Paroxysmal Nocturnal HemoglobinuriaPhase 2NCT06238544
Long-term Safety and Tolerability of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria
A Phase 2 study testing HRS-5965 tablets and HRS-5965 Capsules in people with Paroxysmal Nocturnal Hemoglobinuria, run by Chengdu Suncadia Medicine Co., Ltd. at 2 sites in China.
Trial summary
This is a multicenter, single-arm, open-label study. Patients with Paroxysmal Nocturnal Hemoglobinuria who had previously received and completed the HRS-5965 study well included. All eligible subjects received HRS-5965 tablets or capsules until the end of treatment in this 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 tablets and HRS-5965 Capsules
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 132 participants
- Where
- 2 sitesChina
- Study dates
- 6 Feb 2024to Dec 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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
- 1. Patients who have previously received and completed HRS-5965 study treatment, and are judged by the investigator to have treatment benefit and may benefit from continued treatment of HRS-5965 and patients in the control group of who received eculizumab treatment.
Who cannot join
- Known or suspected hereditary or acquired complement deficiency;
- History of infection with capsular bacteria (e.g., meningococcus, pneumococcus, etc.)
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: An Open Label, Multicenter Study to Evaluate the Long-term Safety and Tolerability of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria
This is a multicenter, single-arm, open-label study. Patients with Paroxysmal Nocturnal Hemoglobinuria who had previously received and completed the HRS-5965 study well included. All eligible subjects received HRS-5965 tablets or capsules until the end of treatment in this study.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Being tested
- HRS-5965 tablets, HRS-5965 Capsules
- 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 tabletsDrug
HRS-5965 tablets until the end of study treatment
HRS-5965 CapsulesDrug
HRS-5965 capsules.
| Study group | Role | What they receive |
|---|---|---|
| HRS-5965 Group | Experimental | Not described Receives: Drug: HRS-5965 tablets; Drug: HRS-5965 Capsules |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and severity of adverse events.
Measured: Every six months,for about 3 years
Secondary outcomes (5)
Proportion of participants achieving sustained hemoglobin levels ≥ 12 g/dL in the absence of red blood cell transfusions was assessed every half a year during treatment..
Measured: Every six months ,for about 3 years
Proportion of participants who remain free from transfusions was assessed every half a year during treatment..
Measured: Every six months,for about 3 years
Rate of breakthrough hemolysis was assessed every half a year during treatment
Measured: Every six months,for about 3 years
Proportion of participants with Major Adverse Vascular Events MAVEs.
Measured: Every six months,for about 3 years
Plasma concentration of HRS-5965.
Measured: Start of Treatment to end of study，for about 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Feb 2024
- Study start6 Feb 2024
- Primary completionDec 2027 (estimated)
- Study completionDec 2027 (estimated)
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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 132 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
- NCT06238544
- Sponsor's protocol ID
- HRS-5965-203
- Status last verified
- Dec 2025
- First submitted
- 22 Jan 2024
- Last updated
- 24 Dec 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 24 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label, Multicenter Study to Evaluate the Long-term Safety and Tolerability of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria
- ClinicalTrials.gov ID
- NCT06238544
- Acronym / sponsor ID
- HRS-5965-203
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 132 participants (actual)
- Lead sponsor
- Chengdu Suncadia Medicine Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 6 Feb 2024 (actual)
- Primary completion
- Dec 2027 (estimated)
- Study completion
- Dec 2027 (estimated)
- First posted
- 2 Feb 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.
This is a multicenter, single-arm, open-label study. Patients with Paroxysmal Nocturnal Hemoglobinuria who had previously received and completed the HRS-5965 study well included. All eligible subjects received HRS-5965 tablets or capsules until the end of treatment in this study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| HRS-5965 Group | Experimental | Drug: HRS-5965 tablets, Drug: HRS-5965 Capsules |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- 1. Patients who have previously received and completed HRS-5965 study treatment, and are judged by the investigator to have treatment benefit and may benefit from continued treatment of HRS-5965 and patients in the control group of who received eculizumab treatment.
Key exclusion criteria (2 listed)
- Known or suspected hereditary or acquired complement deficiency;
- History of infection with capsular bacteria (e.g., meningococcus, pneumococcus, etc.)
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 tabletsHRS-5965 tablets until the end of study treatment | Drug | HRS-5965 Group |
| HRS-5965 CapsulesHRS-5965 capsules. | Drug | HRS-5965 Group |
Primary outcome
Incidence and severity of adverse events.
Time frame: Every six months,for about 3 years
5 secondary outcomes
Proportion of participants achieving sustained hemoglobin levels ≥ 12 g/dL in the absence of red blood cell transfusions was assessed every half a year during treatment..
Time frame: Every six months ,for about 3 years
Proportion of participants who remain free from transfusions was assessed every half a year during treatment..
Time frame: Every six months,for about 3 years
Rate of breakthrough hemolysis was assessed every half a year during treatment
Time frame: Every six months,for about 3 years
Proportion of participants with Major Adverse Vascular Events MAVEs.
Time frame: Every six months,for about 3 years
Plasma concentration of HRS-5965.
Time frame: Start of Treatment to end of study，for about 3 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Dec 2025.
- Individual participant dataSharing planned: Undecided
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Enrolling by invitationObservational study
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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 2 study testing HRS-5965 tablets and HRS-5965 Capsules 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 1 inclusion and 2 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: under way, not looking for participants, as of the sponsor's last update on 24 Dec 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
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?
Incidence and severity of adverse events. (time frame: Every six months,for about 3 years). 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 24 Dec 2025.