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

Paroxysmal Nocturnal Hemoglobinuria (PNH) Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03078582
• Other study ID #: RA101495-01.201
• Secondary ID: 2016-003522-16
• Status: Completed
• Phase: Phase 2
• Start date: March 8, 2017
• Est. completion date: March 28, 2018

Study information

• Verified date: July 2022
• Source: UCB Pharma
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 26
• Est. completion date: March 28, 2018
• Est. primary completion date: March 28, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Hemoglobinuria
• Hemoglobinuria, Paroxysmal
• Paroxysmal Nocturnal Hemoglobinuria (PNH)

Intervention

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

Locations

Country	Name	City	State
Australia	Investigative Site	Gosford	New South Wales
Australia	Investigative Site	Parkville
Canada	Investigative Site	Toronto	Ontario
Denmark	Investigative Site	Copenhagen
Finland	Investigative Site	Helsinki
Germany	Investigative Site	Essen
Germany	Investigative Site	Ulm
Hungary	Investigative Site	Budapest
New Zealand	Investigative Site	Christchurch
New Zealand	Investigative Site	Hamilton
United Kingdom	Investigative Site	Leeds
United Kingdom	Investigative Site	London

Sponsors (1)

Lead Sponsor	Collaborator
Ra Pharmaceuticals

Countries where clinical trial is conducted

Australia,  Canada,  Denmark,  Finland,  Germany,  Hungary,  New Zealand,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change-from-baseline in Serum Lactate Dehydrogenase (LDH) Level.	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.	Through Week 12 of the study
Secondary	Changes From Baseline in Bilirubin Values	Changes from baseline at each of the scheduled post-baseline time-points	Through Week 12 of the study
Secondary	Total Hemoglobin	Changes from baseline at each of the scheduled post-baseline time-points	Through Week 12 of the Study
Secondary	Changes From Baseline in Free Hemoglobin Values	Changes from baseline at each of the scheduled post-baseline time-points	Through Week 12 of the study
Secondary	Haptoglobin Values	Changes from baseline at each of the scheduled post-baseline time-points	Through Week 12 of the Study
Secondary	Reticulocyte Values	Changes from baseline at each of the scheduled post-baseline time-points	Through Week 12 of the Study
Secondary	Hemoglobinuria Values	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.	Through Week 12 of the Study

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