Pharmacokinetics and safety of IV Injection of OCTA-C1-INH in hereditary angioedema

Phase 2

Completed

• Conditions: Hereditary angioedema (HAE) type I or type II; Not Applicable

• Registration Number: ISRCTN36746902
• Lead Sponsor: Octapharma (Austria)

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2020-03-17
• Study Completion: 2021-02-17
• Last Update Posted: 27 June 2022

Recruitment & Eligibility

• Status: Completed
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

1. Documented congenital C1-INH deficiency with C1-INH functional activity less than 50% and C4 level below the laboratory reference range
2. Age =18 years at informed consent date
3. Signed informed consent
4. Patient must be capable to understand and comply with the relevant aspects of the study protocol
5. Women of childbearing potential must have a negative pregnancy test at screening as well as pre-infusion and must agree to use acceptable methods of contraception from screening until final visit
6. Fertile male patients must agree to use acceptable methods of contraception from screening until final visit

Exclusion Criteria

1. Any signs of an HAE attack OR HAE attack within 7 days prior to dosing with the IMP (OCTA-C1-INH) OR more than a total of 9 HAE attacks over the previous 3 months prior to dosing with the IMP
2. Patients who have received prophylactic or acute treatment with C1-INH (Berinert®, Cinryze®, HAEgarda®, Ruconest®, etc.), non-biological bradykinin pathway inhibitors (e.g., ecallantide, icatibant), or treatment with tranexamic acid within 2 weeks prior to dosing with the IMP
3. Patients who have received treatment with lanadelumab within 11 weeks prior to dosing with the IMP
4. Patients with planned dental, medical, or surgical procedures who will need pre-procedural HAE prophylaxis during the study period
5. Female patients taking estrogen-containing contraceptive regimen, hormone replacement therapy (excepting progesterone only contraceptives, which are permitted), or selective estrogen receptor modulators (e.g., tamoxifen). Male patients on specific androgen therapy (e.g., testosterone, danazol, dehydroepiandrosterone/androstenedione). Updated 09/03/2021: Any patients on specific androgen therapy (e.g., testosterone, danazol, dehydroepiandrosterone/androstenedione)
6. Any change (start, stop, or change in dose) in androgen therapy (e.g., oxandrolone, stanozolol) in the last 14 days prior to dosing with the IMP
7. Participated in any other investigational drug evaluation or received blood or a blood product, except for C1-INH, within 30 days prior to dosing with the IMP
8. Live viral vaccination within 30 days prior to screening
9. Acute infectious illness characterized by rapid onset of disease, a relatively brief period of symptoms, and resolution within a short period of time
10. Risk factors for thromboembolic events, including presence of indwelling venous catheter or access device, history of thrombosis, underlying atherosclerosis, morbid obesity (defined as BMI of =35 kg/m2 and experiencing obesity-related health conditions or =40 to 44.9 kg/m2), immobility, or medications known to increase thromboembolic risk
11. History of allergic reaction to C1-INH products or other blood products
12. History of clinically relevant antibody development against C1-INH
13. Any history of B-cell malignancy that was unresolved in the past 5 years
14. Pregnancy or lactation
15. Any clinically significant medical or psychiatric condition that, in the opinion of the Investigator, would interfere with the patient’s ability to participate in the study

Study & Design

• Study Type: Interventional
• Study Design: Not specified