Paroxysmal Nocturnal HemoglobinuriaXH-S003-II-102NCT07657052
A Multicenter, Open-label Study Evaluating the Long-term Safety and Efficacy of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria
A study testing XH-S003 Capsule in people with Paroxysmal Nocturnal Hemoglobinuria, run by Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. at 1 site in China.
Trial summary
This is a multicenter, open-label study to evaluate the long-term safety and efficacy of XH-S003 capsules in PNH patients. About 26 PNH patients who had received and completed XH-S003 study treatment will be enrolled to take XH-S003 capsules orally according to protocol. 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?
- XH-S003 Capsule
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 26 participants
- Where
- 1 siteChina
- Study dates
- 15 Aug 2025to 30 Sep 2029
- Phase
- Interventional
- 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
- PNH patients who have previously received and completed the XH-S003 study treatment, and according to the investigator's evaluation, the benefits of treatment outweigh risks and may benefit from continued treatment with XH-S003;
- Subjects who have been vaccinated against Neisseria meningitidis and Streptococcus pneumoniae as required by previous studies (if the vaccine protection period does not cover the duration of this study, timely booster vaccinations should be administered according to vaccination guidelines and local vaccination requirements);
Who cannot join
- . History of malignancy in any organ or system within the past 5 years prior to screening (excluding localized basal cell carcinoma of the skin or in situ cervical cancer);
- History of bone marrow/hematopoietic stem cell or solid organ transplantation;
- History of splenectomy;
- History of serious comorbidities , including but not limited to: advanced stage heart disease (e.g., New York Heart Association [NYHA] Class IV), severe lung disease (e.g., severe pulmonary hypertension [WHO Class IV]), severe liver function injury, severe renal disease (estimated glomerular filtration rate [eGFR] < 30 mL/min/1.73 m2 or chronic kidney disease [CKD] Stage 4 or dialysis patients), unstable thrombus, other hematologic system diseases (e.g., chronic anemia unrelated to PNH), and deemed unsuitable for study participation by the investigator;
- Pregnant or lactating women, or those with a positive pregnancy test;
- Known or suspected immunodeficiency diseases or hereditary complement deficiency at screening;
- A history of Neisseria meningitidis infection
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
About this study
Official title: A Multicenter, Open-label Study Evaluating the Long-term Safety and Efficacy of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
This is a multicenter, open-label study to evaluate the long-term safety and efficacy of XH-S003 capsules in PNH patients. About 26 PNH patients who had received and completed XH-S003 study treatment will be enrolled to take XH-S003 capsules orally according to protocol. Eligible subjects can maintain XH-S003 treatment until the end of the study.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Being tested
- XH-S003 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.
XH-S003 CapsuleDrug
Optimized dose
| Study group | Role | What they receive |
|---|---|---|
| XH-S003 Capsule | Experimental | XH-S003 capsule orally taken according to protocol until the end of treatment Receives: Drug: XH-S003 Capsule |
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，an average of 1 year
Secondary outcomes (5)
Proportion of subjects with Hb ≥120g/L compared with baseline (without RBC transfusion)
Measured: through study completion，an average of 1 year
Change From Baseline in Hemoglobin in the absence of red blood cell transfusions
Measured: through study completion，an average of 1 year
Proportion of participants who remain free from transfusions
Measured: through study completion，an average of 1 year
Proportion of participants with breakthrough hemolysis (BTH)
Measured: through study completion，an average of 1 year
Proportion of participants with Major Adverse Vascular Events (MAVEs)
Measured: through study completion，an average of 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jun 2026
- Study start15 Aug 2025
- Primary completion30 Sep 2029 (estimated)
- Study completion30 Sep 2029 (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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 26 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Rong Fu, Doctor, Principal investigator, Tianjin Medical University General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07657052
- Sponsor's protocol ID
- XH-S003-II-102
- Acronym
- XH-S003-II-102
- Status last verified
- Jun 2026
- First submitted
- 29 May 2026
- Last updated
- 25 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Open-label Study Evaluating the Long-term Safety and Efficacy of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
