Paroxysmal Nocturnal HemoglobinuriaPhase 3NCT07914894
A Study of Efficacy and Safety of FXS6837 in Anti-C5 Treated PNH Patients.
A Phase 3 study testing FXS6837 Capsule in people with Paroxysmal Nocturnal Hemoglobinuria, run by Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. at 2 sites in China.
Trial summary
This is an single arm, open label designed phase III study to evaluate the efficacy and safety of oral FXS6837 capsules in PNH patients with residual anemia despite the stable treatment with anti-C5 antibody. 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
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 2 sitesChina
- Study dates
- 26 Jan 2027to 10 Aug 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
Inclusion criteria: 1. Male or female subjects aged ≥ 18 years at screening; 2. Body weight ≥ 40 kg and body mass index (BMI) ≥ 18 kg/m2 at screening; 3. Diagnosis of PNH at least 6 months before screening and confirmed by high-sensitivity flow cytometry with a PNH RBC and granulocyte/monocyte population size of ≥10% within 6 months before screening or during screening; 4. Have received a stable treatment regimen (dose and dosing interval) with anti-C5 monoclonal antibody (eculizumab) for at least 6 months prior to the first dose; 5. Have at least one blood transfusion record within 3 months before screening, or Hb level persistently < 100 g/L during stable treatment with anti-C5 monoclonal antibody within 3 months before screening (medical history data/laboratory test reports, at least 2 test records with an interval of ≥ 4 weeks); 6. 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 and all Hb measurements < 105 g/L during the screening period (these two measurements should be 2 to 6 weeks apart; multiple Hb measurements are allowed during the screening period); 7. Vaccinated against Neisseria meningitidis and Streptococcus pneumoniae before the first dose. If patients have not been previously vaccinated or need booster vaccination (according to local vaccination policies), vaccination should be completed at least 2 weeks before the first dose. If the first dose must occur within 2 weeks after vaccination, prophylactic antibiotic treatment should be used until at least 2 weeks after vaccination; 8. Participants of reproductive potential commit to having no plans for pregnancy or sperm/egg donation from screening until 1 month (female participants) or 3 months (male participants) after the last dose and voluntarily take effective physical contraceptive methods (including partners); 9. Have a detailed understanding of the nature, significance, possible benefits, potential inconveniences, and potential risks of the trial, and voluntarily participate in this clinical trial; Able to communicate well with the investigators and comply with the requirements of the entire study; Signed a written informed consent form. Exclusion criteria: 1. 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; 2. 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;
- Hypoxia-inducible factor prolyl hydroxylase inhibitors (HIF-PHI) for at least 8 weeks;
- Androgens for at least 4 weeks; 3. 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; 4. History of bone marrow/hematopoietic stem cell or solid organ transplantation (such as heart, lung, kidney, liver); 5. History of splenectomy or planned surgery during the study period; 6. Subjects with significantly abnormal liver function at screening: any parameter of alanine aminotransferase (ALT), γ-glutamyl transpeptidase (GGT), or alkaline phosphatase (ALP) > 3 × ULN; 7. 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; 8. Concurrent systemic critical 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 hemorrhage, other blood disorders (such as chronic anemia unrelated to PNH), which are deemed unsuitable for study participation by the investigator; 9. Known or suspected by investigators to have immunodeficiency diseases or hereditary complement deficiency; 10. History of Neisseria meningitidis infection; History of ≥ 2 episodes of pneumococcal infection; 11. 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; 12. Suspected or known history of allergies to the IMPs or any ingredient in the IMPs; 13. Received any type of live attenuated vaccine within 4 weeks prior to screening, or plan to receive any live attenuated vaccine during the study; 14. Pregnant or lactating women or those with positive pregnancy test results; 15. Previous history of drug abuse or drug use; 16. Participated in other clinical trials of investigational drugs (or are still within 5 half-lives of the drug, whichever is longer) (except eculizumab) 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; 17. Having diseases or medical conditions that affect drug absorption, distribution, metabolism, and excretion, or may reduce compliance or increase participant risk, such as severe gastrointestinal disease history, major gastrointestinal surgery history, swallowing difficulties, etc., as assessed by the investigator; Participants who may not be able to complete this study for other reasons, or other situations deemed unsuitable for study participation by the investigator.
