baseline at each of the scheduled postbaseline time-points

  4. Change-from-baseline Haptoglobin Values

    Time frame: Through week 12

    Changes from baseline at each of the scheduled postbaseline time-points

  5. Change-from-baseline Reticulocyte Values

    Time frame: Through week 12

    Changes from baseline at each of the scheduled postbaseline time-points

  6. 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.

Sponsors and collaborators

Lead sponsor

Ra Pharmaceuticals

Industry

Registry information

Official study 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

Important dates

Study start
2017
Primary completion
2018
Study completion
2018
First posted
Jan 24, 2017
Registry last updated
Jul 27, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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