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Efficacy and Safety Study of DX-2930 to Prevent Acute Angioedema Attacks in Patients With Type I and Type II HAE

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ClinicalTrials.gov Identifier: NCT02586805
Recruitment Status : Completed
First Posted : October 26, 2015
Results First Posted : April 24, 2018
Last Update Posted : April 16, 2019
Sponsor:
Collaborator:
Dyax Corp.
Information provided by (Responsible Party):
Shire

Brief Summary:
This is a phase 3, multicenter, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of DX-2930 in preventing acute angioedema attacks in patients with Type I and Type II HAE.

Condition or disease Intervention/treatment Phase
Hereditary Angioedema (HAE) Drug: DX-2930 - 300mg/2wk Drug: DX-2930 - 300mg/4wk Drug: DX-2930 - 150mg/4wk Drug: Placebo Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 125 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Prevention
Official Title: HELP Study: A Multicenter, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study to Evaluate DX-2930 For Long-Term Prophylaxis Against Acute Attacks of Hereditary Angioedema (HAE)
Actual Study Start Date : March 3, 2016
Actual Primary Completion Date : April 13, 2017
Actual Study Completion Date : April 13, 2017

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: DX-2930 300 mg every 2 weeks
300 mg DX-2930 administered every 2 weeks by subcutaneous injection.
Drug: DX-2930 - 300mg/2wk
300 mg DX-2930 administered every 2 weeks by subcutaneous injection.

Experimental: DX-2930 300 mg every 4 weeks
300 mg DX-2930 administered every 4 weeks by subcutaneous injection
Drug: DX-2930 - 300mg/4wk
300 mg DX-2930 administered every 4 weeks by subcutaneous injection. To maintain the study blind, subjects will be given placebo injections every other 2 weeks when they are not receiving drug.

Experimental: DX-2930 150 mg every 4 weeks
150 mg DX-2930 administered every 4 weeks by subcutaneous injection
Drug: DX-2930 - 150mg/4wk
150 mg DX-2930 administered every 4 weeks by subcutaneous injection. To maintain the study blind, subjects will be given placebo injections every other 2 weeks when they are not receiving drug.

Placebo Comparator: Placebo
Placebo administered every 2 weeks by subcutaneous injection.
Drug: Placebo
Placebo administered every 2 weeks by subcutaneous injection.




Primary Outcome Measures :
  1. Rate of Investigator Confirmed Hereditary Angioedema (HAE) Attacks During Treatment Period [ Time Frame: From Day 0 to Day 182 ]
    HAE attack was defined as a discrete episode during which the participant progressed from no angioedema to symptoms of angioedema. Rate of investigator confirmed HAE attacks was analyzed using a generalized linear model (GLM) for count data assuming a poisson distribution with a log link function and Pearson chi-square scaling of standard errors to account for potential overdispersion. The logarithm of time in days each subject was observed during the treatment period was used as an offset variable in the model.


Secondary Outcome Measures :
  1. Rate of Investigator Confirmed Hereditary Angioedema (HAE) Attack Requiring Acute Treatment [ Time Frame: From Day 0 to Day 182 ]
    HAE attack was defined as a discrete episode during which the participant progressed from no angioedema to symptoms of angioedema. Rate of investigator confirmed HAE attack was analyzed using the GLM for count data assuming a poisson distribution with a log link function and Pearson chi-square scaling of standard errors to account for potential overdispersion. The logarithm of time in days each subject was observed during the treatment period was used as an offset variable in the model.

  2. Rate of Moderate or Severe Investigator Confirmed Hereditary Angioedema (HAE) Attacks [ Time Frame: From Day 0 to Day 182 ]
    HAE attack was defined as a discrete episode during which the participant progressed from no angioedema to symptoms of angioedema. Moderate and severe investigator-confirmed HAE attacks were the attacks that were moderate or severe as per the HAE attack assessment and reporting procedures (HAARP) defined severity. The overall severity of attack was determined by the investigator using following definitions: mild (transient or mild discomfort), moderate (mild to moderate limitation in activity), severe (marked limitation in activity). Rate of moderate or severe investigator confirmed HAE attack was analyzed using the GLM for count data assuming a poisson distribution with a log link function and Pearson chi-square scaling of standard errors to account for potential overdispersion. The logarithm of time in days each subject was observed during the treatment period was used as an offset variable in the model.

  3. Rate of Investigator Confirmed Hereditary Angioedema (HAE) Attacks During Day 14 Through Day 182 [ Time Frame: From Day 14 to Day 182 ]
    HAE attack was defined as a discrete episode during which the participant progressed from no angioedema to symptoms of angioedema. Rate of investigator confirmed HAE attacks during day 14 after study drug administration through day 182 was analyzed by the same poisson regression model as in the primary endpoint analysis.



Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Males and females 12 years of age or older at time of screening
  • Documented diagnosis of HAE, Type I or II
  • Baseline rate of at least 1 Investigator-confirmed HAE attack per 4 weeks
  • Adult subjects and caregivers of subjects under the age of 18 are willing and able to read, understand, and sign an informed consent form. Subjects age 12 to 17, whose caregiver provides informed consent, are willing and able to read, understand an dsign an assent form.
  • Males and femailes who are fertile and sexually active must adhere to contraception requirements.

Exclusion Criteria:

  • Concomitant diagnosis of another form of chronic, recurrent angioedema, such as acquired angioedema, idiopathic angioedema, or recurrent angioedema associated with urticaria.
  • Participation in a prior DX-2930 study
  • Treatment with any other investigational drug or exposure to an investigational device within 4 weeks prior screening
  • Exposure to angiotensin-converting enzyme (ACE) inhibitors or any estrogen-containing medications within 4 weeks prior to screening.
  • Exposure to androgens within 2 weeks prior to entering the run-in period.
  • Use of long-term prophylactic therapy for HAE within 2 weeks prior to entering the run-in period.
  • Use of short-term prophylaxis for HAE within 7 days prior to entering the run-in period.
  • 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.

