PNH - Paroxysmal Nocturnal HemoglobinuriaPhase 2XH-S003-II-101NCT06978699
A Phase II Study Evaluating the Efficacy and Safety of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
A Phase 2 study testing XH-S003 Capsule in people with PNH - Paroxysmal Nocturnal Hemoglobinuria, run by S-INFINITY Pharmaceuticals Co., Ltd at 1 site in China.
Trial summary
This is a multicenter, randomized, single-blind Phase II trial to evaluate the efficacy and safety of XH-S003 capsules in PNH patients. About 24 PNH patients will be enrolled and randomized to three dose levels and take XH-S003 capsules orally Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- PNH - Paroxysmal Nocturnal Hemoglobinuria
- What is being tested?
- XH-S003 Capsule
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 1 siteChina
- Study dates
- 30 Apr 2025to 30 Oct 2026
- 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
- Male or female with aged ≥18 years old;
- Weight ≥40 kg and BMI≥18 kg/m2 ;
- Diagnosed with PNH: with red blood cell or granulocyte clone levels >10% detected by flow cytopy within 6 months prior to screening or during screening;
- Patients who have not previously received any complement inhibitor therayp;
- LDH > 1.5×ULN detected two times during the screening period (interval of 2 to 8 weeks);
- Hb meets one of the following conditions: (1) Hb <100 g/L at the first screening visit, and subjects receive RBC transfusion because of PNH-related anemia during the screening period; (2) The average Hb of two tests during the screening period <100 g/L (interval of 2~8 weeks);
- Vaccination against Neisseria meningitidis and Streptococcus pneumoniae before the first administration. If the subject has not been vaccinated previously or requires booster vaccination (according to local vaccination policies), vaccination must be administered at least 2 weeks before the first administration. If the first administration must begin less than 2 weeks after vaccination, preventive antibiotic treatment must begin at least 2 weeks after vaccination;
Who cannot join
- Subjects with laboratory evidence of bone marrow failure during the screening period (reticulocyte count <100×109/L, platelet count <30×109/L, or neutrophil count <0.5×109/L);
- Subjects receiving other therapies prior to screening who have not achieved the following treatment durations:
- Erythropoietin or immunosuppressants for at least 8 weeks; • Systemic corticosteroids for at least 4 weeks; • Iron supplements, vitamin B12, or folic acid for at least 4 weeks; • Anticoagulants: Vitamin K antagonists for at least 4 weeks with stable international normalized ratio (INR) (as determined by the investigator), low molecular weight heparin for at least 4 weeks; • Hypoxic-inducing factor prolyl hydroxylase inhibitors (HIF-PHI) for at least 8 weeks; • Androgens for at least 4 weeks;
- A history of bone marrow/hematopoietic stem cell or solid organ transplantation;
- Alanine aminotransferase (ALT), γ-glutamyl transpeptidase (GGT), or alkaline phosphatase (ALP) >3×ULN at screening; - Positive HIV antibody, active syphilis infection, positive HBsAg, active HCV infection, or active tuberculosis infection at screening;
- Known or suspected immunodeficiency diseases or hereditary complement deficiency at screening;
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- A history of Neisseria meningitidis infection;
- Subjects with chronic active or recurrent infections within 1 year prior to screening;
- Subjects with systemic active bacterial, viral (including COVID-19), or fungal infections within 2 weeks prior to the first administration; subjects with body temperature >38°C within 7 days prior to the first administration;
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
TianJin Medical University General Hospital
Tianjin
Hui Liu, +86-13821113189, liuhui8003@qq.com
Rong Fu
About this study
Official title: A Multicenter, Randomized, Single-blind Phase II Study Evaluating the Efficacy and Safety of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
This is a multicenter, randomized, single-blind Phase II trial to evaluate the efficacy and safety of XH-S003 capsules in PNH patients. About 24 PNH patients will be enrolled and randomized to three dose levels and take XH-S003 capsules orally
- Being tested
- XH-S003 Capsule
- Keywords
- Paroxysmal Nocturnal Hemoglobinuria
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
XH-S003 CapsuleDrug
25mg \& 100mg
| Study group | Role | What they receive |
|---|---|---|
| Dose 1 | Experimental | Once daily Receives: Drug: XH-S003 Capsule |
| Dose 2 | Experimental | Once daily Receives: Drug: XH-S003 Capsule |
| Dose 3 | Experimental | Once daily Receives: Drug: XH-S003 Capsule |
What is being measured
The primary outcome is the main question the study is designed to answer.
