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Completed

NCT Number: NCT02966314

Treatment of Idiopathic Angioedema With Xolair as Add-on Therapy

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.

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Key information

Conditions

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

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

UW Madison

Madison, Wisconsin, 53792, United States

About this study

This study is a randomized, double-blind, placebo-controlled, parallel group trial which will study the effects of omalizumab on patients with 2 or more episodes of Idiopathic Angioedema (IAE) in the past 6 months, despite current therapy. This study has three periods; screening, treatment, and follow-up. Subjects in the screening period will be consented and screened for eligibility criteria. 40 qualified individuals will enter the treatment period. Individuals will be randomized to either monthly subcutaneous administration of omalizumab 300mg (20 subjects) versus monthly placebo injection (20 subjects) in addition to their previously prescribed management plan for a total of 6 months. Individuals will then enter a follow-up period of 4 months. Study visits will occur monthly during the treatment period for update of clinical status and administration of omalizumab/placebo injection. After, the treatment period individuals will be seen twice for follow-up period. The entire study will consist of 10 study visits and will last approx. 10 months.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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)

Exclusion criteria

  • 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

Treatment and study plan

Omalizumab

Drug

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

Other names: Xolair

Placebos

Drug

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

Primary outcomes

  1. Mean 7-day Angioedema Activity Score (AAS7)

    Time frame: baseline to end of treatment period at 6 months

    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.

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

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

    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.

Secondary outcomes

  1. Mean Angioedema Quality of Life (AE-QoL) Questionnaire

    Time frame: baseline to end of treatment period at 6 months

    The AE-QoL Questionnaire is a 17-item survey, the total possible range of scores is transformed to a scale of 1-100 where higher score indicate increased impairment.

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

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

    The AE-QoL Questionnaire is a 17-item survey, the total possible range of scores is transformed to a scale of 1-100 where higher score indicate increased impairment.

  3. Mean Visual Analog Scale

    Time frame: baseline to end of treatment period at 6 months

    The effect of treatment compared to placebo on the change in IAE severity from baseline to the end of the treatment period as recorded on a 0-100 visual analog scale. Lower numbers indicate increased severity.

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

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

    The effect of treatment compared to placebo on the change in IAE severity from baseline to the end of the treatment period as recorded on a 0-100 visual analog scale. Lower numbers indicate increased severity.

  5. Mean Number of IAE Episodes 2 Weeks Prior to Randomization

    Time frame: baseline to end of treatment period at 6 months

    Idiopathic angioedema (IAE) refers to episodes of angioedema without urticaria for which no explanation can be found despite a thorough investigation. Patients are sometimes referred to as having IAE while further investigations are being performed, and therefore this term does not necessarily represent a distinct condition, but rather a diagnostic challenge in which the pathway of swelling is yet to be determined.

  6. Number of IAE Events Across All Treatment Visits

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

  7. Change in Duration of IAE Episodes

    Time frame: baseline to end of treatment period at 6 months

    Duration of IAE Episodes is participant reported for the 6 months prior to study (baseline) and again at the 6 month time point for the time on study.

  8. Number of Participants Who Visited Urgent Care or Emergency Room

    Time frame: baseline, end of treatment period at 6 months, follow up at 9 months

    Count of participants who visited urgent care or the emergency room is participant reported. The baseline measure is for the 6 months prior to study, at the 6 month time point for the time on study, and the 9 month time point for the previous 3 months on study.

  9. Number of Participants Who Used Rescue Medication or Corticosteroids

    Time frame: baseline, end of treatment period at 6 months, follow up at 9 months

  10. Number of Times Rescue Medications Were Used During the Treatment Period

    Time frame: up to 26 weeks

Sponsors and collaborators

Lead sponsor

University of Wisconsin, Madison

Other

Collaborators

  • Novartis Pharmaceuticals

Registry information

Official study title

A Phase IV, Randomized, Double-Blind, Placebo-Controlled Exploratory Study of Xolair (Omalizumab) for Treatment of Idiopathic Angioedema in Patients Who Remain Symptomatic Despite Current Therapy

Important dates

Study start
2017
Primary completion
2020
Study completion
2020
First posted
Nov 17, 2016
Registry last updated
Nov 1, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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