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
China2 sites
First Affiliated Hospital of Zhejiang University
Hangzhou, Zhejiang
Hongyan Tong, +86 13958122357, hongyantong@aliyun.com
TianJin Medical University General Hospital
Tianjin
Hui Liu, +86 13821113189, liuhui8003@qq.com
About this study
Official title: A Multicenter, Single-Arm, Open-Label Phase III Clinical Study Evaluating the Efficacy and Safety of FXS6837 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Remain Anemic After Previous Stable Use of the C5 Complement Inhibitor
This is an single arm, open label designed phase III study to evaluate the efficacy and safety of oral FXS6837 capsules in PNH patients with residual anemia despite the stable treatment with anti-C5 antibody.
Read the detailed description
About 20 PNH patients who have received eculizumab stably for at least 6 months before first dosing and still have hemoglobin (Hb) < 100 g/L will be eligible to be enrolled. All patients will take FXS6837 capsules for 24 weeks according to protocol to evaluate the efficacy and safety of FXS6837 Capsules in PNH patients who remain anemic after anti-C5 monoclonal antibody therapy. After completing the 24-week treatment, all participants who are assessed by the investigator as potentially continuing to benefit from FXS6837 may voluntarily enter a separate long-term open-label extension stud and continue to receive FXS6837 treatment.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Being tested
- FXS6837 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.
FXS6837 CapsuleDrug
FXS6837 capsule will be taken orally for 24 weeks
| Study group | Role | What they receive |
|---|---|---|
| FXS6837 | Experimental | 100 mg FXS6837 capsule Receives: Drug: FXS6837 Capsule |
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 Week18 and Week24 in the absence of RBC transfusion (defined as no RBC transfusion after Week2 through Week24)
Measured: 24 weeks from Baseline (Week 18 through Week 24)
Secondary outcomes (22)
Proportion of participants with an increase from baseline in Hb level of ≥ 20 g/L in at least 3 of 4 measurements between Week18 and Week24 in the absence of RBC transfusion (defined as no RBC transfusion after Week2 through Week24)
Measured: 24 weeks from Baseline (Baseline, Week 18 through Week 24)
Proportion of participants without RBC transfusion during the treatment period from between Week2 to Week24
Measured: Week2 to Week24
Change from baseline in Hb as mean of visits between Week18 and Week24
Measured: 24 weeks from Baseline (Baseline, Week 18 through Week 24)
Change from baseline in reticulocyte count during the treatment period from Week18 to Week24
Measured: 24 weeks from Baseline (Baseline, Week 18 through Week 24)
Percent change from baseline in lactate dehydrogenase (LDH) levels during the treatment period from Week18 to Week24
Measured: 24 weeks from Baseline(Baseline, Week 18 through Week 24)
Proportion of participants with LDH ≤ 1.5 × upper limit of normal (ULN) in at least 3 of 4 measurements between Week18 and Week24;
Measured: 24 weeks from Baseline (Week 18 through Week 24)
Change from baseline in Functional Assessment of Chronic Illness Therapy - Fatigue Scale (FACIT-Fatigue) score during the treatment period from Week18 to Week24
Measured: 24 weeks from Baseline (Baseline, Week 18 through Week 24)
Incidence rate of BTH between Week1 and Week24
Measured: up to 24 weeks
Incidence of MAVEs between Week1 and Week24
Measured: up to 24 weeks
Safety evaluation: All participants will be observed for any adverse event (AE) during the clinical study.
