Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients Who Have an Inadequate Response to Eculizumab

Paroxysmal Nocturnal Hemoglobinuria (PNH) Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03030183
• Other study ID #: RA101495-01.203
• Status: Completed
• Phase: Phase 2
• Start date: April 17, 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 RA101495 in patients with paroxysmal nocturnal hemoglobinuria (PNH) who have an inadequate response to eculizumab. Patients will be treated with RA101495 for 12 weeks.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 3
• 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

• 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

Related Conditions & MeSH terms

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

Intervention

Drug:
• Zilucoplan (RA101495)
0.3 mg/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
United States	Investigative Site	Chicago	Illinois
United States	Investigative Site	Dallas	Texas
United States	Investigative Site	Duarte	California
United States	Investigative Site	Durham	North Carolina
United States	Investigative Site	Los Angeles	California
United States	Investigative Site	Manhasset	New York

Sponsors (1)

Lead Sponsor	Collaborator
Ra Pharmaceuticals

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change-from-baseline in Serum Lactate Dehydrogenase (LDH) Levels.	Change-from-baseline through Week 12 in serum lactate dehydrogenase (LDH) levels	Through Week 12 of the study
Secondary	Change-from-baseline Bilirubin Values	Changes from baseline at each of the scheduled postbaseline time-points	Through week 12
Secondary	Change-from-baseline Total Hemoglobin Values	Changes from baseline at each of the scheduled postbaseline time-points	Through week 12
Secondary	Change-from-baseline Free Hemoglobin Values	Changes from baseline at each of the scheduled postbaseline time-points	Through week 12
Secondary	Change-from-baseline Haptoglobin Values	Changes from baseline at each of the scheduled postbaseline time-points	Through week 12
Secondary	Change-from-baseline Reticulocyte Values	Changes from baseline at each of the scheduled postbaseline time-points	Through week 12
Secondary	Change-from-baseline Hemoglobinuria Values	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.	Through week 12

External Links

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