Paroxysmal Nocturnal Hemoglobinuria, PNHNCT07788105
Efficacy and Safety of Lanoracopan in the Treatment of PNH
An observational study of Paroxysmal Nocturnal Hemoglobinuria, PNH, run by Peking Union Medical College Hospital.
Trial summary
This study aimed to evaluate the efficacy and safety of Lanoracopan Hydrochloride in the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria, PNH
- What is being tested?
- Lanoracopan Hydrochloride
- How many people
- 90 participants
- Where
- No sites listed yet
- Study dates
- Sep 2026to Dec 2029
- Phase
- Observational
- 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 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Patients who visited Peking Union Medical College Hospital
Who may be able to join
- Age 18-75 years.
- Diagnosis of paroxysmal nocturnal hemoglobinuria (PNH) meeting any of the following conditions:
- Newly diagnosed PNH with active hemolysis (hemoglobin [Hb] <100 g/L, lactate dehydrogenase [LDH] >1.5×upper limit of normal [ULN]);
- Receiving a stable regimen of complement C5 monoclonal antibody (eculizumab) at standard dose and interval for ≥3 months, with Hb still <120 g/L despite treatment;
- Intolerant to eculizumab therapy;
- Receiving standard iptacopan therapy for ≥3 months, with Hb still <120 g/L;
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- Intolerant to iptacopan therapy.
- Currently receiving standard lanoracopan therapy (patients who have completed lanoracopan clinical trials).
- Vaccination against meningococcal infection (quadrivalent conjugate vaccine, MenACWY) is required prior to the first dose of study drug (Day 1). If not vaccinated within 3 years prior to Day 1, vaccination must be administered at least 14 days before Day 1; if administered within 14 days before Day 1, antibiotic prophylaxis against meningococcal infection is required until 14 days post-vaccination.
- Vaccination against pneumococcal infection is required prior to Day 1. If not vaccinated within 5 years prior to Day 1, vaccination must be administered at least 14 days before Day 1; if administered within 14 days before Day 1, antibiotic prophylaxis against pneumococcal infection is required until 14 days post-vaccination.
- Willing and able to provide written informed consent and comply with study procedures.
Who cannot join
- Previous bone marrow or hematopoietic stem cell transplantation.
- Previous splenectomy.
- Known or suspected hereditary complement deficiency.
- History of recurrent invasive infections caused by encapsulated organisms, e.g. meningococcus or pneumococcus.
- A history of malignancy within 5 years before screening, except cured local basal cell carcinoma of the skin and carcinoma in situ of the cervix.
- Severe concurrent illness, including severe renal disease (e.g., dialysis), advanced cardiac disease (NYHA class IV), severe pulmonary hypertension (WHO class IV), or unstable thrombotic events, judged unsuitable for participation by the investigator.
- Any other condition that, in the investigator's opinion, may interfere with study conduct, increase subject risk, or preclude safe participation and completion, including concomitant disease, treatment, procedure, surgery, or clinically significant laboratory abnormality.
- Pregnant or breastfeeding women.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Efficacy and Safety of Lanoracopan Hydrochloride in the Treatment of Paroxysmal Nocturnal Hemoglobinuria: A Prospective, Single-Center Real-World Study
This study aimed to evaluate the efficacy and safety of Lanoracopan Hydrochloride in the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH)
Read the detailed description
In Chinese multicenter phase 3 trials, lanoracopan 400 mg BID was evaluated over 24 weeks. In the active-controlled study (N=66, complement-naïve), the proportion achieving Hb ≥120 g/L was 50.0% (lanoracopan) versus 9.09% (eculizumab). In the single-arm study (N=20, C5 inhibitor-inadequate responders), 70% reached Hb ≥120 g/L, and 100% had Hb increase ≥20 g/L from baseline. No treatment-discontinuation due to AEs, no severe breakthrough hemolysis, no major vascular events, no encapsulated bacterial infections, and no deaths were reported across both studies. These data demonstrate superior hemoglobin correction and favorable short-term safety. However, long-term real-world effectiveness and safety profiles stratified by prior treatment (C5 inhibitors vs. other factor B inhibitors) remain uncharacterized. This observational study will enroll a diverse cohort to capture durability of Hb response, transfusion independence rate, incidence of breakthrough hemolysis and thrombosis, and adverse event patterns over extended follow-up, providing evidence to guide therapy switching and sequencing in clinical practice.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria, PNH
- Being tested
- Lanoracopan Hydrochloride
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Lanoracopan HydrochlorideDrug
Also called MY008211A
400mg bid
What is being measured
The primary outcome is the main question the study is designed to answer.
rate of adverse events (AEs)
According to CTCAE V5.0
Measured: During treatment, an average of 24 weeks
Secondary outcomes (4)
Proportion of subjects maintaining Hb ≥120 g/L
During the treatment observation period, assess the proportion of subjects maintaining Hb ≥120 g/L every 12 weeks without red blood cell transfusion
Measured: 12 weeks, 24 weeks
changes in hemoglobin (Hb) levels
During the treatment observation period, evaluate changes in Hb levels every 12 weeks without red blood cell transfusion.
