Clinical trial NCT03225287: Extension Study of RA101495 for Patients With PNH Who Have Completed a Zilucoplan (RA101495) Clinical Study
Official title: A Multicenter, Open-label, Uncontrolled, Extension Study of RA101495 in Subjects With Paroxysmal Nocturnal Hemoglobinuria Who Have Completed a RA101495 Clinical Study
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Paroxysmal Nocturnal Hemoglobinuria (PNH)
Lead sponsor: UCB Pharma
Sponsor class: INDUSTRY
First posted: 2017-07-21
Enrollment: 19
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to enable continued access to zilucoplan (RA101495) for patients with paroxysmal nocturnal hemoglobinuria (PNH) after they complete a zilucoplan clinical study.


Eligibility criteria: Inclusion Criteria:

* Completion of a qualifying Ra Pharmaceuticals sponsored zilucoplan (RA101495) PNH study
* Evidence of ongoing clinical benefit in the opinion of the Investigator

Exclusion criteria:

* History of meningococcal disease
* Current systemic infection or suspicion of active bacterial infection

Primary outcomes:
measure: Percentage of Participants With Treatment Emergent Adverse Events (TEAEs)
timeFrame: From Day 1 until the Final Study Visit (up to Month 49)
measure: Percentage of Participants With Serious TEAEs
timeFrame: From Day 1 until the Final Study Visit (up to Month 49)
Secondary outcomes:
measure: Number of Participants With Anti-drug Antibodies (ADA)
timeFrame: At Day 1, Month 1, 2, 3, 6, 9, and 12
measure: Change From Baseline in Serum Lactate Dehydrogenase (LDH) Levels at Each Time Point
timeFrame: Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49)
measure: Change From Baseline in Total Bilirubin Values at Each Time Point
timeFrame: Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49)
measure: Change From Baseline in Total Hemoglobin Values at Each Time Point
timeFrame: Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49)
measure: Change From Baseline in Free Hemoglobin Values at Each Time Point
timeFrame: Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49)
measure: Change From Baseline in Haptoglobin Values at Each Time Point
timeFrame: Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49)
measure: Change From Baseline in Reticulocytes at Each Time Point
timeFrame: Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49)
measure: Change From Baseline in Hemoglobinuria Values at Each Time Point
timeFrame: Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, 48 and Final Study Visit (Month 49)
measure: Plasma Concentrations of RA101495 and Its Major Metabolite(s)
timeFrame: Predose: At Day 1 (Screening), Month 1, 2, 3, 6, 9, 12, and Final Study Visit (Month 49)
measure: Maximum Plasma Concentration (Cmax) of RA101495
timeFrame: At Day 1, Month 1, 2, 3, 6, 9, and 12
measure: Time Corresponding to Cmax (Tmax) of RA101495
timeFrame: At Day 1, Month 1, 2, 3, 6, 9, and 12
measure: Area Under the Drug Concentration-time Curve (AUC0-t) of RA101495
timeFrame: At Day 1, Month 1, 2, 3, 6, 9, and 12
measure: Total Complement (CH50) Levels
timeFrame: At Day 1, Month 1, 2, 3, 6, 9, and 12
measure: Change From Baseline in Sheep Red Blood Cell (sRBC) Values at Each Time Point
timeFrame: Baseline, Month 1, 2, 3, 6, 9, 12 and Final Study Visit (Month 49)
measure: Change From Baseline in Wieslab Enzyme-linked Immunosorbent Assay (ELISA) Values for Alternative Complement Pathway at Each Time Point
timeFrame: Baseline, Month 1, 2, 3, 6, 9, 12 and Final Study Visit (Month 49)
measure: Change From Baseline in Complement Component 5 (C5) Values at Each Time Point
timeFrame: Baseline, Month 1, 2, 3, 6, 9, 12 and Final Study Visit (Month 49)
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Investigative Site 4
state: California
city: Dallas
country: United States
facility: Investigative Site 19
state: Texas
city: Gosford
country: Australia
facility: Investigative Site 3
city: Melbourne
country: Australia
facility: Investigative Site 5
city: Toronto
country: Canada
facility: Investigative Site 10
city: Helsinki
country: Finland
facility: Investigative Site 14
city: Ulm
country: Germany
facility: Investigative Site 9
city: Budapest
country: Hungary
facility: Investigative Site 17
city: Christchurch
country: New Zealand
facility: Investigative Site 13
city: Hamilton
country: New Zealand
facility: Investigative Site 12
city: Leeds
country: United Kingdom
facility: Investigative Site 6
city: London
country: United Kingdom
facility: Investigative Site 7
Study officials:
affiliation: St James' Institute of Oncology
name: Dr. Anita Hill
role: STUDY_CHAIR
References:
citation: Kulasekararaj AG, Lehtinen AE, Forsyth C, Gandhi S, Griffin M, Korper S, Mikala G, Muus P, Overgaard U, Patriquin CJ, Pullon H, Shen YM, Spearing R, Szer J, De la Borderie G, Duda PW, Farzaneh-Far R, Ragunathan S, Sayegh CE, Vadysirisack DD, Schrezenmeier H. Phase II trials of zilucoplan in paroxysmal nocturnal hemoglobinuria. Haematologica. 2024 Mar 1;109(3):929-935. doi: 10.3324/haematol.2022.281780. No abstract available.