Paroxysmal Nocturnal HemoglobinuriaPhase 3NCT06578949

Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adults Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)

A Phase 3 study testing Ravulizumab in people with Paroxysmal Nocturnal Hemoglobinuria and PNH, run by Alexion Pharmaceuticals, Inc. at 8 sites in China.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

The primary objective of this study is to evaluate the efficacy of ravulizumab in adult participants with PNH. Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Paroxysmal Nocturnal Hemoglobinuria, PNH
What is being tested?: Ravulizumab
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 18 participants
Where: 8 sitesChina
Study dates: 10 Oct 2024to 22 Dec 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 naive PNH patients (age>=18), which is confirmed by flow cytometry evaluation.
Must be vaccinated againast N meningitidis.

Who cannot join

Meningitidis infection or unresolved meningococcal disease
History of bone marrow transplantation
Other significant systemic diseases that might have impact on efficacy and safety assessment

Trial locations and contacts

8 sites in 1 country.

City, postcode or hospital

8 sites in 1 country

China8 sites

Research Site
Beijing, CN-100730
Research Site
Guangzhou, 510100
Research Site
Hangzhou, 310003
Research Site
Nantong, 226001
Research Site
Shanghai, 200040
Research Site
Tianjin, 300020
Research Site
Tianjin, 300050
Research Site
Wuhan, 430022

Other trials in China

About this study

Official title: A Phase 3, Single-arm, Open-label, Multicenter Study to Assess the Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Complement Inhibitor Treatment Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China

The primary objective of this study is to evaluate the efficacy of ravulizumab in adult participants with PNH.

Conditions: Paroxysmal Nocturnal Hemoglobinuria, PNH
Being tested: Ravulizumab
Keywords: Paroxysmal Nocturnal Hemoglobinuria, PNH, Ravulizumab
Medical subject headings: Hemoglobinuria, Paroxysmal

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

RavulizumabDrug
Also called ALXN1210
Ravulizumab will be administered by intravenous (IV) infusion.

Study groups in NCT06578949
Study group	Role	What they receive
Ravulizumab	Experimental	During the Primary Treatment Period, participants will receive a weight-based loading dose of ravulizumab on Day 1 followed by weight-based maintenance dose of ravulizumab on Day 15 and once every 8 weeks (q8w) thereafter for a total of 26 weeks. On Day 183, all participants will enter a 32-week Extension Treatment Period and receive ravulizumab. Beginning on Day 183, participants will receive a maintenance dose of ravulizumab q8w for an additional 32 weeks. Receives: Drug: Ravulizumab

What is being measured

The primary outcome is the main question the study is designed to answer.

Percentage Change in Lactate Dehydrogenase (LDH) From Baseline to Day 183 (Week 26)
LDH is an indicator of intravascular hemolysis that occurs in participants with paroxysmal nocturnal hemoglobinuria. A decrease in LDH indicated reduction (improvement) in hemolysis. Baseline was defined as the average of all available on-study assessments prior to the first dose of study drug. The percent change in LDH was analyzed using a mixed-effect model for repeated measures (MMRM) with the fixed categorical effect of visit, fixed continuous effect of the LDH baseline value as covariates, and participant as random effect.
Measured: Baseline, Day 183 (Week 26)

Secondary outcomes (5)

Percentage of Participants Achieving LDH <1.5 * Upper Limit of Normal (ULN) at Day 183 (Week 26)
LDH is an indicator of intravascular hemolysis that occurs in participants with paroxysmal nocturnal hemoglobinuria. A decrease in LDH indicated reduction (improvement) in hemolysis. Baseline was defined as the average of all available on-study assessments prior to the first dose of study drug.
Measured: Day 183 (Week 26)
Percentage of Participants Achieving Transfusion Avoidance Through Day 183 (Week 26)
Transfusion avoidance was defined as the percentage of participants who remained transfusion free and did not require a transfusion per protocol-specified guidelines (hemoglobin value of ≤9 grams (g)/deciliter (dL) with signs or symptoms of sufficient severity to warrant a transfusion, or a hemoglobin value of ≤7 g/dL regardless of presence of clinical signs or symptoms) through Day 183.
Measured: Day 183 (Week 26)
Percentage of Participants Experiencing Breakthrough Hemolysis Through Day 183 (Week 26)
Breakthrough hemolysis was defined as at least one new or worsening symptom or sign of intravascular hemolysis (fatigue, hemoglobinuria, abdominal pain, dyspnea, anemia [hemoglobin <10 g/dL], major adverse vascular event [including thrombosis], dysphagia, or erectile dysfunction) in the presence of elevated LDH ≥2 times the ULN.
Measured: Day 183 (Week 26)
Change in Functional Assessment of Chronic Illness Therapy-Fatigue Scale (FACIT-Fatigue) Score From Baseline to Day 183 (Week 26)
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. Participants scored each item on a 5-point scale: 0 (Not at all) to 4 (Very much). Total scores range from 0 to 52, with a higher score indicating better quality of life. Analysis was using a mixed-effect model for repeated measures (MMRM) with the fixed categorical effect of visit, fixed continuous effect of the baseline value of FACIT-Fatigue score as covariates, and participant as random effect.
Measured: Baseline, Day 183 (Week 26)
Change in Hemoglobin (Hgb) From Baseline to Day 183 (Week 26)
Analysis was performed using MMRM with the fixed categorical effect of visit, fixed continuous effect of the Hgb baseline value as covariates, and participant as random effect.
Measured: Baseline, Day 183 (Week 26)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