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): NCT02586805


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Locations
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United States, Alabama
Clinical Research Center of Alabama, LLC
Birmingham, Alabama, United States, 35209
United States, Arizona
Medical Research of Arizona
Scottsdale, Arizona, United States, 85251
United States, California
UC San Diego School of Medicine
San Diego, California, United States, 92122
AIRE Medical of Los Angeles
Santa Monica, California, United States, 90404
Allergy & Asthma Clinical Research
Walnut Creek, California, United States, 94598
United States, Colorado
IMMUNOe International Health & Research Centers
Centennial, Colorado, United States, 80112
Asthma and Allergy Associates, P.C.
Colorado Springs, Colorado, United States, 80907
United States, Florida
University of South Florida
Tampa, Florida, United States, 33613
United States, Kansas
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160
United States, Maryland
Institute of Asthma & Allergy, P.C.
Chevy Chase, Maryland, United States, 20815
United States, Massachusetts
Massachusetts General Hospital
Boston, Massachusetts, United States, 02421
United States, Michigan
University of Michigan Hospital and Health System
Ann Arbor, Michigan, United States, 48106
United States, Minnesota
Midwest Immunology Clinic
Plymouth, Minnesota, United States, 55446
United States, Missouri
Washington University School of Medicine
Saint Louis, Missouri, United States, 63110
United States, New Jersey
Hudson-Essex Allergy, LLC
Belleville, New Jersey, United States, 07109
Atlantic Research Center, LLC
Ocean City, New Jersey, United States, 07712
United States, New York
Winthrop University Hospital
Mineola, New York, United States, 11501
The Mount Sinai Medical Center
New York, New York, United States, 10029
United States, North Carolina
American Health Research
Charlotte, North Carolina, United States, 28277
Duke Asthma, Allergy and Airway Center
Durham, North Carolina, United States, 27705
United States, Ohio
Bernstein Clinical Research Center, LLC
Cincinnati, Ohio, United States, 45231
Optimed Research, LTD
Columbus, Ohio, United States, 43235
Toledo Institute of Clinical Research
Toledo, Ohio, United States, 43617
United States, Pennsylvania
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, United States, 17033
United States, Texas
Austin Regional Clinic
Austin, Texas, United States, 78731
AARA Research Center
Dallas, Texas, United States, 75231
United States, Utah
Intermountain Clinical Research
Draper, Utah, United States, 84020
Allergy Associates of Utah
Murray, Utah, United States, 84107
United States, Virginia
Virginia Commonwealth University
Richmond, Virginia, United States, 23219
United States, Washington
Marycliff Allergy Specialists
Spokane, Washington, United States, 99202
United States, Wisconsin
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, United States, 53226
Canada, Ontario
Ottawa Allergy Research Corporation
Ottawa, Ontario, Canada, K1G 6C6
Gordon Sussman Clinical Research Inc.
Toronto, Ontario, Canada, M4V 1R2
Canada, Quebec
Clinique Specialisee en Allergie de la Capitale
Quebec City, Quebec, Canada, G1V 4M6
Germany
Charité - University of Berlin
Berlin, Germany, 10117
Hautklinik und Poliklinik der Universitätsmedizin
Mainz, Germany, 55131
HZRM Hamophilie-Zentrum Rhein Main
Morfelden-Walldorf, Germany, 64546
Italy
Hospital L. Sacco, Milan University
Milan, Italy, 20157
Jordan
Triumpharma
Amman, Jordan, 11941
Puerto Rico
Sociedad Alergologica
San Juan, Puerto Rico, 00918
United Kingdom
Barts Health NHS Trust Clinical Research Centre
London, United Kingdom, E1 2ES
Sponsors and Collaborators
Shire
Dyax Corp.
Investigators
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Study Director: Shire Physician Shire
  Study Documents (Full-Text)

Documents provided by Shire:
Study Protocol: Original  [PDF] September 14, 2015
Study Protocol: Amendment 1  [PDF] December 14, 2015
Study Protocol: Amendment 2  [PDF] April 21, 2016
Study Protocol: Amendment 3  [PDF] January 9, 2017


Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT02586805     History of Changes
Other Study ID Numbers: DX-2930-03
2015-003943-20 ( EudraCT Number )
First Posted: October 26, 2015    Key Record Dates
Results First Posted: April 24, 2018
Last Update Posted: April 16, 2019
Last Verified: April 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Shire provides access to the de-identified individual participant data for eligible studies to aid qualified researchers in addressing legitimate scientific objectives. These IPDs will be provided following approval of a data sharing request, and under the terms of a data sharing agreement.
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Informed Consent Form (ICF)
Clinical Study Report (CSR)
Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described in the Data Sharing section of the www.shiretrials.com website. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
URL: https://www.shiretrials.com/en/our-commitment-to-transparency/data-sharing-with-researchers
Keywords provided by Shire:
DX-2930
Hereditary Angioedema
Dyax
Additional relevant MeSH terms:
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Angioedema
Angioedemas, Hereditary
Vascular Diseases
Cardiovascular Diseases
Urticaria
Skin Diseases, Vascular
Skin Diseases
Hypersensitivity, Immediate
Hypersensitivity
Immune System Diseases
Genetic Diseases, Inborn
Antibodies, Monoclonal
Immunologic Factors
Physiological Effects of Drugs