This study evaluated the safety, tolerability, efficacy, pharmacokinetics, pharmacodynamics, and immunogenicity of multiple intravenous (IV) doses of ALXN1210 administered to participants with PNH who have not previously been treated with complement inhibitor.
The data presented is up to the Primary Completion date of the study and is for the 24-week Primary Evaluation period. The study also includes an Extension Period of up to 5 years.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
13
Participants were administered ravulizumab as an IV infusion every 4 weeks.
Clinical Trial Site
Liverpool, New South Wales, Australia
Clinical Trial Site
Woolloongabba, Queensland, Australia
Clinical Trial Site
Seoul, South Korea
Clinical Trial Site
Seoul, South Korea
Percent Change In Lactate Dehydrogenase (LDH) Levels From Baseline To Day 169
Baseline was defined as the average of all available assessments prior to first ALXN1210 infusion.
Time frame: Baseline, Day 169
Percent Change In Free Hemoglobin Levels From Baseline To Day 169 And Day 1821
Baseline was defined as the last non-missing assessment value prior to the first ALXN1210 infusion.
Time frame: Baseline, Day 169, Day 1821
Percent Change In Haptoglobin Levels From Baseline To Day 169 And Day 1821
Baseline was defined as the last non-missing assessment value prior to the first ALXN1210 infusion.
Time frame: Baseline, Day 169, Day 1821
Percent Change In Reticulocyte/Erythrocyte Count From Baseline To Day 169 And Day 1821
Baseline was defined as the last non-missing assessment value prior to the first ALXN1210 infusion.
Time frame: Baseline, Day 169, Day 1821
Percent Change In Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Clones From Baseline To Day 169 And Day 1933
Baseline was defined as the last non-missing assessment value prior to the first ALXN1210 infusion.
Time frame: Baseline, Day 169, Day 1933
Percent Change In D-dimer Levels From Baseline To Day 169 And Day 1821
Baseline was defined as the last non-missing assessment value prior to the first ALXN1210 infusion.
Time frame: Baseline, Day 169, Day 1821
Change In Clinical Manifestations Of PNH From Baseline To Day 169 And Day 1821
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Clinical Trial Site
Seoul, South Korea
Clinical Trial Site
Seoul, South Korea
Clinical Trial Site
Seoul, South Korea
Clinical Trial Site
Ulsan, South Korea
Clinical manifestations are defined as fatigue, abdominal pain, dyspnea, dysphagia, chest pain, and erectile dysfunction (ED) by cohort. Improvement is defined as present at baseline and absent at Day 169 endpoint. Worsening is defined as absent at Baseline and present at Day 169 endpoint.
Time frame: Baseline, Day 169, Day 1821
Area Under The Serum Concentration-versus-time-curve From Time 0 (Dosing) To The Last Quantifiable Concentration (AUCt) At Day 1
AUCt reported in hours\*microgram/milliliter (h\*ug/mL).
Time frame: Day 1
AUCt/ Dose-normalized (D) At Day 1
Time frame: Day 1
Area Under The Serum Concentration-versus-time-curve From Time 0 (Dosing) To The End Of The Dosing Interval (AUCtau) At Day 141
Time frame: Day 141
AUCtau/D At Day 141
Time frame: Day 141
Maximum Observed Serum Concentration (Cmax) At Day 1 And Day 141
Time frame: Day 1 and Day 141
Cmax/D At Day 1 And Day 141
Time frame: Day 1 and Day 141
Concentration At The End Of The Dosage Interval (Ctrough) At Day 1 And At Day 141
Time frame: Day 1 and Day 141
Time To Maximum Observed Serum Concentration (Tmax) At Day 1 And Day 141
Time frame: Day 1 and Day 141
Percent Change In Chicken Red Blood Cell (cRBC) Hemolysis From Baseline To Day 1709
Time frame: Baseline, Day 1709
Percent Change In Free Complement Component 5 (C5) Concentration From Baseline To Day 1709
Time frame: Baseline, Day 1709
Percent Change In Total C5 Concentration From Baseline To Day 1709
Time frame: Baseline, Day 1709
Participants Experiencing Antidrug Antibodies (ADAs)
Time frame: Day 1821