Efficacy and Safety of Lanoracopan in the Treatment of PNH

NCT07788105 | Not Yet Recruiting

• 1 other identifier: K11574
• Study Type: observational
• Target: 90
• Locations: 0 countries
• Sites: N/A

Brief Summary

This study aimed to evaluate the efficacy and safety of Lanoracopan Hydrochloride in the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH)

Conditions

Paroxysmal Nocturnal Hemoglobinuria, PNH

Outcome Measures

Primary Outcomes (1)

• rate of adverse events (AEs)

  According to CTCAE V5.0

  During treatment, an average of 24 weeks

Secondary Outcomes (4)

• Proportion of subjects maintaining Hb ≥120 g/L

  12 weeks, 24 weeks

• changes in hemoglobin (Hb) levels

  12 weeks, 24 weeks

• incidence of clinically significant hemolysis

  12 weeks, 24 weeks

• proportion of subjects experiencing a major adverse vascular event (MAVE)

  12 weeks, 24 weeks

Interventions

Lanoracopan Hydrochloride DRUG

400mg bid

Also known as: MY008211A

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

Patients who visited Peking Union Medical College Hospital

You may qualify if:

• 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;
• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Peking Union Medical College Hospital: lead

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases

Central Study Contacts

Bing Han

CONTACT

+86 13601059938; hanbing_li@sina.com.cn

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: August 23, 2026
• First Posted: August 26, 2026
• Study Start (Estimated): September 1, 2026
• Primary Completion (Estimated): December 1, 2028
• Study Completion (Estimated): December 1, 2029
• Last Updated: August 26, 2026

Record last verified: 2026-08

Data Sharing

• IPD Sharing: Will not share