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

Phase 2

Completed

• Conditions: Paroxysmal Nocturnal Hemoglobinuria (PNH)
• Interventions: Drug: Zilucoplan (RA101495)

• Registration Number: NCT03030183
• Lead Sponsor: Ra Pharmaceuticals

Brief 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2017-01-20
• Study First Submitted QC: 2017-01-20
• Study First Posted: 2017-01-24
• Study Start: 2017-04-17
• Primary Completion: 2018-03-28
• Study Completion: 2018-03-28
• Results First Submitted: 2019-08-13
• Results First Submitted QC: 2020-02-25
• Results First Posted: 2020-02-26
• Status Verified: 2022-07
• Last Update Submitted: 2022-07-26
• Last Update Posted: 2022-07-27

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 3

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Zilucoplan (RA101495)	Zilucoplan (RA101495)	Subjects will receive RA101495 at the dose of 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 Outcome Measures

Name	Time	Method
Change-from-baseline in Serum Lactate Dehydrogenase (LDH) Levels.	Through Week 12 of the study	Change-from-baseline through Week 12 in serum lactate dehydrogenase (LDH) levels

Secondary Outcome Measures

Name	Time	Method
Change-from-baseline Bilirubin Values	Through week 12	Changes from baseline at each of the scheduled postbaseline time-points
Change-from-baseline Hemoglobinuria Values	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.
Change-from-baseline Free Hemoglobin Values	Through week 12	Changes from baseline at each of the scheduled postbaseline time-points
Change-from-baseline Haptoglobin Values	Through week 12	Changes from baseline at each of the scheduled postbaseline time-points
Change-from-baseline Reticulocyte Values	Through week 12	Changes from baseline at each of the scheduled postbaseline time-points
Change-from-baseline Total Hemoglobin Values	Through week 12	Changes from baseline at each of the scheduled postbaseline time-points

Trial Locations

Locations (1)

Investigative Site; 🇺🇸 Dallas, Texas, United States