Paroxysmal Nocturnal HemoglobinuriaPhase 2NCT06745622
Long-term Safety and Tolerability of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria
A Phase 2 study testing HSK39297 tablets in people with Paroxysmal Nocturnal Hemoglobinuria, run by Haisco Pharmaceutical Group Co., Ltd. at 1 site in China.
Trial summary
This is a multicenter, open-label study to evaluate the long-term safety, tolerability and efficacy of HSK39297. Adult patients with PNH who had previously received and completed HSK39297 study treatment will be included. Eligible subjects can maintain HSK39297 treatment until the end of 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?
- HSK39297 tablets
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 47 participants
- Where
- 1 siteChina
- Study dates
- 3 Jan 2025to Jun 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
- Patients With PNH who have previously received and completed HSK39297 treatment , and According to the researchers' judgment, the benefits of treatment outweigh the risks and may benefit from continued treatment with HSK39297;
- Understand the study procedures and methods, voluntarily participate in this trial.
Who cannot join
- Hereditary or acquired complement deficiency;
- Active primary or secondary immunodeficiency;
- History of splenectomy, bone marrow/ hematopoietic stem cell or solid organ transplants;
- History of recurrent invasive infections caused by encapsulated organisms( e.g. meningococcus or pneumococcus) or Mycobacterium tuberculosis;
- History of serious comorbidities that have been determined to be unsuitable for participation in the study;
- Pregnant or Lactating women.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
The First Affiliated Hospital of Nanjing Medical University
Nanjing, Jiangsu
He, +8615312052798, heguangsheng1972@sina.com
About this study
Official title: A Multicenter, Open-label Study to Evaluate the Long-term Safety, Tolerability and Efficacy of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria（PNH）
This is a multicenter, open-label study to evaluate the long-term safety, tolerability and efficacy of HSK39297. Adult patients with PNH who had previously received and completed HSK39297 study treatment will be included. Eligible subjects can maintain HSK39297 treatment until the end of the study.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Being tested
- HSK39297 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.
HSK39297 tabletsDrug
HSK39297 tablets until the end of treatment
| Study group | Role | What they receive |
|---|---|---|
| HSK39297 | Experimental | Not described Receives: Drug: HSK39297 tablets |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and severity of adverse events
Measured: through study completion，about 2 years
Secondary outcomes (5)
Proportion of participants achieving hemoglobin levels ≥ 12 g/dL in the absence of red blood cell transfusions
Measured: through study completion，about 2 years
Change From Baseline in Hemoglobin in the absence of red blood cell transfusions
Measured: through study completion，about 2 years
Proportion of participants who remain free from transfusions
Measured: through study completion，about 2 years
Rate of breakthrough hemolysis (BTH)
Measured: through study completion，about 2 years
Proportion of participants with Major Adverse Vascular Events (MAVEs)
Measured: through study completion，about 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Dec 2024
- Study start3 Jan 2025 (estimated)
- Primary completionJun 2027 (estimated)
- Study completionJun 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.
- More trials run by Haisco Pharmaceutical Group Co., Ltd.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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 47 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Haisco Pharmaceutical Group Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06745622
- Sponsor's protocol ID
- HSK39297-203
- Status last verified
- Dec 2024
- First submitted
- 12 Dec 2024
- Last updated
- 20 Dec 2024
- Results posted
- No
- Data monitoring committee
- 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 20 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Open-label Study to Evaluate the Long-term Safety, Tolerability and Efficacy of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria（PNH）
- ClinicalTrials.gov ID
- NCT06745622
- Acronym / sponsor ID
- HSK39297-203
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 47 participants (estimated)
- Lead sponsor
- Haisco Pharmaceutical Group Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 3 Jan 2025 (estimated)
- Primary completion
- Jun 2027 (estimated)
- Study completion
- Jun 2027 (estimated)
- First posted
- 20 Dec 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, open-label study to evaluate the long-term safety, tolerability and efficacy of HSK39297. Adult patients with PNH who had previously received and completed HSK39297 study treatment will be included. Eligible subjects can maintain HSK39297 treatment until the end of the study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| HSK39297 | Experimental | Drug: HSK39297 tablets |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Patients With PNH who have previously received and completed HSK39297 treatment , and According to the researchers' judgment, the benefits of treatment outweigh the risks and may benefit from continued treatment with HSK39297;
- Understand the study procedures and methods, voluntarily participate in this trial.
Key exclusion criteria (6 listed)
- Hereditary or acquired complement deficiency;
- Active primary or secondary immunodeficiency;
- History of splenectomy, bone marrow/ hematopoietic stem cell or solid organ transplants;
- History of recurrent invasive infections caused by encapsulated organisms( e.g. meningococcus or pneumococcus) or Mycobacterium tuberculosis;
- History of serious comorbidities that have been determined to be unsuitable for participation in the study;
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 |
|---|---|---|
| HSK39297 tabletsHSK39297 tablets until the end of treatment | Drug | HSK39297 |
Primary outcome
Incidence and severity of adverse events
Time frame: through study completion，about 2 years
5 secondary outcomes
Proportion of participants achieving hemoglobin levels ≥ 12 g/dL in the absence of red blood cell transfusions
Time frame: through study completion，about 2 years
Change From Baseline in Hemoglobin in the absence of red blood cell transfusions
Time frame: through study completion，about 2 years
Proportion of participants who remain free from transfusions
Time frame: through study completion，about 2 years
Rate of breakthrough hemolysis (BTH)
Time frame: through study completion，about 2 years
Proportion of participants with Major Adverse Vascular Events (MAVEs)
Time frame: through study completion，about 2 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 20 Dec 2024.
- Individual participant dataSharing planned: Undecided
Related trials for Paroxysmal Nocturnal Hemoglobinuria
Enrolling by invitationObservational study
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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
Severe Aplastic Anemia, MDS (Myelodysplastic Syndrome)
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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
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing HSK39297 tablets in people with Paroxysmal Nocturnal Hemoglobinuria, run by Haisco Pharmaceutical Group Co., Ltd. at 1 site 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 6 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: looking for participants now, as of the sponsor's last update on 20 Dec 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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?
Contact Li (+8602867258840, lifangq@haisco.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence and severity of adverse events (time frame: through study completion，about 2 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 20 Dec 2024.