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

Phase 2

Completed

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

• Registration Number: NCT03078582
• Lead Sponsor: Ra Pharmaceuticals

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2017-03-08
• Study First Submitted: 2017-03-08
• Study First Submitted QC: 2017-03-08
• Study First Posted: 2017-03-13
• Primary Completion: 2018-03-28
• Study Completion: 2018-03-28
• Results First Submitted: 2019-06-19
• Results First Submitted QC: 2020-02-25
• Results First Posted: 2020-03-10
• Status Verified: 2022-07
• Last Update Submitted: 2022-07-26
• Last Update Posted: 2022-07-27

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 26

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Zilucoplan (RA101495) treatment naive	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 (treatment naïve)
Zilucoplan (RA101495) previously on eculizumab	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 (previously on eculizumab)

Primary Outcome Measures

Name	Time	Method
Change-from-baseline in Serum Lactate Dehydrogenase (LDH) Level.	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 Outcome Measures

Name	Time	Method
Changes From Baseline in Bilirubin Values	Through Week 12 of the study	Changes from baseline at each of the scheduled post-baseline time-points
Total Hemoglobin	Through Week 12 of the Study	Changes from baseline at each of the scheduled post-baseline time-points
Changes From Baseline in Free Hemoglobin Values	Through Week 12 of the study	Changes from baseline at each of the scheduled post-baseline time-points
Haptoglobin Values	Through Week 12 of the Study	Changes from baseline at each of the scheduled post-baseline time-points
Reticulocyte Values	Through Week 12 of the Study	Changes from baseline at each of the scheduled post-baseline time-points
Hemoglobinuria Values	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.

Trial Locations

Locations (1)

Investigative Site; 🇬🇧 London, United Kingdom