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Completed

NCT Number: NCT03078582

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

The purpose of the study is to evaluate the safety and efficacy of zilucoplan (RA101495) in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). There will be two groups of patients in the study: the first group will include patients who have never received eculizumab for treatment of PNH. The second group will include patients who have received treatment with eculizumab for at least 6 months prior to the study. 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, Gosford, New South Wales, Australia

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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
  • For treatment naive patients: subjects must not have received treatment with eculizumab prior to or during the Screening Period and must have a lactate dehydrogenase (LDH) level ≥2 times the upper limit of normal (xULN) during Screening
  • For patients who previously received eculizumab: subjects must have received treatment with eculizumab for at least 6 months prior to Screening

Exclusion criteria

  • History of meningococcal disease
  • Current systemic infection or suspicion of active bacterial infection

Treatment and study plan

zilucoplan (RA101495)

Drug

0.3mg/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) Level.

    Time frame: Through Week 12 of the study

    The primary efficacy endpoint is the change-from-baseline in serum LDH levels during this period, defined as the mean LDH values of Weeks 6, 8, 10, and 12 minus the baseline value of LDH.

Secondary outcomes

  1. Changes From Baseline in Bilirubin Values

    Time frame: Through Week 12 of the study

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

  2. Total Hemoglobin

    Time frame: Through Week 12 of the Study

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

  3. Changes From Baseline in Free Hemoglobin Values

    Time frame: Through Week 12 of the study

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

  4. Haptoglobin Values

    Time frame: Through Week 12 of the Study

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

  5. Reticulocyte Values

    Time frame: Through Week 12 of the Study

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

  6. Hemoglobinuria Values

    Time frame: Through Week 12 of the Study

    Changes from baseline at each of the scheduled post-baseline 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

Phase 2 Multicenter, Open-Label, Uncontrolled Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of RA101495 in Subjects With Paroxysmal Nocturnal Hemoglobinuria

Important dates

Study start
2017
Primary completion
2018
Study completion
2018
First posted
Mar 13, 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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