Paroxysmal Nocturnal HemoglobinuriaPhase 3NCT06593938
A Study to Evaluate the Efficacy and Safety of Oral HRS-5965 in Adult Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Who Are Naive to Complement Inhibitor Therapy
A Phase 3 study testing HRS-5965 capsule and Eculizumab Injection in people with Paroxysmal Nocturnal Hemoglobinuria, run by Chengdu Suncadia Medicine Co., Ltd. at 2 sites in China.
Trial summary
A study of the efficacy and safety of HRS-5965 capsules compared to eculizumab for 24 weeks in patients with PNH. 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?
- HRS-5965 capsule and Eculizumab Injection
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 76 participants
- Where
- 2 sitesChina
- Study dates
- 25 Oct 2024to 31 Jul 2025
- Phase
- Phase 3
- Are there results?
- None posted in the registry
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
- Diagnosis of PNH confirmed by flow cytometry with clone size > 10%.
- Have not received complement inhibitor therapy;
- LDH > 1.5*ULN at screening.
- Hemoglobin level < 10 g/dL at screening.
Who cannot join
- Known or suspected hereditary or acquired complement deficiency;
- Patients with laboratory evidence of bone marrow failure (reticulocytes <100x109/L; platelets <30x109/L; neutrophils <0.5x109/L);
- Presence or suspicion of a systemic active bacterial, viral, or fungal infection (based on judgment of the investigator) within 2 weeks prior to the first dose of HRS-5965;
- History of infection with capsular bacteria (e.g., meningococcus, pneumococcus, etc.)
- Positive of HIV, HBsAg or HCVAb.
Trial locations and contacts
2 sites in 1 country.
China2 sites
Peking Union Medical College Hospital, Chinese Academy of Medical Sciences
Beijing, Beijing Municipality, 100730
The Blood Disease Hospital of the Chinese Academy of Medical Sciences
Tianjin, Tianjin Municipality, 300020
About this study
Official title: A Multicenter, Randomized, Open-label, Active-comparator Controlled Phase III Study to Evaluate the Efficacy and Safety of HRS-5965 Capsule in Patients With Paroxysmal Nocturnal Hemoglobinuria
A study of the efficacy and safety of HRS-5965 capsules compared to eculizumab for 24 weeks in patients with PNH.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Being tested
- HRS-5965 capsule, Eculizumab Injection
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HRS-5965 capsuleDrug
HRS-5965 capsule for 24 weeks
Eculizumab InjectionDrug
Eculizumab Injection for 24 weeks
| Study group | Role | What they receive |
|---|---|---|
| HRS-5965 capsule | Experimental | Not described Receives: Drug: HRS-5965 capsule |
| Eculizumab Injection | Experimental | Not described Receives: Drug: Eculizumab Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of subjects with the hemoglobin levels are ≥ 12 g/dL at least on three out of four measurements in the absence of red blood cell transfusions
Measured: 24 weeks
Secondary outcomes (6)
Proportion of subjects with the change from baseline in hemoglobin is ≥ 2 g/dL at least on three out of four measurements in the absence of red blood cell transfusions
Measured: 24 weeks
Proportion of subjects who absence of administration of red blood cell transfusions
Measured: 24 weeks
Mean change from baseline in hemoglobin
Measured: 24 weeks
Mean percent change from baseline in LDH levels
Measured: 24 weeks
Mean change from baseline in reticulocyte counts
Measured: 24 weeks
Mean change from baseline in FACIT-Fatigue scores
Measured: 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Sep 2024
- Study start25 Oct 2024
- Primary completion31 Jul 2025
- Study completion31 Jul 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Paroxysmal Nocturnal HemoglobinuriaStudies of the same condition, open and under way.
- 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 76 participants (actual)
Sponsor and investigators
- Lead sponsor
- Chengdu Suncadia Medicine Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06593938
- Sponsor's protocol ID
- HRS-5965-301
- Status last verified
- Sep 2025
- First submitted
- 10 Sep 2024
- Last updated
- 11 Sep 2025
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 11 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Randomized, Open-label, Active-comparator Controlled Phase III Study to Evaluate the Efficacy and Safety of HRS-5965 Capsule in Patients With Paroxysmal Nocturnal Hemoglobinuria
- ClinicalTrials.gov ID
- NCT06593938
- Acronym / sponsor ID
- HRS-5965-301
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 76 participants (actual)
- Lead sponsor
- Chengdu Suncadia Medicine Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 25 Oct 2024 (actual)
- Primary completion
- 31 Jul 2025 (actual)
- Study completion
- 31 Jul 2025 (actual)
- First posted
- 19 Sep 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A study of the efficacy and safety of HRS-5965 capsules compared to eculizumab for 24 weeks in patients with PNH.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| HRS-5965 capsule | Experimental | Drug: HRS-5965 capsule |
| Eculizumab Injection | Experimental | Drug: Eculizumab Injection |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Diagnosis of PNH confirmed by flow cytometry with clone size > 10%.
- Have not received complement inhibitor therapy;
- LDH > 1.5*ULN at screening.
- Hemoglobin level < 10 g/dL at screening.
Key exclusion criteria (5 listed)
- Known or suspected hereditary or acquired complement deficiency;
- Patients with laboratory evidence of bone marrow failure (reticulocytes <100x109/L; platelets <30x109/L; neutrophils <0.5x109/L);
- Presence or suspicion of a systemic active bacterial, viral, or fungal infection (based on judgment of the investigator) within 2 weeks prior to the first dose of HRS-5965;
- History of infection with capsular bacteria (e.g., meningococcus, pneumococcus, etc.)
- Positive of HIV, HBsAg or HCVAb.
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 |
|---|---|---|
| HRS-5965 capsuleHRS-5965 capsule for 24 weeks | Drug | HRS-5965 capsule |
| Eculizumab InjectionEculizumab Injection for 24 weeks | Drug | Eculizumab Injection |
Primary outcome
Proportion of subjects with the hemoglobin levels are ≥ 12 g/dL at least on three out of four measurements in the absence of red blood cell transfusions
Time frame: 24 weeks
6 secondary outcomes
Proportion of subjects with the change from baseline in hemoglobin is ≥ 2 g/dL at least on three out of four measurements in the absence of red blood cell transfusions
Time frame: 24 weeks
Proportion of subjects who absence of administration of red blood cell transfusions
Time frame: 24 weeks
Mean change from baseline in hemoglobin
Time frame: 24 weeks
Mean percent change from baseline in LDH levels
Time frame: 24 weeks
Mean change from baseline in reticulocyte counts
Time frame: 24 weeks
Mean change from baseline in FACIT-Fatigue scores
Time frame: 24 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Sep 2025.
- Individual participant dataSharing planned: Undecided
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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
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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.
2 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 HRS-5965 capsule and Eculizumab Injection in people with Paroxysmal Nocturnal Hemoglobinuria, run by Chengdu Suncadia Medicine Co., Ltd. at 2 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 4 inclusion and 5 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 11 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Proportion of subjects with the hemoglobin levels are ≥ 12 g/dL at least on three out of four measurements in the absence of red blood cell transfusions (time frame: 24 weeks). 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 11 Sep 2025.