Hereditary angioedema
Conditions
Interventions
Dose group 1:Complete intravenous injection of 25 IU/kg within 2 minutes
Dose group 2:Complete intravenous injection of 50 IU/kg within 5 minutes
Dose group 3:Complete intravenous injection of 100 IU/kg within 10 minutes
Sponsors
Peking Union Medical College Hospital, Chinese Academy of Medical Sciences
Eligibility
Sex/Gender
All
Age
18 Years to 65 Years
Inclusion criteria
Inclusion criteria: 1. From 18 years old to 65 years old, regardless of gender; 2. Clinical diagnosis of C1INH deficiency type hereditary angioedema (HAE-I/II type) patients; 3. During the screening period, the C1INH functional level is below 50% of the normal level, the C4 level is below the normal range, and the C1q level is normal (if applicable); 4. The HAE disease status before screening was defined as no disease, defined as the number of acute attacks of HAE <=5 within 3 months before screening; <=1 time within 1 month before screening; No seizures within 1 week before screening; 5. Voluntarily participate in this experiment and sign informed consent.
Exclusion criteria
Exclusion criteria: 1. Individuals who are allergic to the experimental drug or its excipients, or other human derived C1INH, rabbit derived protein, or rabbit derived drugs; 2. Individuals who test positive for anti-C1INH antibodies during the screening period; 3. Identify individuals who have experienced HAE throat edema symptoms within the previous 3 months; 4. Combine other chronic and recurrent angioedema, such as acquired angioedema (AAE-C1INH), HAE with normal C1INH (type III HAE), idiopathic angioedema, mast cell-mediated angioedema, angioedema caused by angiotensin-converting enzyme inhibitors, or recurrent angioedema associated with urticaria; 5. Individuals undergoing short-term or long-term preventive treatment for HAE, including danazol (half-life: approximately 4.5 hours), tranexamic acid (half-life: approximately 2 hours), lanarimumab injection, etc. (half-life: approximately 2 weeks) (excluding those who have stopped preventive drug treatment for more than 5 half lives before screening); 6. Patients who had used any HAE treatment drugs within 7 days before screening, including blood-derived C1INH, recombinant human C1INH, icatiban acetate, etc. (except stopping drug treatment for more than 5 half-lives before screening); 7. Individuals who have received any blood or plasma derived medication (such as fresh or frozen plasma) within the past 7 days prior to screening; 8. Individuals who have used angiotensin-converting enzyme inhibitors, any systemically absorbed estrogen containing drugs (such as oral contraceptives or hormone replacement therapy), or tissue type plasminogen activator within the 4 weeks prior to screening (excluding those who have consistently used these drugs within the 4 weeks prior to the screening of the investigational drug); 9. Use of nonsteroidal anti-inflammatory drugs within the week prior to screening (excluding continuous stable users within the week prior to screening); 10. Screen for drugs such as heparin and other glucosamine glycosaminoglycans that have been used within the first 5 half lives; 11. Individuals with a history of substantial organ or bone marrow transplantation; 12. Active infections that are severe or currently require systemic use of antibiotics or antiviral therapy, including active bacteria, viruses, fungi, mycobacteria, parasites, or other infections (excluding nail bed fungal infections); 13. Patients with concomitant diseases and disorders that may worsen or affect the test evaluation during the study period, including but not limited to: mental diseases, immune and endocrine system diseases beyond the control of drug treatment (including decompensated diabetes and thyroid diseases), blood diseases requiring chemotherapy, malignant tumors that have not been cured or cured for less than five years, decompensated liver diseases, serious autoimmune diseases, serious cerebrovascular diseases, etc; 14. Patients with severe comorbidities, such as cardiovascular disease (including unstable angina, malignant arrhythmia, acute myocardial infarction, heart failure grade 3 or above, hypertension that cannot be controlled by one or more antihypertensive drugs, with poor control criteria: systolic blood pressure >= 160mmHg and/or diastolic blood pressure >= 100mmHg), significant liver and kidney function damage: ALT or AST>2 × ULN, or total bilirubin>1.5 × ULN or serum creatinine>1.5 × ULN, who are deemed unsuitable for inclusion by the researchers; 15. HIV antibody positive, active hepatitis B or C pa
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety and tolerability; | — |
Secondary
| Measure | Time frame |
|---|---|
| Pharmacokinetics;Pharmacodynamics;Immunogenicity; | — |
Countries
China
Contacts
Public ContactZhi Yuxiang
Peking Union Medical College Hospital
Outcome results
None listed