A Randomized, Double Blind, Placebo Controlled, Phase 2a Study to Assess the Clinical Efficacy of ISIS 721744, a Second Generation Ligand Conjugated Antisense Inhibitor of Prekallikrein, in Patients with Hereditary Angioedema

Ionis Pharmaceuticals, Inc.0 sites24 target enrollmentMay 5, 2020

Overview

No summary available.

Registry

who.int

Start Date

May 5, 2020

End Date

TBD

Last Updated

5 years ago

Study Type

Interventional clinical trial of medicinal product

Sex

All

Sponsor

•1\)Documented diagnosis of HAE\-1/HAE\-2 (for inclusion in Part A) or HAE\-nC1\-INH (for inclusion in Part B)
•2\)Participants must experience a minimum of 2 HAE attacks (assessed by the Angioedema Activity Score \[AAS] and confirmed by the Investigator) during the Screening Period. Complete the AAS questionnaire on a daily basis (minimum of 4 daily assessments per week) for the duration of the Screening Period.
•3\)Access to, and the ability to use, \= 1 acute medication(s) to treat angioedema attacks
•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 24
•F.1\.3 Elderly (\>\=65 years) no
•F.1\.3\.1 Number of subjects for this age range

•1\) Anticipated use of short\-term prophylaxis for angioedema attacks for a pre\-planned procedure during the screening or study periods
•2\) Concurrent diagnosis of any other type of recurrent angioedema, including acquired or idiopathic angioedema
•3\) History of acquired coagulopathies or bleeding diasthesis
•4\) Active infection with HIV, hepatitis C or hepatitis B diagnosed by initial serological testing and confirmed with RNA testing, or prior treatment for hepatitis C. Patients at Screening who test positive by serology, but negative by RNA may be allowed in consultation with the Sponsor Medical Monitor.
•5\) Patients with a history of acquired coagulopathies or bleeding diathesis (e.g. thrombocytopenia, disseminated intravascular coagulation, coagulopathy of liver disease, drug\-induced platelet dysfunction, hyperfibrinolysis, acquired clotting factor inhibitors) and inherited bleeding disorders (e.g., hemophilia A, hemophilia B, other clotting
•factor deficiencies, qualitative platelet disorders, inherited thrombocytopenia, vascular abnormalities).
•6\) Any clinically significant renal or hepatic diseases.
•7\) Malignancy within 5 years, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated
•8\) Treatment with another investigational drug or biological agent within 1 month or 5 half\-lives, whichever is longer, of screening
•9\) Exposure to any of the following medications: