Treatment of Idiopathic Angioedema With Xolair as Add-on Therapy

NCT02966314 | Completed Phase 4 Results DMC FDA Drug

• 4 other identifiers: 2016-0645SMPH/MEDICINE/MEDICINE*AA534220Protocol Version 5/22/2018
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

The overall hospitalizations for a diagnosis of angioedema doubled from the year 2000 to 2009. Although some of the cases represented hereditary angioedema or ace-inhibitor induced angioedema, the majority of episodes were idiopathic. Idiopathic Angioedema (IAE) can be life- threatening especially when affecting tissues within the respiratory tract. No clear guidelines exist for management of this important condition for clinicians. Current therapies typically include avoidance of potential triggers and use of medications either for prophylaxis or for acute events, such as antihistamines, corticosteroids, and epinephrine. There remains a critical need for therapeutic options to provide more effective prophylaxis.

Conditions

Idiopathic Angioedema

Keywords

Angioedema; Idiopathic Angioedema; IAE

Outcome Measures

Primary Outcomes (2)

• Mean 7-day Angioedema Activity Score (AAS7)

  The AAS7 Score is a measure of the frequency and intensity of angioedema episodes. The total possible range of scores over 7 days is 0-15 (mean day sum score) where higher scores indicate increased angioedema activity.

  baseline to end of treatment period at 6 months

• Presence of a 7-day Angioedema Activity Score (AAS7) Greater Than 0 Across All Treatment Visits

  The AAS7 Score is a measure of the frequency and intensity of angioedema episodes. The total possible range of scores over 7 days is 0-15 (mean day sum score) where higher scores indicate increased angioedema activity.

  across all visits during treatment period (up to 6 months)

Secondary Outcomes (10)

• Mean Angioedema Quality of Life (AE-QoL) Questionnaire

  baseline to end of treatment period at 6 months

• Mean Angioedema Quality of Life (AE-QoL) Questionnaire Across All Treatment Visits

  across all visits during treatment period (up to 6 months)

• Mean Visual Analog Scale

  baseline to end of treatment period at 6 months

• Presence of Visual Analog Scale Less Than 100 Across All Treatment Visits

  across all visits during treatment period (up to 6 months)

• Mean Number of IAE Episodes 2 Weeks Prior to Randomization

  baseline to end of treatment period at 6 months

Study Arms (2)

Placebo

PLACEBO COMPARATOR

Placebo contains sodium chloride 0.9% and will be provided by Novartis. Placebo will be administered as a subcutaneous injection.

Drug: Placebos

Omalizumab

ACTIVE COMPARATOR

Omalizumab is a sterile, white, preservative-free, lyophilized powder, contained in a single-use vial that will be reconstituted with sterile water for injection (SWFI), USP, and administered as a subcutaneous injection. Each omalizumab vial contains 202.5 mg of omalizumab, 145.5 mg sucrose, 2.8 mg L-histidine hydrochloride monohydrate, 1.8 mg L-histidine, and 0.5 mg polysorbate 20. Each vial is designed to deliver 150 mg of omalizumab in 1.2 mL after reconstitution with 1.4 mL SWFI, USP.

Drug: Omalizumab

Interventions

Omalizumab DRUG

Omalizumab 300mg subcutaneous injection once monthly for 6 months. Total dose will be divided into two 150mg/1.2mL injections.

Also known as: Xolair

Omalizumab

Placebos DRUG

Placebo Group (Sodium Chloride 0.9%) two 1.2mL subcutaneous injections once monthly for 6 months.

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Adults or adolescents who are 18 years or older at the time of screening with physician diagnosis of idiopathic angioedema
• Minimum of two episodes of idiopathic angioedema in the past 6 months at the time of screening
• Management of idiopathic angioedema with a stable controller treatment plan for the prior 6 months
• Complement profile (C1 Esterase inhibitor panel) within normal reference values
• If a woman is of child-bearing potential, she must agree to a reliable form of birth control including: abstinence, oral contraceptives (birth control pills), Depo-provera, an intrauterine device (IUD), or double-barrier contraception (partner using condom and participant using diaphragm, contraceptive sponge or cervical cap, and spermicidal)

You may not qualify if:

• Diagnosis of Hereditary Angioedema (HAE), Acquired Angioedema, or Ace-inhibitor associated angioedema, which are forms of angioedema with known mechanisms and alternate treatment options
• Chronic Urticaria (itching and/or hives) with or without Angioedema which are known mast cell mediated processes previously shown to be responsive to the use of omalizumab
• Previous usage of omalizumab in the last 3 months which can affect the patient-related outcomes and biomarker assessments if not "washed out" of the system
• Patients, who in the judgment of the investigator, have a history or condition that might compromise patient safety or compliance, interfere with evaluations, or preclude completion of the study

Sponsors & Collaborators

• University of Wisconsin, Madison: lead
• Novartis Pharmaceuticals: collaborator

Study Sites (1)

UW Madison

Madison, Wisconsin, 53792, United States

Location

Related Publications (1)

• Goswamy VP, Lee KE, McKernan EM, Fichtinger PS, Mathur SK, Viswanathan RK. Omalizumab for treatment of idiopathic angioedema. Ann Allergy Asthma Immunol. 2022 Nov;129(5):605-611.e1. doi: 10.1016/j.anai.2022.07.017. Epub 2022 Jul 30.

  PMID: 35914662 RESULT

MeSH Terms

Conditions

Angioedema

Interventions

Omalizumab

Condition Hierarchy (Ancestors)

Vascular Diseases; Cardiovascular Diseases; Urticaria; Skin Diseases, Vascular; Skin Diseases; Skin and Connective Tissue Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases

Intervention Hierarchy (Ancestors)

Antibodies, Anti-Idiotypic; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Antibodies, Monoclonal, Humanized; Antibodies, Monoclonal; Serum Globulins; Globulins

Results Point of Contact

• Title: Ravi Viswanathan
• Organization: University of Wisconsin - Madison

rviswanathan@medicine.wisc.edu

(608) 263-1081

Study Officials

• Sameer Mathur, MD/PhD

  UW Madison

  PRINCIPAL INVESTIGATOR

• Ravi Viswanathan, MD

  UW Madison

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, CARE PROVIDER
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 15, 2016
• First Posted: November 17, 2016
• Study Start: March 30, 2017
• Primary Completion: January 6, 2020
• Study Completion: January 6, 2020
• Last Updated: November 1, 2022
• Results First Posted: November 22, 2021

Record last verified: 2022-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)