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Paroxysmal Nocturnal HemoglobinuriaPhase 3NCT06715943

Efficacy and Safety of HRS-5965 in Patients With PNH Who Are Still Anemia After Anti-C5 Antibody Treatment

A Phase 3 study testing HRS-5965 capsule in people with Paroxysmal Nocturnal Hemoglobinuria, run by Chengdu Suncadia Medicine Co., Ltd. at 2 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

This trial is a multi-center, single-arm, open-label phase III clinical trial. A total of approximately 35 patients with paroxysmal nocturnal hemoglobinuria who remained anemic despite stable use of C5 complement inhibitor (eculizumab/Kevacumab) for the first 6 months before randomization were included in the study. 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
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
39 participants
Where
2 sitesChina
Study dates
7 Jan 2025to 6 Jan 2026
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

  • Understand the specific process of the experiment, voluntarily participate in this experiment, and sign a written informed consent form.
  • Age ≥18 on the day of signing the informed consent, regardless of gender
  • It was confirmed to be PNH during screening, and the clone size of red blood cells or granulocytes was detected by flow cytopy >10%
  • Stable use of C5 complement inhibitor ikuzumab/covalimab for the first 6 months of random treatment
  • Have at least one blood transfusion record within the last 3 months, or sustain a hemoglobin level below 10g/dL during the eculizumab treatment within the last 3 months prior to screening.
  • The average hemoglobin level from two tests conducted by the central laboratory at the time of screening is less than 10 g/dL, with each individual Hb value being less than 10.5 g/dL.
  • Inoccution of Neisseris meningitis and Streptococcus pneumoniae vaccine at least 2 weeks before the first administration of HRS-5965; if HRS-5965 treatment must begin less than 2 weeks after vaccination, preventive antibiotic treatment must begin at least 2 weeks after vaccination.
  • Male and female subjects with fertility must agree to adopt efficient contraceptive measures with their partners within 30 days from the signing of the informed consent form to the last administration, and have no family planning and no sperm/egg donation.

Who cannot join

  • In addition to C5 complement inhibitors (ikuzuzumab/covalimamab), those who have participated in clinical trials of any other drug or medical device within 1 month before and are expected to have a residual effect of experimental treatment (judged by researchers), or those who were still in the follow-up period of a clinical trial or the 5 half-life of the experimental drug before screening Inside (whichever is longer)
  • Known or suspected hereditary or acquired complement deficiency
  • Currently active primary or secondary immunodeficiency
  • Those who have a history of splenectomy or plan to perform surgery during the trial
  • History of bone marrow/hematopoietic stem cells or solid organ transplantation
  • Diagnosed malignant tumors in the past 5 years
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  • There is laboratory evidence for patients with bone marrow failure during screening
  • History of infection with pod bacteria (such as Neisseris meningitis, Streptococcus pneumoniae, etc.)
  • There is or is suspected of systemic active bacteria, virus or fungal infection 2 weeks before the first administration of HRS-5965 (according to the researcher's judgment)
  • Fever occurs within 1 week before the first administration of HRS-5965 (body temperature ≥38 ℃)
  • Human immunodeficiency virus (HIV) infection
  • Hepatitis B surface antigen (HBsAg) and hepatitis C antibody (HCVAb) are positive during screening, or abnormal liver function test during screening
  • Use any of the following drugs, unless there is a stable treatment plan before screening: a) erythropoietin (ESA), hypoxic-inducing factor proaminoyl hydroxylase inhibitor (HIF-PHI) or immunosuppressant for at least 8 weeks b) Systemic use of glucocorticoids (≤15 mg/day Prednisone or equivalent doses of glucocorticoids) at least 4 weeks c) Vitamin K antagonists (such as warfarin) have a stable international standardized ratio (INR) at least 4 weeks d) Low molecular weight heparin, oral anticoagulants such as aspirin, rvaroxaban, apifloxaban, etc. at least 4 weeks e) Iron supplements , vitamin B12, folic acid or androgen for at least 4 weeks
  • During screening, there are serious concurrent diseases, such as severe kidney disease (such as eGFR<30 mL/min/1.73 m2, dialysis), advanced heart disease (such as NYHA level IV), severe lung disease such as pulmonary hypertension (WHO level IV) or liver disease (such as active hepatitis), etc. , judged by the researcher that it is not suitable to participate in the researcher
  • Any medical condition determined by the researcher that it may affect the patient's participation in the trial, chronic anemia or unstable thrombosis events that may exist for other causes, and other conditions judged by the researcher to be unsuitable for participation in the trial
  • Those who are suspected of being allergic to experimental drugs or any ingredient in experimental drugs
  • Screening positive blood pregnancy test and breastfeeding women at the time of the visit

Trial locations and contacts

2 sites in 1 country.

