anadelumab tested in patients suffering from hereditary angioedema with normal C1-Inhibitor.

Phase 1

• Conditions: anadelumab in patients with Hereditary angioedema; MedDRA version: 20.0Level: PTClassification code 10019860Term: Hereditary angioedemaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; MedDRA version: 20.0Level: LLTClassification code 10074782Term: Hereditary angioedema breakthrough attackSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]

• Registration Number: EUCTR2018-004136-30-DE
• Lead Sponsor: Charité - Universitätsmedizin Berlin Klinik für Dermatologie und Allergologie

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-03-14
• Last Update Posted: 25 November 2019

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 5

Inclusion Criteria

•Adults (18 years or older)
•Documented HAE-nC1 (adapted from the definition by Zuraw et al., Allergy Asthma Proc 2012):
-A history of recurrent angioedema in the absence of concomitant hives or concomitant use of a medication known to cause angioedema.
-Documented normal or near normal C4, C1-INH antigen, and C1-INH function.
-One of the following: A) Demonstration of a FXII / PLG / ANGPT1 mutation that is associated with the disease. B) A positive family history of angioedema and documented evidence of lack of efficacy of chronic high dose antihistamine therapy (cetirizine at 40 mg/day or the equivalent, for at least 1 month and an interval expected to be associated with three or more attacks of angioedema).
•Clinical symptoms of angioedema attacks (at least 2 attacks within the last 3 months prior to screening; and at least 2 attacks within 12 weeks at maximum in the run-in period).
•Able to read, understand and willing to sign the informed consent form and abide with study procedures.
•No participation in other clinical trials 4 weeks before and after participation in this study.

Males and females who are fertile and sexually active must adhere to contraception requirements for the duration of the study:
•Females of childbearing potential must agree to be abstinent or else use any two of the following medically acceptable forms of contraception from the screening period through 30 days after the final study visit: progestin-only oral contraceptive, condom with or without spermicidal jelly, diaphragm or cervical cap with spermicidal jelly, or intra-uterine device (IUD, all types). A female whose male partner has had a vasectomy must agree to use one additional form of medically acceptable contraception.
•Females of non-childbearing potential, defined as surgically sterile (status post hysterectomy, bilateral oophorectomy, or bilateral tubal ligation) or post-menopausal for at least 12 months do not require contraception during the study.
•Males, including males who are surgically sterile (post vasectomy), with female partners of childbearing potential must agree to be abstinent or else use a medically acceptable form of contraception from the screening period through 60 days after the last IMP injection.

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 5
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

•Any other forms of angioedema not related to HAE-nC1.
•Treatment with a live (attenuated) virus vaccine within 4 weeks prior to Baseline visit.
•Exposure to angiotensin-converting enzyme (ACE) inhibitors or any estrogen-containing medications with systemic absorption (such as oral contraceptives or hormonal replacement therapy) within 4 weeks prior to screening.
•Use of long-term prophylactic therapy for HAE (C1-INH, attenuated androgens, or antifibrinolytics) within 2 weeks prior to the start of the treatment period (Day 0).
•Use of short-term prophylaxis for HAE by non-rollover subjects within 7 days prior to the start of the treatment period (Day 0). Short-term prophylaxis is defined as C1-INH, attenuated androgens, or anti-fibrinolytics used to avoid angioedema complications from medically indicated procedures.
•Any of the following liver function test abnormalities: alanine aminotransferase (ALT) > 3x upper limit of normal, or aspartate aminotransferase (AST) > 3x upper limit of normal, or total bilirubin > 2x upper limit of normal (unless the bilirubin elevation is a result of Gilbert’s Syndrome).
•Pregnancy or breastfeeding.
•Subject has any condition that, in the opinion of the Investigator or Sponsor, may compromise their safety or compliance, preclude successful conduct of the study, or interfere with interpretation of the results (e.g., history of substance abuse or dependence, a significant pre-existing illness or other major comorbidity that the Investigator considers may confound the interpretation of study results).
•Significant medical condition rendering the patient immunocompromised or not suitable for a clinical trial.
•Enrollment in another investigational treatment or device study or use of an investigational agent, or less than 4 weeks or 5 half-lives, whichever is longer, since end of another investigational device or drug trial.
•Subjects for whom there is concern about compliance with the protocol procedures.
•Patients with known hypersensitivity to any constituent of the products of lanadelumab.
•Dementia, altered mental status, or any psychiatric condition, or stay in an institution further to an official or court order that would prohibit the understanding or rendering of informed consent or participation in the study.
•Subjects who are study site employees, or immediate family members of a study site or sponsor employee.

Study & Design

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