Measured: up to 24 weeks
Population Pharmacokinetics (PK): Maximum Observed Concentration (Cmax) of FXS6837
Measured: From pre-dose on Day1 to Week24
Population Pharmacokinetics (PK): Plasma trough concentration (Ctrough) of FXS6837
Measured: From pre-dose on Day1 to Week24
Population Pharmacokinetics (PK): Area Under the Concentration-Time Curve (AUC) of FXS6837
Measured: From pre-dose on Day1 to Week24
The exposure-response correlation between Steady-State trough Concentration (Ctrough) of FXS6837 and hemoglobin (Hb) level
Measured: From pre-dose on Day1 to Week24
The exposure-response correlation between Steady-State trough Concentration (Ctrough) of FXS6837 and lactate dehydrogenase (LDH) level
Measured: From pre-dose on Day1 to Week24
The exposure-response correlation between Steady-State trough Concentration (Ctrough) of FXS6837 and Alternative Pathway(AP) activity
Measured: From pre-dose on Day1 to Week24
The exposure-response correlation between Steady-State trough Concentration (Ctrough) of FXS6837 and Fragment Bb of Complement Factor B level
Measured: From pre-dose on Day1 to Week24
The exposure-response correlation between Steady-State trough Concentration (Ctrough) of FXS6837 and soluble C5b-9 (sC5b-9) level
Measured: From pre-dose on Day1 to Week24
The exposure-response correlation between Steady-State trough Concentration (Ctrough) of FXS6837 and PNH granulocyte clone percentage
Measured: From pre-dose on Day1 to Week24
The exposure-response correlation between Steady-State trough Concentration (Ctrough) of FXS6837 and PNH monocyte clone percentage
Measured: From pre-dose on Day1 to Week24
The exposure-response correlation between Steady-State trough Concentration (Ctrough) of FXS6837 and C3d+PNH red cell clone percentage
Measured: From pre-dose on Day1 to Week24
The exposure-response correlation between Steady-State trough Concentration (Ctrough) of FXS6837 and PNH red cell clone percentage
Measured: From pre-dose on Day1 to Week24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Nov 2026
- Study start26 Jan 2027 (estimated)
- Primary completion17 May 2028 (estimated)
- Study completion10 Aug 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Rong Fu, Doctor, Principal investigator, Tianjin Medical University General HospitalHongyan Tong, Principal investigator, First Affiliated Hospital of Zhejiang University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07914894
- Sponsor's protocol ID
- FXS6837-PNH-302
- Status last verified
- Oct 2026
- First submitted
- 13 Oct 2026
- Last updated
- 4 Nov 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 4 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Single-Arm, Open-Label Phase III Clinical Study Evaluating the Efficacy and Safety of FXS6837 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Remain Anemic After Previous Stable Use of the C5 Complement Inhibitor
- ClinicalTrials.gov ID
- NCT07914894
- Acronym / sponsor ID
- FXS6837-PNH-302
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.Industry (pharma, biotech, devices)
- Study start
- 26 Jan 2027 (estimated)
- Primary completion
- 17 May 2028 (estimated)
- Study completion
- 10 Aug 2028 (estimated)
- First posted
- 4 Nov 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 an single arm, open label designed phase III study to evaluate the efficacy and safety of oral FXS6837 capsules in PNH patients with residual anemia despite the stable treatment with anti-C5 antibody.
Detailed description
About 20 PNH patients who have received eculizumab stably for at least 6 months before first dosing and still have hemoglobin (Hb) < 100 g/L will be eligible to be enrolled. All patients will take FXS6837 capsules for 24 weeks according to protocol to evaluate the efficacy and safety of FXS6837 Capsules in PNH patients who remain anemic after anti-C5 monoclonal antibody therapy. After completing the 24-week treatment, all participants who are assessed by the investigator as potentially continuing to benefit from FXS6837 may voluntarily enter a separate long-term open-label extension stud and continue to receive FXS6837 treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| FXS6837 | Experimental | Drug: FXS6837 Capsule100 mg FXS6837 capsule |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
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 will be taken orally for 24 weeks | Drug | FXS6837 |
Primary outcome
Proportion of participants with hemoglobin (Hb) level ≥ 120 g/L in at least 3 of 4 measurements between Week18 and Week24 in the absence of RBC transfusion (defined as no RBC transfusion after Week2 through Week24)
