Paroxysmal Nocturnal HemoglobinuriaPhase 2NCT06051357
Proof of Concept Study to Assess the Efficacy, Safety of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria
A Phase 2 study testing HRS-5965 tablets in people with Paroxysmal Nocturnal Hemoglobinuria, run by Chengdu Suncadia Medicine Co., Ltd. at 2 sites in China.
Trial summary
This is a multicenter, randomized, open-label phase II clinical trial. A total of 24 patients with paroxysmal nocturnal hemoglobinuria naïve to complement inhibitor therapy were included. Subjects were treated with HRS-5965 for 12 weeks. 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
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 26 participants
- Where
- 2 sitesChina
- Study dates
- 15 Nov 2023to 25 Mar 2024
- Phase
- Phase 2
- 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;
- Hemoglobin level < 10 g/dL.
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: An Multicenter, Randomized, Open-label Proof of Concept Study to Assess the Efficacy, Safety of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
This is a multicenter, randomized, open-label phase II clinical trial. A total of 24 patients with paroxysmal nocturnal hemoglobinuria naïve to complement inhibitor therapy were included. Subjects were treated with HRS-5965 for 12 weeks.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Being tested
- HRS-5965 tablets
- 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 for 12 weeks
| Study group | Role | What they receive |
|---|---|---|
| Treatment group A | Experimental | Not described Receives: Drug: HRS-5965 tablets |
| Treatment group B | Experimental | Not described Receives: Drug: HRS-5965 tablets |
What is being measured
The primary outcome is the main question the study is designed to answer.
Changes in hemoglobin.
Measured: 12 weeks
Secondary outcomes (10)
Changes in LDH.
Measured: 12 weeks
Changes in haptoglobin.
Measured: 12 weeks
Changes in bilirubin.
Measured: 12 weeks
Changes in reticulocyte counts.
Measured: 12weeks
Changes in C3 complement fragment deposition.
Measured: 12 week
Percentage of patients who did not receive a blood transfusion.
Measured: 12 weeks
Number of RBC units of transfused.
Measured: 12weeks
Incidence and severity of adverse events
Measured: 16 weeks
Incidence of thromboembolic events.
Measured: 16 weeks
Plasma concentration of HRS-5965.
Measured: 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Sep 2023
- Study start15 Nov 2023
- Primary completion25 Mar 2024
- Study completion25 Mar 2024
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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 26 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
- NCT06051357
- Sponsor's protocol ID
- HRS-5965-202
- Status last verified
- Oct 2024
- First submitted
- 5 Sep 2023
- Last updated
- 15 Oct 2024
- 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 15 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Multicenter, Randomized, Open-label Proof of Concept Study to Assess the Efficacy, Safety of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
- ClinicalTrials.gov ID
- NCT06051357
- Acronym / sponsor ID
- HRS-5965-202
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 26 participants (actual)
- Lead sponsor
- Chengdu Suncadia Medicine Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 15 Nov 2023 (actual)
- Primary completion
- 25 Mar 2024 (actual)
- Study completion
- 25 Mar 2024 (actual)
- First posted
- 22 Sep 2023 (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, randomized, open-label phase II clinical trial. A total of 24 patients with paroxysmal nocturnal hemoglobinuria naïve to complement inhibitor therapy were included. Subjects were treated with HRS-5965 for 12 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Treatment group A | Experimental | Drug: HRS-5965 tablets |
| Treatment group B | Experimental | Drug: HRS-5965 tablets |
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;
- Hemoglobin level < 10 g/dL.
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 tabletsHRS-5965 tablets for 12 weeks | Drug | Treatment group A, Treatment group B |
Primary outcome
Changes in hemoglobin.
Time frame: 12 weeks
10 secondary outcomes
Changes in LDH.
Time frame: 12 weeks
Changes in haptoglobin.
Time frame: 12 weeks
Changes in bilirubin.
Time frame: 12 weeks
Changes in reticulocyte counts.
Time frame: 12weeks
Changes in C3 complement fragment deposition.
Time frame: 12 week
Percentage of patients who did not receive a blood transfusion.
Time frame: 12 weeks
Number of RBC units of transfused.
Time frame: 12weeks
Incidence and severity of adverse events
Time frame: 16 weeks
Incidence of thromboembolic events.
Time frame: 16 weeks
Plasma concentration of HRS-5965.
Time frame: 12 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 15 Oct 2024.
- Individual participant dataSharing planned: Undecided
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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 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 15 Oct 2024. 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?
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?
Changes in hemoglobin. (time frame: 12 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 15 Oct 2024.