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A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Naïve Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Naïve Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) - PNH-301

Status
Unknown
Phases
Phase 3
Study type
Interventional
Source
NL-OMON
Registry ID
NL-OMON45425
Enrollment
5
Registered
2017-04-26
Start date
Unknown
Completion date
Unknown
Last updated
2024-02-28

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

bleeding disorder

Interventions

Administration of study studydrug ALXN1210 and eculizumab (active control) via IV infusion. Dosage is based on patients weight.
ALXN1210
complement inhibitor treatment
lactate dehydrogenase normalization

Sponsors

Alexion Pharmaceuticals
Lead Sponsor

Eligibility

Age
18 Years to 64 Years

Inclusion criteria

Inclusion criteria: 1. Male or female * 18 years of age 2. PNH diagnosis confirmed documented by high-sensitivity flow cytometry 3. Presence of 1 or more of the following PNH-related signs or symptoms within 3 months of Screening: fatigue, hemoglobinuria, abdominal pain, shortness of breath (dyspnea), anemia (hemoglobin

Exclusion criteria

Exclusion criteria: 1. Treatment with a complement inhibitor at any time. 2. History of bone marrow transplantation. 3. Body weight

Design outcomes

Primary

MeasureTime frame
The coprimary efficacy endpoints of the study are: * Transfusion avoidance, defined as the proportion of patients who remain transfusion-free and do not require a transfusion per protocol-specified guidelines through Day 183 (Week 26) * Hemolysis as directly measured by LDH-N levels from Day 29 (first scheduled evaluation status post initiation of maintenance dosing) through Day 183 (Week 26)—

Secondary

MeasureTime frame
1. Percentage change in LDH from Baseline to Day 183 (Week 26) 2. Change in quality of life (QoL) assessed via the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale, Version 4, from Baseline to Day 183 (Week 26) 3. Proportion of patients with breakthrough hemolysis, defined as at least one new or worsening symptom or sign of intravascular hemolysis (fatigue, hemoglobinuria, abdominal pain, shortness of breath [dyspnea], anemia [hemoglobin—

Countries

Argentina, Australia, Austria, Belgium, Brazil, Canada, Colombia, Czechia, Denmark, Finland, France, Germany, Ireland, Italy, Japan, Korea (the Republic of), Malaysia, Mexico, Netherlands, Norway, Poland, Portugal, Russian Federation, Singapore, Spain, Sweden, Switzerland, Taiwan (Province of China), Thailand, Turkey, United Kingdom, United States of America

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)