China2 sites

  • Hematology Hospital of Chinese Academy of Medical Sciences

    Tianjin, Tianjin Municipality, 30000

  • Tianjin Medical University General Hospital

    Tianjin, Tianjin Municipality, 30000

Other trials in China

About this study

Official title: Multicenter, Single-arm, Open-label Phase III Study to Evaluate Efficacy and Safety of HRS-5965 Capsule in Patients With PNH Who Are Still Anemia After Anti-C5 Antibody Treatment

This trial is a multi-center, single-arm, open-label phase III clinical trial. A total of approximately 35 patients with paroxysmal nocturnal hemoglobinuria who remained anemic despite stable use of C5 complement inhibitor (eculizumab/Kevacumab) for the first 6 months before randomization were included in the study. Approximately 40% of the subjects had received at least one red blood cell (RBC) transfusion within the first 6 months before receiving the experimental intervention. Subjects who met the criteria were all treated with HRS-5965 capsules. This trial includes an 8-week screening period, a 24-week treatment period, a 2-week dose reduction period, and a 4-week safety follow-up period.

Being tested
HRS-5965 capsule
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 treatment

Study groups in NCT06715943
Study groupRoleWhat they receive
HRS-5965 capsuleExperimentalNot described
Receives: Drug: HRS-5965 capsule

What is being measured

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

  1. In the absence of red blood cell infusion (defined as no red blood cell infusion after week 2 to week 24), the proportion of subjects with at least 3 times of ≥12 g/dL of hemoglobin level measured 4 times between weeks 18 and 24.

    Measured: From the 18th to the 24th week.

Secondary outcomes (10)

  1. The proportion of subjects who did not receive red blood cell infusion from week 2 to week 24

    Measured: From the 18th to the 24th week

  2. The average change of hemoglobin compared with baseline during weeks 18 to 24

    Measured: From the 18th to the 24th week

  3. The average percentage change of lactate dehydrogenase (LDH) level compared with the baseline during weeks 18 to 24

    Measured: From the 18th to the 24th week

  4. The average change of reticular red blood cell count compared with the baseline during weeks 18 to 24

    Measured: From the 18th to the 24th week

  5. Chronic Disease Treatment Function Assessment (FACIT) from weeks 18 to 24 - the average change of the score compared to the baseline

    Measured: From the 18th to the 24th week

  6. The incidence of breakthrough hemolysis from week 1 to week 24

    Measured: From the 18th to the 24th week

  7. The incidence of MAVEs (including thrombosis) from week 1 to week 24

    Measured: From the 1st to the 24th week

  8. The incidence and severity of adverse events from week 1 to week 24

    Measured: From the 1st to the 24th week

  9. The concentration of HRS-5965 in plasma

    Measured: From the 1st to the 24th week

  10. The percentage of the change of complement bypass pathway (AP) activity at each point in time compared with the baseline

    Measured: From the 1st to the 24th week

Timeline

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

  1. First posted4 Dec 2024
  2. Study start7 Jan 2025
  3. Primary completion6 Jan 2026
  4. Study completion6 Jan 2026

Resources

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
39 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06715943
Sponsor's protocol ID
HRS-5965-302
Status last verified
Jan 2026
First submitted
28 Nov 2024
Last updated
8 Jan 2026
Results posted
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Undecided

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing HRS-5965 capsule 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 8 inclusion and 17 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 8 Jan 2026. 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?

In the absence of red blood cell infusion (defined as no red blood cell infusion after week 2 to week 24), the proportion of subjects with at least 3 times of ≥12 g/dL of hemoglobin level measured 4 times between weeks 18 and 24. (time frame: From the 18th to the 24th week.). 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 NCT06715943.

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 8 Jan 2026.

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