Time frame: 24 weeks from Baseline (Week 18 through Week 24)
22 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 Week18 and Week24 in the absence of RBC transfusion (defined as no RBC transfusion after Week2 through Week24)
Time frame: 24 weeks from Baseline (Baseline, Week 18 through Week 24)
Proportion of participants without RBC transfusion during the treatment period from between Week2 to Week24
Time frame: Week2 to Week24
Change from baseline in Hb as mean of visits between Week18 and Week24
Time frame: 24 weeks from Baseline (Baseline, Week 18 through Week 24)
Change from baseline in reticulocyte count during the treatment period from Week18 to Week24
Time frame: 24 weeks from Baseline (Baseline, Week 18 through Week 24)
Percent change from baseline in lactate dehydrogenase (LDH) levels during the treatment period from Week18 to Week24
Time frame: 24 weeks from Baseline(Baseline, Week 18 through Week 24)
Proportion of participants with LDH ≤ 1.5 × upper limit of normal (ULN) in at least 3 of 4 measurements between Week18 and Week24;
Time frame: 24 weeks from Baseline (Week 18 through Week 24)
Change from baseline in Functional Assessment of Chronic Illness Therapy - Fatigue Scale (FACIT-Fatigue) score during the treatment period from Week18 to Week24
Time frame: 24 weeks from Baseline (Baseline, Week 18 through Week 24)
Incidence rate of BTH between Week1 and Week24
Time frame: up to 24 weeks
Incidence of MAVEs between Week1 and Week24
Time frame: up to 24 weeks
Safety evaluation: All participants will be observed for any adverse event (AE) during the clinical study.
Time frame: up to 24 weeks
Population Pharmacokinetics (PK): Maximum Observed Concentration (Cmax) of FXS6837
Time frame: From pre-dose on Day1 to Week24
Population Pharmacokinetics (PK): Plasma trough concentration (Ctrough) of FXS6837
Time frame: From pre-dose on Day1 to Week24
Population Pharmacokinetics (PK): Area Under the Concentration-Time Curve (AUC) of FXS6837
Time frame: From pre-dose on Day1 to Week24
The exposure-response correlation between Steady-State trough Concentration (Ctrough) of FXS6837 and hemoglobin (Hb) level
Time frame: From pre-dose on Day1 to Week24
The exposure-response correlation between Steady-State trough Concentration (Ctrough) of FXS6837 and lactate dehydrogenase (LDH) level
Time frame: From pre-dose on Day1 to Week24
The exposure-response correlation between Steady-State trough Concentration (Ctrough) of FXS6837 and Alternative Pathway(AP) activity
Time frame: From pre-dose on Day1 to Week24
The exposure-response correlation between Steady-State trough Concentration (Ctrough) of FXS6837 and Fragment Bb of Complement Factor B level
Time frame: From pre-dose on Day1 to Week24
The exposure-response correlation between Steady-State trough Concentration (Ctrough) of FXS6837 and soluble C5b-9 (sC5b-9) level
Time frame: From pre-dose on Day1 to Week24
The exposure-response correlation between Steady-State trough Concentration (Ctrough) of FXS6837 and PNH granulocyte clone percentage
Time frame: From pre-dose on Day1 to Week24
The exposure-response correlation between Steady-State trough Concentration (Ctrough) of FXS6837 and PNH monocyte clone percentage
Time frame: From pre-dose on Day1 to Week24
The exposure-response correlation between Steady-State trough Concentration (Ctrough) of FXS6837 and C3d+PNH red cell clone percentage
Time frame: From pre-dose on Day1 to Week24
The exposure-response correlation between Steady-State trough Concentration (Ctrough) of FXS6837 and PNH red cell clone percentage
Time frame: From pre-dose on Day1 to Week24
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 4 Nov 2026.
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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.
Rong Fu, Doctor principal investigator
Tianjin Medical University General Hospital
Hongyan Tong principal investigator
First Affiliated Hospital of Zhejiang University
2 sites 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 in people with Paroxysmal Nocturnal Hemoglobinuria, run by Shanghai Fosun Pharmaceutical Industrial Development 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?
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 4 Nov 2026. 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 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 Week18 and Week24 in the absence of RBC transfusion (defined as no RBC transfusion after Week2 through Week24) (time frame: 24 weeks from Baseline (Week 18 through Week 24)). 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 4 Nov 2026.