paroxysmal nocturnal hemoglobinuria
Conditions
Interventions
Observation group:None
Sponsors
West China Hospital of Sichuan University
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Inclusion criteria
Inclusion criteria: 1.Age >= 18 years old; 2.High-sensitivity flow cytometry to evaluate PNH clones >= 10%; 3.LDH level >= 1.5 × ULN; 4.At least one of the following signs or symptoms related to PNH: anemia, fatigue, hemoglobinuria, abdominal pain, shortness of breath, history of thrombosis, dysphagia or erectile dysfunction. 5.At least 2 weeks before the start of the treatment, administer the meningococcal and pneumococcal vaccines; 6.If need to use Iptcopan urgently,continue taking antibiotics until 14 days after the vaccination is completed, and complete the vaccination as soon as possible.
Exclusion criteria
Exclusion criteria: 1.Currently receiving or having previously received treatment with complement inhibitor; 2.Having an active bacterial, viral or fungal infection within 14 days prior to the first administration of the medication; 3.Has a history of HIV infection; 4.Pregnant or lactating women who are currently in the pregnancy or lactation period or who plan to become pregnant during the study period; 5.Allergic to Iptcopan or any other excipients.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| hemoglobin; | — |
Secondary
| Measure | Time frame |
|---|---|
| Size of PNH cell clones;LDH;Fatigue score; | — |
Countries
China
Contacts
Public ContactChuan He
West China Hospital of Sichuan University
Outcome results
None listed