10 Oct 202422 Dec 2025

First posted30 Aug 2024
Study start10 Oct 2024
Primary completion15 May 2025
Study completion22 Dec 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
Masking: None (open label)
Enrolment: 18 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT06578949
Sponsor's protocol ID: D9289C00008
Other IDs: ALXN1210-PNH-323
Status last verified: May 2026
First submitted: 28 Aug 2024
Last updated: 5 Jun 2026
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: Yes: Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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)

All Paroxysmal Nocturnal Hemoglobinuria trials

Study at a glance

From the registry record, last updated by the sponsor on 5 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 3, Single-arm, Open-label, Multicenter Study to Assess the Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Complement Inhibitor Treatment Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China
ClinicalTrials.gov ID: NCT06578949
Acronym / sponsor ID: D9289C00008
Conditions: Paroxysmal Nocturnal Hemoglobinuria, PNH
Keywords: Paroxysmal Nocturnal Hemoglobinuria, PNH, Ravulizumab
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 18 participants (actual)
Lead sponsor: Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
Study start: 10 Oct 2024 (actual)
Primary completion: 15 May 2025 (actual)
Study completion: 22 Dec 2025 (actual)
First posted: 30 Aug 2024 (actual)
Results: Posted 5 Jun 2026

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

The primary objective of this study is to evaluate the efficacy of ravulizumab in adult participants with PNH.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Ravulizumab	Experimental	Drug: RavulizumabDuring the Primary Treatment Period, participants will receive a weight-based loading dose of ravulizumab on Day 1 followed by weight-based maintenance dose of ravulizumab on Day 15 and once every 8 weeks (q8w) thereafter for a total of 26 weeks. On Day 183, all participants will enter a 32-week Extension Treatment Period and receive ravulizumab. Beginning on Day 183, participants will receive a maintenance dose of ravulizumab q8w for an additional 32 weeks.

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 naive PNH patients (age>=18), which is confirmed by flow cytometry evaluation.
Must be vaccinated againast N meningitidis.

Key exclusion criteria (3 listed)

Meningitidis infection or unresolved meningococcal disease
History of bone marrow transplantation
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
RavulizumabAlso: ALXN1210Ravulizumab will be administered by intravenous (IV) infusion.	Drug	Ravulizumab

Primary outcome

Percentage Change in Lactate Dehydrogenase (LDH) From Baseline to Day 183 (Week 26)
Time frame: Baseline, Day 183 (Week 26)
LDH is an indicator of intravascular hemolysis that occurs in participants with paroxysmal nocturnal hemoglobinuria. A decrease in LDH indicated reduction (improvement) in hemolysis. Baseline was defined as the average of all available on-study assessments prior to the first dose of study drug. The percent change in LDH was analyzed using a mixed-effect model for repeated measures (MMRM) with the fixed categorical effect of visit, fixed continuous effect of the LDH baseline value as covariates, and participant as random effect.

5 secondary outcomes

Percentage of Participants Achieving LDH <1.5 * Upper Limit of Normal (ULN) at Day 183 (Week 26)
Time frame: Day 183 (Week 26)
Percentage of Participants Achieving Transfusion Avoidance Through Day 183 (Week 26)
Time frame: Day 183 (Week 26)
Percentage of Participants Experiencing Breakthrough Hemolysis Through Day 183 (Week 26)
Time frame: Day 183 (Week 26)
Change in Functional Assessment of Chronic Illness Therapy-Fatigue Scale (FACIT-Fatigue) Score From Baseline to Day 183 (Week 26)
Time frame: Baseline, Day 183 (Week 26)
Change in Hemoglobin (Hgb) From Baseline to Day 183 (Week 26)
Time frame: Baseline, Day 183 (Week 26)

Results and safety

Results posted to the registry on 5 Jun 2026, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Ravulizumab	18	18

Primary outcome: Percentage Change in Lactate Dehydrogenase (LDH) From Baseline to Day 183 (Week 26)

Time frame: Baseline, Day 183 (Week 26). Least squares mean (95% Confidence Interval). Unit: percent change

Measure	Ravulizumab n = 17
Value	-81.3 (-84.2 to -78.3)

Ravulizumab: p < 0.001, Mixed Models Analysis

Population analysed: FAS included all enrolled participants who received at least 1 dose (full or partial) of the study intervention. 'Overall number of participants analyzed' = participants evaluable for this outcome measure.

5 secondary outcomes are reported in the full results.

Adverse events

Time frame: Baseline up to Week 26. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Ravulizumab	2 of 18 (11.1%)	16 of 18 (88.9%)	0 of 18

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 5 Jun 2026.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related InfoLink provided by the sponsor.
Individual participant dataSharing planned: Yes. Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.

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.

8 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 Ravulizumab in people with Paroxysmal Nocturnal Hemoglobinuria and PNH, run by Alexion Pharmaceuticals, Inc. at 8 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 5 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 8 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 5 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 in Lactate Dehydrogenase (LDH) From Baseline to Day 183 (Week 26) (time frame: Baseline, Day 183 (Week 26)). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06578949.

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 5 Jun 2026.