Paroxysmal Nocturnal HemoglobinuriaPhase 3NCT07747090
A Study of the Efficacy and Safety of FXS6837 Capsules Compared to Eculizumab for 24 Weeks in Patients With PNH.
A Phase 3 study testing FXS6837 Capsule and Eculizumab infusion in people with Paroxysmal Nocturnal Hemoglobinuria, run by Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. at 1 site in China.
Trial summary
An open label study to evaluate the efficacy and safety of FXS6837 capsules compared to eculizumab in naive PNH patients. About 90 PNH patients who are naive to complement inhibitor therapies will be enrolled to take FXS6837 capsules or eculizumab for 24 weeks 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?
- FXS6837 Capsule and Eculizumab infusion
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 90 participants
- Where
- 1 siteChina
- Study dates
- 10 Dec 2026to 31 Jul 2028
- Phase
- Phase 3
- 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 subjects aged ≥ 18 years at screening;
- Body weight ≥ 40 kg and body mass index (BMI) ≥ 18 kg/m2 at screening;
- Diagnosis of PNH by the investigator according to the PNH diagnostic criteria, with red blood cell and white blood cell (monocytes or neutrophil) clone levels > 10% detected by high sensitivity flow cytometry within 6 months before screening or during the screening period;
- PNH patients with no prior treatment involving any complement inhibitors;
- At least two measurements during the screening period (2 to 6 weeks apart) showing LDH > 1.5 × ULN (multiple measurements are allowed during the screening period);
- Hb meeting one of the following conditions: (1) Hb concentration < 100 g/L at the first screening visit, and received RBC transfusion therapy due to PNH-related anemia during the screening period; (2) Mean Hb concentration from two measurements during the screening period < 100 g/L (these two measurements should be 2 to 6 weeks apart; multiple Hb measurements are allowed during the screening period);
Who cannot join
- With laboratory evidence of bone marrow failure during screening (reticulocyte count < 100 × 109/L, or platelet count < 30 × 109/L, or neutrophil count < 0.5 × 109/L); Received acute treatment (such as platelet transfusion, granulocyte colony-stimulating factor) for thrombocytopenia or neutropenia within 30 days before screening;
- Participants receiving other therapies before screening that have not reached the following stable treatment durations:
- Erythropoietin for at least 8 weeks
- Immunosuppressants for at least 8 weeks, systemic corticosteroids (≤ 15 mg/day) for at least 4 weeks
- Iron, vitamin B12, or folic acid supplementation for at least 4 weeks
- Anticoagulants: Vitamin K antagonists (such as warfarin) used for at least 4 weeks with a stable international normalized ratio (INR) (as determined by the investigator), low molecular weight heparin, oral anticoagulants such as aspirin, rivaroxaban, apixaban, etc., for at least 4 weeks;
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- Hypoxia-inducible factor prolyl hydroxylase inhibitors (HIF-PHI) for at least 8 weeks;
- Androgens for at least 4 weeks;
- History of malignant tumors of any organ or system within 5 years before screening (except local basal cell carcinoma of the skin or carcinoma in situ of the cervix), regardless of whether treatment was received and whether there is evidence of local recurrence or metastasis;
- History of bone marrow/hematopoietic stem cell or solid organ transplantation (such as heart, lung, kidney, liver);
- History of splenectomy or planned surgery during the study period;
- Subjects with significantly abnormal liver function at screening: any parameter of alanine aminotransferase (ALT), γ-glutamyl transpeptidase (GGT), or alkaline phosphatase (ALP) > 3 × ULN;
- Human immunodeficiency virus (HIV) infection (HIV antibody positive), active syphilis infection, hepatitis B virus infection (hepatitis B surface antigen positive), active hepatitis C virus infection, or active tuberculosis infection at screening;
- Subjects with concurrent systemic major diseases, including but not limited to: advanced heart disease (such as New York Heart Association [NYHA] class IV), severe lung disease (such as severe pulmonary hypertension [WHO class IV]), active hepatitis, severe kidney disease (estimated glomerular filtration rate eGFR < 30 mL/min/1.73 m2 or chronic kidney disease [CKD] stage 4 or dialysis patients), unstable thrombosis, active gastrointestinal bleeding, other hematologic diseases (such as chronic anemia unrelated to PNH), and deemed unsuitable for study participation by the investigator;
- Known or suspected by investigators to have immunodeficiency diseases or hereditary complement deficiency;
- History of Neisseria meningitidis infection; History of ≥ 2 episodes of pneumococcal infection;
- Detected or suspected (as assessed by the investigator) systemic active bacterial, viral (including COVID-19), or fungal infections within 2 weeks before the first dose; Axillary temperature > 38 °C within 7 days before the first dose;
- Suspected or known history of allergies to the IPs or any ingredient in the IPs;
- Received any type of live attenuated vaccine within 4 weeks prior to screening, or plan to receive any live attenuated vaccine during the study;
- Pregnant or lactating women or those with positive pregnancy test results;
- Previous history of drug abuse or drug use;
- Participated in other clinical trials of investigational drugs (or are still within 5 half-lives of the drug, whichever is longer) or medical device clinical trials within 30 days prior to screening, plans to participate in other clinical trials during the study, or having residual effects as assessed by the investigator;
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
TianJin Medical University General Hospital
Tianjin, Tianjin Municipality
Hui Liu, +86 13821113189, liuhui8003@qq.com
About this study
Official title: A Phase III, Multicenter, Randomized, Open-Label, Active-Controlled Study of FXS6837 Versus Eculizumab in Complement Inhibitor-Naïve Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
An open label study to evaluate the efficacy and safety of FXS6837 capsules compared to eculizumab in naive PNH patients. About 90 PNH patients who are naive to complement inhibitor therapies will be enrolled to take FXS6837 capsules or eculizumab for 24 weeks according to protocol.