- ClinicalTrials.gov ID
- NCT07657052
- Acronym / sponsor ID
- XH-S003-II-102 / XH-S003-II-102
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Study type
- Interventional
- Recruitment status
- Enrolling by invitation
- Enrolment
- 26 participants (estimated)
- Lead sponsor
- Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.Industry (pharma, biotech, devices)
- Study start
- 15 Aug 2025 (actual)
- Primary completion
- 30 Sep 2029 (estimated)
- Study completion
- 30 Sep 2029 (estimated)
- First posted
- 18 Jun 2026 (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 and efficacy of XH-S003 capsules in PNH patients. About 26 PNH patients who had received and completed XH-S003 study treatment will be enrolled to take XH-S003 capsules orally according to protocol. Eligible subjects can maintain XH-S003 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 |
|---|---|---|
| XH-S003 Capsule | Experimental | Drug: XH-S003 CapsuleXH-S003 capsule orally taken according to protocol until the end of treatment |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- PNH patients who have previously received and completed the XH-S003 study treatment, and according to the investigator's evaluation, the benefits of treatment outweigh risks and may benefit from continued treatment with XH-S003;
- Subjects who have been vaccinated against Neisseria meningitidis and Streptococcus pneumoniae as required by previous studies (if the vaccine protection period does not cover the duration of this study, timely booster vaccinations should be administered according to vaccination guidelines and local vaccination requirements);
Key exclusion criteria (7 listed)
- . History of malignancy in any organ or system within the past 5 years prior to screening (excluding localized basal cell carcinoma of the skin or in situ cervical cancer);
- History of bone marrow/hematopoietic stem cell or solid organ transplantation;
- History of splenectomy;
- History of serious comorbidities , including but not limited to: advanced stage heart disease (e.g., New York Heart Association [NYHA] Class IV), severe lung disease (e.g., severe pulmonary hypertension [WHO Class IV]), severe liver function injury, severe renal disease (estimated glomerular filtration rate [eGFR] < 30 mL/min/1.73 m2 or chronic kidney disease [CKD] Stage 4 or dialysis patients), unstable thrombus, other hematologic system diseases (e.g., chronic anemia unrelated to PNH), and deemed unsuitable for study participation by the investigator;
- Pregnant or lactating women, or those with a positive pregnancy test;
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 CapsuleOptimized dose | Drug | XH-S003 Capsule |
Primary outcome
Incidence and severity of adverse events
Time frame: through study completion，an average of 1 year
5 secondary outcomes
Proportion of subjects with Hb ≥120g/L compared with baseline (without RBC transfusion)
Time frame: through study completion，an average of 1 year
Change From Baseline in Hemoglobin in the absence of red blood cell transfusions
Time frame: through study completion，an average of 1 year
Proportion of participants who remain free from transfusions
Time frame: through study completion，an average of 1 year
Proportion of participants with breakthrough hemolysis (BTH)
Time frame: through study completion，an average of 1 year
Proportion of participants with Major Adverse Vascular Events (MAVEs)
Time frame: through study completion，an average of 1 year
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 25 Sep 2026.
Related trials for Paroxysmal Nocturnal Hemoglobinuria
Active, not recruitingPhase 3
A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors
Paroxysmal Nocturnal Hemoglobinuria
Active, not recruitingPhase 3
A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors
Paroxysmal Nocturnal Hemoglobinuria
RecruitingObservational study
Enhancing the Clinical Management of CMF Through a National Pediatric and Adult Network Based on Biological and Genetic Data
Inherited Bone Marrow Failure Syndrome, Fanconi Anemia
RecruitingPhase 2
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)
Enrolling by invitationObservational study
Molecular and Clinical Analysis of Bone Marrow Failure: A Secondary Research Study
Bone Marrow Failure Disorders, VEXAS Syndrome
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.
Rong Fu, Doctor principal investigator
Tianjin Medical University General Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing XH-S003 Capsule in people with Paroxysmal Nocturnal Hemoglobinuria, run by Shanghai Fosun Pharmaceutical Industrial Development 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 7 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: enrolling by invitation, as of the sponsor's last update on 25 Sep 2026. 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.
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: through study completion，an average of 1 year). 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 25 Sep 2026.