Your session is about to expire
← Back to Search
Peptide Inhibitor
Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients
Phase 2
Waitlist Available
Led By Dr. Anita Hill
Research Sponsored by Ra Pharmaceuticals
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up through week 12 of the study
Awards & highlights
No Placebo-Only Group
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.
Eligible Conditions
- Paroxysmal Nocturnal Hemoglobinuria
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ through week 12 of the study
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~through week 12 of the study
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
Change-from-baseline in Serum Lactate Dehydrogenase (LDH) Level.
Secondary study objectives
Changes From Baseline in Bilirubin Values
Changes From Baseline in Free Hemoglobin Values
Haptoglobin Values
+3 moreSide effects data
From 2018 Phase 2 trial • 3 Patients • NCT0303018333%
Haemoglobinuria
33%
Nephrolithiasis
33%
Nausea
33%
Influenza like illness
33%
Lipase increased
33%
Musculoskeletal chest pain
33%
Pain in extremity
33%
Headache
100%
80%
60%
40%
20%
0%
Study treatment Arm
Zilucoplan (RA101495)
Awards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Trial Design
2Treatment groups
Experimental Treatment
Group I: Zilucoplan (RA101495) treatment naiveExperimental Treatment1 Intervention
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)
Group II: Zilucoplan (RA101495) previously on eculizumabExperimental Treatment1 Intervention
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)
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Zilucoplan
Not yet FDA approved
Find a Location
Who is running the clinical trial?
Ra PharmaceuticalsLead Sponsor
7 Previous Clinical Trials
630 Total Patients Enrolled
Dr. Anita HillPrincipal InvestigatorSt James' Institute of Oncology
2 Previous Clinical Trials
22 Total Patients Enrolled