Hereditary angioedema type I and type II MedDRA version: 20.0 Level: PT Classification code 10019860 Term: Hereditary angioedema System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.0 Level: LLT Classification code 10080956 Term: Hereditary angioedema type I System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.0 Level: LLT Classification code 10080960 Term: Hereditary angioedema type II System Organ Class: 1001033
Conditions
Interventions
Product Name: C1 Esterase Inhibitor Human
Product Code: OCTA-C1-INH
Pharmaceutical Form: Powder for solution for injection
Current Sponsor code: OCTA-C1-INH
Other descriptive name: C1 ESTERASE INHIBIT
Sponsors
Octapharma Pharmazeutika Produktionsges.m.b.H.
Eligibility
Sex/Gender
All
Inclusion criteria
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. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 17 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 3
Exclusion criteria
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). 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Primary end point(s): The primary endpoint is the PK parameters of OCTA-C1-INH, measured as C1-INH activity. The PK parameters that will be assessed for OCTA-C1-INH include: • Blood concentrations at each sampling time • Maximum blood concentration (Cmax) • Time to maximum concentration (Tmax) • Clearance (CL) • Area under the concentration-time curve (AUC) • AUC normalized by the dose (AUCnorm) • Mean residence time (MRT) • Incremental recovery (IR) • Volume of distribution (Vd) • Elimination half-life (T1/2);Timepoint(s) of evaluation of this end point: PK sampling (pre-injection, post-injection: at 0 and 15 minutes, 1 hour, 6, 12, 24, 48, 72, 120, 144, and 168 hours);Main Objective: The primary objective of this study is to assess the pharmacokinetic (PK) characteristics of OCTA-C1-INH after a single intravenous (IV) administration in HAE patients who are not experiencing an HAE attack.;Secondary Objective: • To assess blood level changes of C1-INH antigen and C4 level after a single IV administration of OCTA-C1-INH. • To assess the safety of OCTA-C1-INH IV administration. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • PK parameters of the following analytes: - C1-INH antigen - C4 level All PK parameters determined for the primary PK endpoint will also be determined for the secondary PK endpoints. • Safety Parameters: - Number and severity of adverse events (AEs) - Change in vital signs from pre- to post-injection - Change in laboratory parameters from pre- to post-injection - Blood nuclear antigen tests for hepatitis A virus [HAV], hepatitis B virus [HBV], hepatitis C virus [HCV], human immunodeficiency virus [HIV]-1/2, and parvovirus B19 - Anti-C1-INH antibodies;Timepoint(s) of evaluation of this end point: PK sampling (pre-injection, post-injection: at 0 and 15 minutes, 1 hour, 6, 12, 24, 48, 72, 120, 144, and 168 hours) safety (at all visits from the timepoint of injection until end of study) | — |
Countries
Belarus, Bulgaria, Czech Republic, Germany, Hungary, Poland, Russian Federation, Serbia, Ukraine
Contacts
Public ContactGlobal Clinical Project Manager
Octapharma Pharmazeutika Produktionsges.m.b.H.
Outcome results
None listed