Study of ALXN1210 compared to eculizumab in PNH patients who have never been treated with a complement inhibitor.

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

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2016-002025-11-CZ

Enrollment

214

Registered

2016-10-17

Start date

2017-02-02

Completion date

Unknown

Last updated

2025-01-13

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

Conditions

Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1 Level: LLT Classification code 10055629 Term: Paroxysmal nocturnal hemoglobinuria System Organ Class: 100000004857

Interventions

Product Code: ALXN1210 Pharmaceutical Form: Concentrate for solution for infusion INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Current Sponsor code: ALXN1210 Othe

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Male or female = 18 years of age 2. PNH diagnosis confirmed by 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 =65 years) yes F.1.3.1 Number of subjects for this age range 22

Exclusion criteria

Exclusion criteria: 1. Treatment with a complement inhibitor at any time 2. History of bone marrow transplantation 3. Body weight < 40 kilograms 4. Females who are pregnant, breastfeeding or who have a positive pregnancy test at screening or Day 1 5. Participation in another interventional clinical study or use of any experimental therapy within 30 days before initiation of study drug on Day 1 in this study or within 5 half-lives of that investigational product, whichever is greater. 6. History of or ongoing major cardiac, pulmonary, renal, endocrine, or hepatic disease that, in the opinion of the investigator or sponsor, would preclude participation 7. Unstable medical conditions (eg, myocardial ischemia, active gastrointestinal bleed, severe congestive heart failure, anticipated need for major surgery within 6 months of randomization, coexisting chronic anemia unrelated to PNH)

Design outcomes

Primary

Measure	Time frame
Main Objective: The purpose of this study is to assess ALXN1210 compared to eculizumab in adult patients with PNH who have never been treated with a complement inhibitor.;Secondary Objective: - safety and tolerability of ALXN1210 - efficacy - PK/PD and immunogenicity - long-term safety and efficacy - evaluate the safety and efficacy in patients who switch from eculizumab to ALXN1210 in the Extension Period;Primary end point(s): - Percentage of patients who achieve transfusion avoidance (TA) - Normalization of lactate dehydrogenase (LDH) levels ;Timepoint(s) of evaluation of this end point: 26 weeks	—

Measure

Time frame

Main Objective: The purpose of this study is to assess ALXN1210 compared to eculizumab in adult patients with PNH who have never been treated with a complement inhibitor.;Secondary Objective: - safety and tolerability of ALXN1210 - efficacy - PK/PD and immunogenicity - long-term safety and efficacy - evaluate the safety and efficacy in patients who switch from eculizumab to ALXN1210 in the Extension Period;Primary end point(s): - Percentage of patients who achieve transfusion avoidance (TA) - Normalization of lactate dehydrogenase (LDH) levels ;Timepoint(s) of evaluation of this end point: 26 weeks

—

Secondary

Measure	Time frame
Secondary end point(s): - Percentage change from baseline in lactate dehydrogenase ( LDH) levels - Change from baseline in quality of life as assessed by the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue - Percentage of patients with breakthrough hemolysis - Percentage of patients with stabilized hemoglobin ;Timepoint(s) of evaluation of this end point: 26 weeks	—

Countries

Austria, Belgium, Czech Republic, Denmark, Estonia, Finland, Germany, Italy, Korea, Republic of, Netherlands, Poland, Portugal, Spain, Sweden, United Kingdom, United States

Contacts

Public ContactEuropean Clinical Trial Information

Alexion Europe SAS

clinicaltrials.eu@alexion.com+33147100615

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026