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Treatment of Idiopathic Angioedema With Xolair as Add-on Therapy

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ClinicalTrials.gov Identifier: NCT02966314
Recruitment Status : Completed
First Posted : November 17, 2016
Results First Posted : November 22, 2021
Last Update Posted : November 1, 2022
Sponsor:
Collaborator:
Novartis Pharmaceuticals
Information provided by (Responsible Party):
University of Wisconsin, Madison

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.

Condition or disease Intervention/treatment Phase
Idiopathic Angioedema Drug: Omalizumab Drug: Placebos Phase 4

Detailed Description:
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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 12 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Care Provider)
Primary Purpose: Treatment
Official 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
Actual Study Start Date : March 30, 2017
Actual Primary Completion Date : January 6, 2020
Actual Study Completion Date : January 6, 2020

Resource links provided by the National Library of Medicine

Drug Information available for: Omalizumab

Arm Intervention/treatment
Placebo Comparator: Placebo
Placebo contains sodium chloride 0.9% and will be provided by Novartis. Placebo will be administered as a subcutaneous injection.
Drug: Placebos
Placebo Group (Sodium Chloride 0.9%) two 1.2mL subcutaneous injections once monthly for 6 months.

Active Comparator: Omalizumab
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
Omalizumab 300mg subcutaneous injection once monthly for 6 months. Total dose will be divided into two 150mg/1.2mL injections.
Other Name: Xolair




Primary Outcome Measures :
  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 Outcome Measures :
  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 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02966314


Locations
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United States, Wisconsin
UW Madison
Madison, Wisconsin, United States, 53792
Sponsors and Collaborators
University of Wisconsin, Madison
Novartis Pharmaceuticals
Investigators
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Principal Investigator: Sameer Mathur, MD/PhD UW Madison
Principal Investigator: Ravi Viswanathan, MD UW Madison
  Study Documents (Full-Text)

Documents provided by University of Wisconsin, Madison:
Publications of Results:
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Responsible Party: University of Wisconsin, Madison
ClinicalTrials.gov Identifier: NCT02966314    
Other Study ID Numbers: 2016-0645
SMPH/MEDICINE/MEDICINE*A ( Other Identifier: UW Madison )
A534220 ( Other Identifier: UW Madison )
Protocol Version 5/22/2018 ( Other Identifier: UW Madison )
First Posted: November 17, 2016    Key Record Dates
Results First Posted: November 22, 2021
Last Update Posted: November 1, 2022
Last Verified: October 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by University of Wisconsin, Madison:
Angioedema
Idiopathic Angioedema
IAE
Additional relevant MeSH terms:
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Angioedema
Vascular Diseases
Cardiovascular Diseases
Urticaria
Skin Diseases, Vascular
Skin Diseases
Hypersensitivity, Immediate
Hypersensitivity
Immune System Diseases
Omalizumab
Anti-Allergic Agents
Anti-Asthmatic Agents
Respiratory System Agents