Prospective, Multicenter, Randomized, Double-blind, Parallel Group, Placebo- Controlled, Efficacy and Safety Phase 3 Study of an Intravenous Human Plasma- Derived C1 Esterase Inhibitor (C1-INH) Concentrate in Participants With Congenital C1-INH Deficiency for the Treatment and Pre-procedure Prevention of Acute Hereditary Angioedema Attacks

Prospective, multicenter, randomized, double-blind, parallel group, placebo- controlled, efficacy and safety phase 3 study of an intravenous human plasma- derived C1 esterase inhibitor (C1-INH) concentrate in participants with congenital C1-INH deficiency for the treatment and pre-procedure prevention of acute hereditary angioedema attacks

Start Date30 Apr 2024

Sponsor / Collaborator

Octapharma AG

100 Clinical Results associated with Complement-C1 inhibitor protein(Octapharma AG)

100 Translational Medicine associated with Complement-C1 inhibitor protein(Octapharma AG)

100 Patents (Medical) associated with Complement-C1 inhibitor protein(Octapharma AG)

Literatures (Medical) associated with Complement-C1 inhibitor protein(Octapharma AG)

01 Feb 2024·Journal of Allergy and Clinical Immunology: Global

Plasma-derived C1 esterase inhibitor pharmacokinetics and safety in patients with hereditary angioedema

Article

Author: Zabrodska, Liudmyla ; Nenasheva, Natalia ; Totolyan, Areg ; Krivenchuk, Vitaliy ; Martinez-Saguer, Inmaculada ; Bork, Konrad ; Latysheva, Tatiana ; Chopyak, Valentyna

BackgroundOver 40 years of use demonstrates that complement 1 esterase inhibitor (C1-INH) concentrate is effective and well tolerated for acute edema attacks and prophylaxis in patients with hereditary angioedema. OCTA-C1-INH is a new stable, virus-inactivated, nanofiltrated concentrate of C1-INH derived from human plasma.ObjectiveWe investigated the pharmacokinetics and safety profile of new C1-INH in people with hereditary angioedema during an attack-free period.MethodsIn this prospective, multicenter, open-label, single-arm study, adults with hereditary angioedema type I/II received a single intravenous dose of 20 IU/kg C1-INH. Blood samples were taken ≤30 minutes before infusion, and 0, 0.25, 1, 2, 6, 12, 24, 48, 72, 120, 144, and 168 hours after infusion. The primary end point was assessing the pharmacokinetic parameters of C1-INH measured by C1-INH activity. Safety end points were also examined.ResultsTwenty patients received a single dose of 20 IU/kg new C1-INH with a mean (standard deviation) total dose of 1457.3 (356.51) IU. Mean (standard deviation) area under the curve normalized by dose was 51.6 (17.9) h∙IU/mL/IU, maximum blood concentration was 1.14 (0.989) IU/mL, incremental recovery was 0.0466 (0.051) (IU∙kg)/(IU∙mL), half-life was 0.598 (0.716) hours, and time to maximum concentration was 0.598 (0.716) hours. No thromboembolic events were recorded. No treatment-emergent adverse events were rated as severe/serious.ConclusionPK parameters of new C1-INH were in line with those reported for other C1-INH concentrates. New C1-INH demonstrated a favorable safety profile in patients with C1-INH deficiency. Further studies are warranted to determine the effectiveness and longer-term safety of new C1-INH.

01 Jul 2019·Nature Chemical BiologyQ1 · BIOLOGY

A small-molecule inhibitor of C5 complement protein

Q1 · BIOLOGY

Article

Author: Tichkule, Ritesh ; Murphy, Jason ; Bolduc, Philippe ; Duca, Jose ; Mogi, Muneto ; Capparelli, Michael ; Jendza, Keith ; Mainolfi, Nello ; Wiesmann, Christian ; De Erkenez, Andrea ; Salcius, Michael ; Horton, Patricia A ; Meredith, Erik ; Nguyen, Anh ; Schirle, Markus ; Heyder, Jessica ; McLaughlin, Doug ; Darsigny, Veronique ; Kato, Mitsunori ; Romanowski, Michael J ; Srinivas, Honnappa ; Anderson, Karen ; Michaud, Gregory A ; Ferrara, Luciana ; Namil, Abdel ; Liu, Fang ; Be, Celine

The complement pathway is an important part of the immune system, and uncontrolled activation is implicated in many diseases. The human complement component 5 protein (C5) is a validated drug target within the complement pathway, as an anti-C5 antibody (Soliris) is an approved therapy for paroxysmal nocturnal hemoglobinuria. Here, we report the identification, optimization and mechanism of action for the first small-molecule inhibitor of C5 complement protein.

01 Mar 2019·BMJ Case Reports

New approach in prophylactic treatment of a challenged HAE patient

Article

Author: Bygum, Anette ; Jordal, Oda

Hereditary angioedema (HAE) is a relapsing swelling disorder which can cause severe pain, affect quality of life and potentially be life threatening with involvement of the airways. We present a 34-year-old immigrant who suffered from very frequent and severe HAE attacks. The attacks often involved the face, mouth and the airways. She often went to the hospital for treatment, where the language barrier made the situation complicated. The traditional therapy for HAE was not successful treating this patient. In June 2017, off-label treatment with prophylactic subcutaneous complement C1-inhibitor concentrate was initiated. The treatment was very successful and the patient has not been hospitalised since. Treatment for HAE is nowadays under investigation, and many drugs are under development. Especially, medication which works prophylactically and is administered orally or subcutaneously is in the horizon.

100 Deals associated with Complement-C1 inhibitor protein(Octapharma AG)

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Indication	Highest Phase	Country/Location	Organization	Date
Hereditary Angioedema	Phase 3	US	Octapharma AG	30 Apr 2024
Hereditary Angioedema	Phase 3	PE	Octapharma AG	30 Apr 2024
Hereditary Angioedema	Phase 3	RO	Octapharma AG	30 Apr 2024
Hereditary Angioedema	Phase 3	RS	Octapharma AG	30 Apr 2024
Hereditary Angioedema	Phase 3	UA	Octapharma AG	30 Apr 2024
Hereditary Angioedema	Phase 3	MX	Octapharma AG	30 Apr 2024
Hereditary Angioedema	Phase 3	AM	Octapharma AG	30 Apr 2024
Hereditary Angioedema	Phase 3	BG	Octapharma AG	30 Apr 2024

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