Changes of hemoglobin compared with baseline
Changes of hemoglobin compared with baseline
Measured: 8 weeks
Secondary outcomes (5)
Proportion of subjects with an increase in Hb ≥20g/L compared with baseline (without RBC transfusion);
Proportion of subjects with an increase in Hb ≥20g/L compared with baseline (without RBC transfusion);
Measured: 8 weeks
Changes in Indirect Bilirubin compared to baseline;
Changes in Indirect Bilirubin compared to baseline;
Measured: 8 weeks
Changes in reticulocyte counts compared to baseline;
Changes in reticulocyte counts compared to baseline;
Measured: 8 weeks
Proportion of subjects without RBC transfusion
Proportion of subjects without RBC transfusion
Measured: 8 weeks
Changes in LDH compared with baseline
Changes in LDH compared with baseline
Measured: 8 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 May 2025
- Study start30 Apr 2025
- Primary completion30 Aug 2025 (estimated)
- Study completion30 Oct 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PNH - 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
- Single blind
- Who is masked
- Participant
- Enrolment
- 24 participants (estimated)
Sponsor and investigators
- Lead sponsor
- S-INFINITY Pharmaceuticals Co., Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06978699
- Sponsor's protocol ID
- XH-S003-II-101
- Acronym
- XH-S003-II-101
- Status last verified
- May 2025
- First submitted
- 8 May 2025
- Last updated
- 18 May 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Randomized, Single-blind Phase II Study Evaluating the Efficacy and Safety of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
- ClinicalTrials.gov ID
- NCT06978699
- Acronym / sponsor ID
- XH-S003-II-101 / XH-S003-II-101
- Keywords
- Paroxysmal Nocturnal Hemoglobinuria
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 24 participants (estimated)
- Lead sponsor
- S-INFINITY Pharmaceuticals Co., LtdIndustry (pharma, biotech, devices)
- Study start
- 30 Apr 2025 (actual)
- Primary completion
- 30 Aug 2025 (estimated)
- Study completion
- 30 Oct 2026 (estimated)
- First posted
- 18 May 2025 (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, single-blind Phase II trial to evaluate the efficacy and safety of XH-S003 capsules in PNH patients. About 24 PNH patients will be enrolled and randomized to three dose levels and take XH-S003 capsules orally
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (participant)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Dose 1 | Experimental | Drug: XH-S003 CapsuleOnce daily |
| Dose 2 | Experimental | Drug: XH-S003 CapsuleOnce daily |
| Dose 3 | Experimental | Drug: XH-S003 CapsuleOnce daily |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Male or female with aged ≥18 years old;
- Weight ≥40 kg and BMI≥18 kg/m2 ;
- Diagnosed with PNH: with red blood cell or granulocyte clone levels >10% detected by flow cytopy within 6 months prior to screening or during screening;
- Patients who have not previously received any complement inhibitor therayp;
- LDH > 1.5×ULN detected two times during the screening period (interval of 2 to 8 weeks);
Key exclusion criteria (9 listed)
- Subjects with laboratory evidence of bone marrow failure during the screening period (reticulocyte count <100×109/L, platelet count <30×109/L, or neutrophil count <0.5×109/L);
- Subjects receiving other therapies prior to screening who have not achieved the following treatment durations:
- A history of bone marrow/hematopoietic stem cell or solid organ transplantation;
- Alanine aminotransferase (ALT), γ-glutamyl transpeptidase (GGT), or alkaline phosphatase (ALP) >3×ULN at screening; - Positive HIV antibody, active syphilis infection, positive HBsAg, active HCV infection, or active tuberculosis infection at screening;
- Known or suspected immunodeficiency diseases or hereditary complement deficiency at screening;
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 |
|---|---|---|
| XH-S003 Capsule25mg \& 100mg | Drug | Dose 1, Dose 2, Dose 3 |
Primary outcome
Changes of hemoglobin compared with baseline
Time frame: 8 weeks
Changes of hemoglobin compared with baseline
5 secondary outcomes
Proportion of subjects with an increase in Hb ≥20g/L compared with baseline (without RBC transfusion);
Time frame: 8 weeks
Changes in Indirect Bilirubin compared to baseline;
Time frame: 8 weeks
Changes in reticulocyte counts compared to baseline;
Time frame: 8 weeks
Proportion of subjects without RBC transfusion
Time frame: 8 weeks
Changes in LDH compared with baseline
Time frame: 8 weeks
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 18 May 2025.
- Individual participant dataSharing planned: No
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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 XH-S003 Capsule in people with PNH - Paroxysmal Nocturnal Hemoglobinuria, run by S-INFINITY Pharmaceuticals 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 7 inclusion and 9 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 18 May 2025. 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 Rong Fu (+86-13920350233, furong8369@tmu.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Changes of hemoglobin compared with baseline (time frame: 8 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 18 May 2025.