Measured: 12 weeks, 24 weeks
incidence of clinically significant hemolysis
During the treatment observation period, assess the incidence of clinically significant hemolysis every 12 weeks.
Measured: 12 weeks, 24 weeks
proportion of subjects experiencing a major adverse vascular event (MAVE)
During the treatment observation period, assess the proportion of subjects experiencing a MAVE every 12 weeks.
Measured: 12 weeks, 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Aug 2026
- Study startSep 2026 (estimated)
- Primary completionDec 2028 (estimated)
- Study completionDec 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 Hemoglobinuria, PNHStudies of the same condition, open and under way.
- More trials run by Peking Union Medical College HospitalThe 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 90 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking Union Medical College Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Bing Han, Peking Union Medical College Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07788105
- Sponsor's protocol ID
- K11574
- Status last verified
- Aug 2026
- First submitted
- 23 Aug 2026
- Last updated
- 26 Aug 2026
- 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 26 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Lanoracopan Hydrochloride in the Treatment of Paroxysmal Nocturnal Hemoglobinuria: A Prospective, Single-Center Real-World Study
- ClinicalTrials.gov ID
- NCT07788105
- Acronym / sponsor ID
- K11574
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria, PNH
- Study type
- Observational
- Recruitment status
- Not yet recruiting
- Enrolment
- 90 participants (estimated)
- Lead sponsor
- Peking Union Medical College HospitalUniversity, hospital or other
- Study start
- Sep 2026 (estimated)
- Primary completion
- Dec 2028 (estimated)
- Study completion
- Dec 2029 (estimated)
- First posted
- 26 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.
This study aimed to evaluate the efficacy and safety of Lanoracopan Hydrochloride in the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH)
Detailed description
In Chinese multicenter phase 3 trials, lanoracopan 400 mg BID was evaluated over 24 weeks. In the active-controlled study (N=66, complement-naïve), the proportion achieving Hb ≥120 g/L was 50.0% (lanoracopan) versus 9.09% (eculizumab). In the single-arm study (N=20, C5 inhibitor-inadequate responders), 70% reached Hb ≥120 g/L, and 100% had Hb increase ≥20 g/L from baseline. No treatment-discontinuation due to AEs, no severe breakthrough hemolysis, no major vascular events, no encapsulated bacterial infections, and no deaths were reported across both studies. These data demonstrate superior hemoglobin correction and favorable short-term safety. However, long-term real-world effectiveness and safety profiles stratified by prior treatment (C5 inhibitors vs. other factor B inhibitors) remain uncharacterized. This observational study will enroll a diverse cohort to capture durability of Hb response, transfusion independence rate, incidence of breakthrough hemolysis and thrombosis, and adverse event patterns over extended follow-up, providing evidence to guide therapy switching and sequencing in clinical practice.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients who visited Peking Union Medical College Hospital
Key inclusion criteria (11 listed)
- Age 18-75 years.
- Diagnosis of paroxysmal nocturnal hemoglobinuria (PNH) meeting any of the following conditions:
- Vaccination against meningococcal infection (quadrivalent conjugate vaccine, MenACWY) is required prior to the first dose of study drug (Day 1). If not vaccinated within 3 years prior to Day 1, vaccination must be administered at least 14 days before Day 1; if administered within 14 days before Day 1, antibiotic prophylaxis against meningococcal infection is required until 14 days post-vaccination.
- Vaccination against pneumococcal infection is required prior to Day 1. If not vaccinated within 5 years prior to Day 1, vaccination must be administered at least 14 days before Day 1; if administered within 14 days before Day 1, antibiotic prophylaxis against pneumococcal infection is required until 14 days post-vaccination.
- Willing and able to provide written informed consent and comply with study procedures.
Key exclusion criteria (8 listed)
- Previous bone marrow or hematopoietic stem cell transplantation.
- Previous splenectomy.
- Known or suspected hereditary complement deficiency.
- History of recurrent invasive infections caused by encapsulated organisms, e.g. meningococcus or pneumococcus.
- A history of malignancy within 5 years before screening, except cured local basal cell carcinoma of the skin and carcinoma in situ of the cervix.
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 |
|---|---|---|
| Lanoracopan HydrochlorideAlso: MY008211A400mg bid | Drug | – |
Primary outcome
rate of adverse events (AEs)
Time frame: During treatment, an average of 24 weeks
According to CTCAE V5.0
4 secondary outcomes
Proportion of subjects maintaining Hb ≥120 g/L
Time frame: 12 weeks, 24 weeks
changes in hemoglobin (Hb) levels
Time frame: 12 weeks, 24 weeks
incidence of clinically significant hemolysis
Time frame: 12 weeks, 24 weeks
proportion of subjects experiencing a major adverse vascular event (MAVE)
Time frame: 12 weeks, 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 26 Aug 2026.
- 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.
Questions about this trial
What is this clinical trial studying?
An observational study of Paroxysmal Nocturnal Hemoglobinuria, PNH, run by Peking Union Medical College Hospital.
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 11 inclusion and 8 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 26 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
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?
Contact Bing Han (+86 13601059938, hanbing_li@sina.com.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
rate of adverse events (AEs) (time frame: During treatment, an average of 24 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 26 Aug 2026.