Read the detailed description
A Phase III, randomized, open-label, active-controlled study to evaluate the efficacy and safety of FXS6837 capsules compared with eculizumab in approximately 90 complement inhibitor-naïve patients with paroxysmal nocturnal hemoglobinuria (PNH). Patients will receive FXS6837 capsules or eculizumab for 24 weeks.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Being tested
- FXS6837 Capsule, Eculizumab infusion
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FXS6837 CapsuleDrug
FXS6837 capsule orally taken for 24 weeks
Eculizumab infusionBiological
Eculizumab infusion treated for 24 weeks
| Study group | Role | What they receive |
|---|---|---|
| FXS6837 Capsules | Experimental | FXS6837 capsule orally taken for 24 weeks Receives: Drug: FXS6837 Capsule |
| Eculizumab infusion | Active comparator | Eculizumab infusion treated for 24 weeks Receives: Biological: Eculizumab infusion |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of participants with hemoglobin (Hb) level ≥ 120 g/L in at least 3 of 4 measurements between W18 and W24 in the absence of RBC transfusion (defined as no RBC transfusion after W2 through W24)
Measured: 24 weeks from Baseline
Secondary outcomes (10)
Proportion of participants with an increase from baseline in Hb level of ≥ 20 g/L in at least 3 of 4 measurements between W18 and W24 in the absence of RBC transfusion (defined as no RBC transfusion after Week 2 through Week 24)
Measured: 24 weeks from Baseline
Proportion of subjects without RBC transfusion during the treatment period from Week 2 to Week 24
Measured: 24 weeks from Baseline
Change from baseline in Hb during the treatment period from Week 18 to Week 24
Measured: 24 weeks from Baseline
Change from baseline in reticulocyte count during the treatment period from Week 18 to Week 24
Measured: 24 weeks from Baseline
Percentage change from baseline in lactate dehydrogenase (LDH) during the treatment period from Week 18 to Week 24
Measured: 24 weeks from Baseline
Proportion of participants with LDH ≤ 1.5 × upper limit of normal (ULN) in 3 of 4 measurements between W18 and W24
Measured: 24 weeks from Baseline
Change from baseline in Functional Assessment of Chronic Illness Therapy - Fatigue Scale (FACIT-Fatigue) score during the treatment period from Week 18 to Week 24
The FACIT-Fatigue is a 13-item questionnaire which is used to assess patient self-reported fatigue and its impact on daily life. The score of each of the 13 items ranges from 0-4, so the range of total score is 0-52, with 0 being the worst and 52 being the best.
Measured: 24 weeks from Baseline
Incidence of BTH during treatment
Measured: 24 weeks from Baseline
Incidence of MAVEs during treatment
Measured: 24 weeks from Baseline
Safety evaluation: All participants will be observed for any adverse event (AE) during the clinical study
Measured: Up to 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Aug 2026
- Study start10 Dec 2026 (estimated)
- Primary completion5 May 2028 (estimated)
- Study completion31 Jul 2028 (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 Shanghai Fosun Pharmaceutical Industrial Development 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 90 participants (estimated)
Sponsor and investigators
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Rong Fu, Principal investigator, Tianjin Medical University General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07747090
- Sponsor's protocol ID
- FXS6837-PNH-301
- Status last verified
- Jul 2026
- First submitted
- 22 Jul 2026
- Last updated
- 5 Aug 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 5 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase III, Multicenter, Randomized, Open-Label, Active-Controlled Study of FXS6837 Versus Eculizumab in Complement Inhibitor-Naïve Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
- ClinicalTrials.gov ID
- NCT07747090
- Acronym / sponsor ID
- FXS6837-PNH-301
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 90 participants (estimated)
- Lead sponsor
- Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.Industry (pharma, biotech, devices)
- Study start
- 10 Dec 2026 (estimated)
- Primary completion
- 5 May 2028 (estimated)
- Study completion
- 31 Jul 2028 (estimated)
- First posted
- 5 Aug 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.
An open label study to evaluate the efficacy and safety of FXS6837 capsules compared to eculizumab in naive PNH patients. About 90 PNH patients who are naive to complement inhibitor therapies will be enrolled to take FXS6837 capsules or eculizumab for 24 weeks according to protocol.
