Clinical trial NCT03078582: Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients
Official 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Paroxysmal Nocturnal Hemoglobinuria (PNH)
Keywords: PNH
Lead sponsor: UCB Pharma
Sponsor class: INDUSTRY
First posted: 2017-03-13
Enrollment: 26
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: 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

Primary outcomes:
measure: Change-from-baseline in Serum Lactate Dehydrogenase (LDH) Level.
timeFrame: Through Week 12 of the study
Secondary outcomes:
measure: Changes From Baseline in Bilirubin Values
timeFrame: Through Week 12 of the study
measure: Total Hemoglobin
timeFrame: Through Week 12 of the Study
measure: Changes From Baseline in Free Hemoglobin Values
timeFrame: Through Week 12 of the study
measure: Haptoglobin Values
timeFrame: Through Week 12 of the Study
measure: Reticulocyte Values
timeFrame: Through Week 12 of the Study
measure: Hemoglobinuria Values
timeFrame: Through Week 12 of the Study
Other outcomes:
Locations:
city: Gosford
country: Australia
facility: Investigative Site
state: New South Wales
city: Parkville
country: Australia
facility: Investigative Site
city: Toronto
country: Canada
facility: Investigative Site
state: Ontario
city: Copenhagen
country: Denmark
facility: Investigative Site
city: Helsinki
country: Finland
facility: Investigative Site
city: Essen
country: Germany
facility: Investigative Site
city: Ulm
country: Germany
facility: Investigative Site
city: Budapest
country: Hungary
facility: Investigative Site
city: Christchurch
country: New Zealand
facility: Investigative Site
city: Hamilton
country: New Zealand
facility: Investigative Site
city: Leeds
country: United Kingdom
facility: Investigative Site
city: London
country: United Kingdom
facility: Investigative Site
Study officials:
affiliation: St James' Institute of Oncology
name: Dr. Anita Hill
role: PRINCIPAL_INVESTIGATOR
References: