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Completed

NCT Number: NCT05886244

Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China

This is a Phase 3b, single-arm, open-label, multicenter study to evaluate the efficacy, safety, pharmacokinetics (PK), pharmacodynamics (PD), and immunogenicity of eculizumab in adult participants with paraxysmal noturnal hemoglobinuria (PNH) in China.

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Key information

Conditions

Paroxysmal Nocturnal Hemoglobinuria Anemia Anemia, Hemolytic Bone Marrow Diseases Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinuria, Paroxysmal Myelodysplastic Syndromes

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Research Site, Beijing, China

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About this study

This is a Phase 3b, single-arm, open-label, multicenter study to evaluate the efficacy, safety, pharmacokinetics (PK), pharmacodynamics (PD), and immunogenicity of eculizumab in adult participants with PNH in China who previously have not been treated with complement inhibitors. Approximately 25 eligible participants in China will be enrolled.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Adult C5 inhibitor naïve PNH patients (age>=18), which is confirmed by flow cytometry evaluation
  • Must be vaccinated against N meningitidis

Exclusion criteria

  • Meningitidis infection or unresolved meningococcal disease
  • Significant bone marrow failure
  • Other significant systemic diseases that might have impact on efficacy and safety assessment

Treatment and study plan

Eculizumab

Drug

Participants will receive 600 milligrams (mg) once a week on Day 1, 8, 15, and 22 followed by 900 mg every 2 weeks from Day 29 to Day 435.

Primary outcomes

  1. Percentage Change From Baseline in LDH at Week 12

    Time frame: Baseline, Week 12

    Blood samples were collected for measurement of serum LDH at specified timepoints. Statistical analysis was performed using a mixed model for repeated measures (MMRM), including the percentage change from baseline of LDH at the scheduled visits from Week 1 to Week 12 as the dependent variable, with the fixed categorical effect of visit, fixed continuous effect of LDH baseline value as covariates, and participant as random effect. Assessed at baseline, 1, 2, 3, 4, 6, 8, 10, and 12 weeks, Week 12 reported. A decrease indicated a reduction in disease activity.

Secondary outcomes

  1. Number of Participants With Treatment-emergent Adverse Events (TEAEs)

    Time frame: Baseline through Week 64

    An adverse event (AE) was any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product that did not necessarily have to have a causal relationship with the study intervention. A TEAE was any AE that started during or after the first infusion of the study intervention. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.

  2. Serum Concentration of Eculizumab

    Time frame: Day 1, Day 29, Day 85, Day 169, Day 253, Day 337, Day 421, Day 449

    Blood samples were collected for measurement of serum concentrations of eculizumab at specified timepoints. No study intervention administered on Day 449. Results reported as micrograms/milliliter (ug/mL).

  3. Change From Baseline in Serum Free Complement 5 (C5) Concentration

    Time frame: Day 1, Day 29, Day 85, Day 169, Day 253, Day 337, Day 421, Day 449

    Blood samples were collected for measurement of serum concentration of free C5 at specified timepoints. No study intervention administered on Day 449. A decrease indicated a reduction in disease activity.

  4. Change From Baseline in Serum Total C5 Concentration

    Time frame: Day 1, Day 29, Day 85, Day 169, Day 253, Day 337, Day 421, Day 449

    Blood samples were collected for measurement of serum concentration of total C5 at specified timepoints. No study intervention administered on Day 449. A decrease indicated a reduction in disease activity.

  5. Number of Participants With Treatment-emergent Antidrug Antibodies (ADAs) to Eculizumab

    Time frame: Baseline through Week 64

    Blood samples were collected for measurement of treatment-emergent ADAs to eculizumab at specified timepoints.

  6. Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at Week 12

    Time frame: Baseline, Week 12

    The FACIT-Fatigue is a 13-item questionnaire that assesses self-reported fatigue and its impact upon daily activities and function over the preceding 7 days. The FACIT-Fatigue scale is a collection of quality of life (QoL) questionnaires pertaining to the management of fatigue symptoms due to a chronic illness. Participants scored each item on a 5-point scale: 0 (Not at all) to 4 (Very much). Total scores were calculated as the sum from all 13 items and ranged from 0 to 52, with higher scores indicating an increased QoL. Analysis was performed using a MMRM, including FACIT-Fatigue score change from baseline values at the scheduled visits from Week 1 to Week 12 as the dependent variable, with the fixed categorical effect of study visit, fixed continuous effect of baseline value of FACIT-Fatigue, and participant as random effect. Assessed at baseline, 1, 2, 3, 4, 6, 8, 10, and 12 weeks, Week 12 reported.

  7. Percentage of Participants With Breakthrough Hemolysis

    Time frame: Baseline through Week 12

    Breakthrough hemolysis was defined as at least 1 new or worsening symptom or sign of intravascular hemolysis (fatigue, hemoglobinuria, abdominal pain, shortness of breath [dyspnea], anemia [hemoglobin <10 grams/deciliter], major adverse vascular event [including thrombosis], dysphagia, or erectile dysfunction) in the presence of elevated LDH ≥2 times (*) upper limit of normal (ULN), after prior LDH reduction to <1.5* ULN on therapy.

  8. Percentage of Participants Achieving LDH Normalization

    Time frame: Baseline through Week 12

    Blood samples were collected for measurement of LDH at specified timepoints. LDH normalization was defined as LDH ≤ULN.

  9. Percentage of Participants Achieving Transfusion Avoidance

    Time frame: Baseline through Week 12

    Transfusion avoidance was defined as remaining transfusion free (that is, had not received any transfusion) and did not require transfusion as per protocol.

  10. Percentage of Participants With LDH ≤1.5* ULN at Week 12

    Time frame: Baseline through Week 12

    Blood samples were collected for measurement of LDH at specified timepoints.

Sponsors and collaborators

Lead sponsor

Alexion Pharmaceuticals, Inc.

Industry

Collaborators

  • AstraZeneca

Registry information

Official study title

Open-Label, Multicenter Study to Assess the Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Eculizumab in Complement Inhibitor Treatment Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China

Acronym: Soliris

Important dates

Study start
2023
Primary completion
2025
Study completion
2025
First posted
Jun 2, 2023
Registry last updated
Jun 1, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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