Single-arm pharmacokinetic phase 2a study of a single dose intravenous human plasma-derived C1-INH involving 20 HAE type I or type II patients, aged 18 years or older

Phase 1

• Conditions: Hereditary angioedema type I and type II; MedDRA version: 20.0Level: PTClassification code 10019860Term: Hereditary angioedemaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; MedDRA version: 21.0Level: LLTClassification code 10080956Term: Hereditary angioedema type ISystem Organ Class: 10010331 - Congenital, familial and genetic disorders; MedDRA version: 21.0Level: LLTClassification code 10080960Term: Hereditary angioedema type IISystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Body processes [G] - Immune system processes [G12]

• Registration Number: EUCTR2019-001693-28-DE
• Lead Sponsor: Octapharma Pharmazeutika Produktionsges.m.b.H.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-09-02
• Study Completion: 2021-02-17
• Last Update Posted: 4 September 2023

Recruitment & Eligibility

• Status: ot Recruiting
• 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.
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

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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified