Paroxysmal Nocturnal HemoglobinuriaPhase 3SolirisNCT05886244
Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China
A Phase 3 study testing Eculizumab in people with Paroxysmal Nocturnal Hemoglobinuria, run by Alexion Pharmaceuticals, Inc. at 7 sites in China.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria
- What is being tested?
- Eculizumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 7 sitesChina
- Study dates
- 5 Jul 2023to 14 Apr 2025
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Adult C5 inhibitor naïve PNH patients (age>=18), which is confirmed by flow cytometry evaluation
- Must be vaccinated against N meningitidis
Who cannot join
- Meningitidis infection or unresolved meningococcal disease
- Significant bone marrow failure
- Other significant systemic diseases that might have impact on efficacy and safety assessment
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
China7 sites
Research Site
Beijing, CN-100730
Research Site
Hangzhou, 310003
Research Site
Nantong, 226001
Research Site
Shanghai, 200040
Research Site
Tianjin, 300020
Research Site
Tianjin, 300050
Research Site
Wuhan, 430022
About this study
Official 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
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.
Read the detailed description
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.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Being tested
- Eculizumab
- Keywords
- paroxysmal nocturnal hemoglobinuria, PNH
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EculizumabDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| Eculizumab | Experimental | Eculizumab will be administered by IV infusion. Receives: Drug: Eculizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage Change From Baseline in LDH at 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.
Measured: Baseline, Week 12
Secondary outcomes (10)
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
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.
Measured: Baseline through Week 64
Serum Concentration of Eculizumab
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).
Measured: Day 1, Day 29, Day 85, Day 169, Day 253, Day 337, Day 421, Day 449
Change From Baseline in Serum Free Complement 5 (C5) Concentration
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.
Measured: Day 1, Day 29, Day 85, Day 169, Day 253, Day 337, Day 421, Day 449
Change From Baseline in Serum Total C5 Concentration
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.
Measured: Day 1, Day 29, Day 85, Day 169, Day 253, Day 337, Day 421, Day 449
Number of Participants With Treatment-emergent Antidrug Antibodies (ADAs) to Eculizumab
Blood samples were collected for measurement of treatment-emergent ADAs to eculizumab at specified timepoints.
Measured: Baseline through Week 64
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at 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.
Measured: Baseline, Week 12
Percentage of Participants With Breakthrough Hemolysis
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.
Measured: Baseline through Week 12
Percentage of Participants Achieving LDH Normalization
Blood samples were collected for measurement of LDH at specified timepoints. LDH normalization was defined as LDH ≤ULN.
Measured: Baseline through Week 12
Percentage of Participants Achieving Transfusion Avoidance
Transfusion avoidance was defined as remaining transfusion free (that is, had not received any transfusion) and did not require transfusion as per protocol.
Measured: Baseline through Week 12
Percentage of Participants With LDH ≤1.5* ULN at Week 12
Blood samples were collected for measurement of LDH at specified timepoints.
Measured: Baseline through Week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Jun 2023
- Study start5 Jul 2023
- Primary completion14 Apr 2025
- Study completion14 Apr 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Paroxysmal Nocturnal HemoglobinuriaStudies of the same condition, open and under way.
- More trials run by Alexion Pharmaceuticals, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- open label
- Masking
- None (open label)
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- AstraZeneca
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05886244
- Sponsor's protocol ID
- D7414C00001
- Other IDs
- ECU-PNH-301
- Acronym
- Soliris
- Status last verified
- May 2026
- First submitted
- 24 May 2023
- Last updated
- 1 Jun 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal. All request will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure. Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.
Study at a glance
From the registry record, last updated by the sponsor on 1 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT05886244
- Acronym / sponsor ID
- Soliris / D7414C00001
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Keywords
- paroxysmal nocturnal hemoglobinuria, PNH
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Collaborators
- AstraZeneca
- Study start
- 5 Jul 2023 (actual)
- Primary completion
- 14 Apr 2025 (actual)
- Study completion
- 14 Apr 2025 (actual)
- First posted
- 2 Jun 2023 (actual)
- Results
- Posted 1 Jun 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- open label
| Arm | Type | Receives |
|---|---|---|
| Eculizumab | Experimental | Drug: EculizumabEculizumab will be administered by IV infusion. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Adult C5 inhibitor naïve PNH patients (age>=18), which is confirmed by flow cytometry evaluation
- Must be vaccinated against N meningitidis
Key exclusion criteria (3 listed)
- Meningitidis infection or unresolved meningococcal disease
- Significant bone marrow failure
- Other significant systemic diseases that might have impact on efficacy and safety assessment
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EculizumabParticipants 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. | Drug | Eculizumab |
Primary outcome
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.
10 secondary outcomes
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Time frame: Baseline through Week 64
Serum Concentration of Eculizumab
Time frame: Day 1, Day 29, Day 85, Day 169, Day 253, Day 337, Day 421, Day 449
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
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
Number of Participants With Treatment-emergent Antidrug Antibodies (ADAs) to Eculizumab
Time frame: Baseline through Week 64
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at Week 12
Time frame: Baseline, Week 12
Percentage of Participants With Breakthrough Hemolysis
Time frame: Baseline through Week 12
Percentage of Participants Achieving LDH Normalization
Time frame: Baseline through Week 12
Percentage of Participants Achieving Transfusion Avoidance
Time frame: Baseline through Week 12
Percentage of Participants With LDH ≤1.5* ULN at Week 12
Time frame: Baseline through Week 12
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Jun 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Redacted CSR SynopsisLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal. All request will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure. Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.
Related trials for Paroxysmal Nocturnal Hemoglobinuria
Enrolling by invitationObservational study
An Observational Post-Authorization Safety and Effectiveness Study of Ravulizumab in Adult Patients Aged 18 and Older Diagnosed With Paroxysmal Nocturnal Hemoglobinuria Using Multicenter Electronic Medical Records in South Korea
Hemoglobinuria, Paroxysmal
RecruitingPhase 2
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells
Severe Aplastic Anemia, MDS (Myelodysplastic Syndrome)
RecruitingPhase 3
A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works
Paroxysmal Nocturnal Hemoglobinuria
Not yet recruitingPhase 3
A Study of Efficacy and Safety of FXS6837 in Anti-C5 Treated PNH Patients.
Paroxysmal Nocturnal Hemoglobinuria
RecruitingPhase 2
Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
Severe Aplastic Anemia (SAA), Hypo-Plastic Myelodysplastic Syndrome (MDS)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Eculizumab in people with Paroxysmal Nocturnal Hemoglobinuria, run by Alexion Pharmaceuticals, Inc. at 7 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 1 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 1 Jun 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage Change From Baseline in LDH at Week 12 (time frame: Baseline, Week 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Jun 2026.