Detailed description
A Phase III, randomized, open-label, active-controlled study to evaluate the efficacy and safety of FXS6837 capsules compared with eculizumab in approximately 90 complement inhibitor-naïve patients with paroxysmal nocturnal hemoglobinuria (PNH). Patients will receive FXS6837 capsules or eculizumab for 24 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| FXS6837 Capsules | Experimental | Drug: FXS6837 CapsuleFXS6837 capsule orally taken for 24 weeks |
| Eculizumab infusion | Active comparator | Biological: Eculizumab infusionEculizumab infusion treated for 24 weeks |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Male or female subjects aged ≥ 18 years at screening;
- Body weight ≥ 40 kg and body mass index (BMI) ≥ 18 kg/m2 at screening;
- Diagnosis of PNH by the investigator according to the PNH diagnostic criteria, with red blood cell and white blood cell (monocytes or neutrophil) clone levels > 10% detected by high sensitivity flow cytometry within 6 months before screening or during the screening period;
- PNH patients with no prior treatment involving any complement inhibitors;
- At least two measurements during the screening period (2 to 6 weeks apart) showing LDH > 1.5 × ULN (multiple measurements are allowed during the screening period);
Key exclusion criteria (22 listed)
- With laboratory evidence of bone marrow failure during screening (reticulocyte count < 100 × 109/L, or platelet count < 30 × 109/L, or neutrophil count < 0.5 × 109/L); Received acute treatment (such as platelet transfusion, granulocyte colony-stimulating factor) for thrombocytopenia or neutropenia within 30 days before screening;
- Participants receiving other therapies before screening that have not reached the following stable treatment durations:
- History of malignant tumors of any organ or system within 5 years before screening (except local basal cell carcinoma of the skin or carcinoma in situ of the cervix), regardless of whether treatment was received and whether there is evidence of local recurrence or metastasis;
- History of bone marrow/hematopoietic stem cell or solid organ transplantation (such as heart, lung, kidney, liver);
- History of splenectomy or planned surgery during the study period;
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 |
|---|---|---|
| FXS6837 CapsuleFXS6837 capsule orally taken for 24 weeks | Drug | FXS6837 Capsules |
| Eculizumab infusionEculizumab infusion treated for 24 weeks | Biological | Eculizumab infusion |
Primary outcome
Proportion of participants with hemoglobin (Hb) level ≥ 120 g/L in at least 3 of 4 measurements between W18 and W24 in the absence of RBC transfusion (defined as no RBC transfusion after W2 through W24)
Time frame: 24 weeks from Baseline
10 secondary outcomes
Proportion of participants with an increase from baseline in Hb level of ≥ 20 g/L in at least 3 of 4 measurements between W18 and W24 in the absence of RBC transfusion (defined as no RBC transfusion after Week 2 through Week 24)
Time frame: 24 weeks from Baseline
Proportion of subjects without RBC transfusion during the treatment period from Week 2 to Week 24
Time frame: 24 weeks from Baseline
Change from baseline in Hb during the treatment period from Week 18 to Week 24
Time frame: 24 weeks from Baseline
Change from baseline in reticulocyte count during the treatment period from Week 18 to Week 24
Time frame: 24 weeks from Baseline
Percentage change from baseline in lactate dehydrogenase (LDH) during the treatment period from Week 18 to Week 24
Time frame: 24 weeks from Baseline
Proportion of participants with LDH ≤ 1.5 × upper limit of normal (ULN) in 3 of 4 measurements between W18 and W24
Time frame: 24 weeks from Baseline
Change from baseline in Functional Assessment of Chronic Illness Therapy - Fatigue Scale (FACIT-Fatigue) score during the treatment period from Week 18 to Week 24
Time frame: 24 weeks from Baseline
Incidence of BTH during treatment
Time frame: 24 weeks from Baseline
Incidence of MAVEs during treatment
Time frame: 24 weeks from Baseline
Safety evaluation: All participants will be observed for any adverse event (AE) during the clinical study
Time frame: Up to 24 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 5 Aug 2026.
Related trials for Paroxysmal Nocturnal Hemoglobinuria
Enrolling by invitationObservational study
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Hemoglobinuria, Paroxysmal
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)
RecruitingPhase 3
A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works
Paroxysmal Nocturnal Hemoglobinuria
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
Severe Aplastic Anemia (SAA), Hypo-Plastic Myelodysplastic Syndrome (MDS)
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 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 Phase 3 study testing FXS6837 Capsule and Eculizumab infusion 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 6 inclusion and 22 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: opening soon, as of the sponsor's last update on 5 Aug 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.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
Proportion of participants with hemoglobin (Hb) level ≥ 120 g/L in at least 3 of 4 measurements between W18 and W24 in the absence of RBC transfusion (defined as no RBC transfusion after W2 through W24) (time frame: 24 weeks from Baseline). 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 5 Aug 2026; AzTrials last refreshed its copy on 8 Nov 2026.