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Long-term Safety and Efficacy Study of DX-2930 (SHP643) to Prevent Acute Angioedema Attacks in Patients With Type I and Type II HAE

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02741596
Recruitment Status : Active, not recruiting
First Posted : April 18, 2016
Last Update Posted : April 12, 2019
Sponsor:
Information provided by (Responsible Party):
Shire

Brief Summary:
This study is an open-label, long term safety and efficacy study to evaluate DX-2930 in preventing acute angioedema attacks in participants with Type I and Type II HAE.

Condition or disease Intervention/treatment Phase
Hereditary Angioedema (HAE) Drug: DX-2930 Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 220 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Prevention
Official Title: HELP Study ExtensionTM: An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of DX-2930 for Prevention Against Acute Attacks of Hereditary Angioedema (HAE)
Actual Study Start Date : May 26, 2016
Estimated Primary Completion Date : November 4, 2019
Estimated Study Completion Date : November 4, 2019

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Rollover Participants
Participants who rollover from the DX-2930-03 study will receive 300 milligram (mg) DX-2930 subcutaneous injection at Day 0 followed by second dose following the first HAE attack and then once in every 2 weeks until the end of the treatment period (up to 924 days). A wash-out period of a minimum of 10 days and a maximum of 18 days is required between subsequent administrations.
Drug: DX-2930
Participants who rollover from the DX-2930-03 study will receive 300 milligram (mg) DX-2930 subcutaneous injection at Day 0 followed by second dose following the first HAE attack and then once in every 2 weeks until the end of the treatment period (up to 924 days). A wash-out period of a minimum of 10 days and a maximum of 18 days is required between subsequent administrations.

Experimental: Non-rollover Participants
Participants who were not participants in DX-2930-03 will receive 300 milligram (mg) DX-2930 subcutaneous injection once in every 2 weeks until the end of the treatment period (up to 924 days).
Drug: DX-2930
Participants who were not participants in DX-2930-03 will receive 300 milligram (mg) DX-2930 subcutaneous injection once in every 2 weeks until the end of the treatment period (up to 924 days).




Primary Outcome Measures :
  1. Evaluate the long-term safety of repeated subcutaneous (SC) administrations of DX-2930 through analyses based on treatment-emergent Adverse Events (AEs) [ Time Frame: From start of study drug administration up to Day 956 ]
    An adverse event (AE) is any untoward medical occurrence in a clinical trial participant whether or not it appears to have a causal relationship with the treatment administered. Treatment-emergent AEs are defined as AEs with onset at the time of or following the first exposure to open-label DX-2930 in this study, or medical conditions present prior to the start of treatment but increasing in severity or relationship at the time of or following the start of treatment.


Secondary Outcome Measures :
  1. Evaluate the long-term efficacy of DX-2930 in preventing HAE attacks in accordance with the HAE Attack Assessment and Reporting Procedures (HAARP) [ Time Frame: Baseline up to Day 956 ]
    The number of investigator-confirmed HAE attacks requiring acute treatment, number of moderate or severe investigator-confirmed HAE attacks and the number of high-morbidity investigator-confirmed HAE attacks during the treatment period will be assessed.

  2. Characterize the outer bounds of dosing frequency for DX-2930 by assessing the duration of time between a rollover participant's first and second open-label dose [ Time Frame: Baseline up to Day 956 ]
    Dosing frequency of DX-2930 will be assessed.



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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:

  • Male and female HAE participants who are 12 years of age or older at the time of screening
  • Documented diagnosis of HAE (Type I or II) based on

    1. Documented clinical history consistent with HAE (subcutaneous or mucosal, nonpruritic swelling episodes without accompanying urticaria).
    2. Diagnostic testing results obtained during screening (or a prior DX-2930 study) that confirm HAE Type I or II: C1 inhibitor (C1-INH) functional level less than (<) 40 percentage (%) of the normal level. Participants with functional C1-INH level 40-50% of the normal level may be enrolled if they also have a C4 level below the normal range. Participants may be retested if results are incongruent with clinical history or believed by the investigator to be confounded by long-term prophylactic (LTP) use. (It is understood that C1-INH therapy may alter the lab results of C1-INH assessments; therefore, the investigator's discretion in collaboration with Medical Monitor is advised for proper documentation of eligibility).
    3. At least one of the following: Age at reported onset of first angioedema symptoms less than or equal to (<=) 30 years, a family history consistent with HAE Type I or II, or C1q within normal range.
  • A historical baseline HAE attack rate of at least 1 attack per 12 weeks
  • Adult participants and caregivers of participants under the age of 18 are willing and able to read, understand, and sign an informed consent form. Participants age 12 to 17, whose caregiver has provided informed consent, are willing and able to read, understand and sign an assent form.
  • Males and females who are fertile and sexually active must adhere to contraception requirements for the duration of the study as

    1. Females (NOTE: Female rollover participants (those who previously participated in Study DX-2930-03 [NCT02586805]) of childbearing potential may continue to use the birth control method used during Study DX-2930-03 (NCT02586805).) of childbearing potential must agree to be abstinent or it is recommended to use highly effective forms of contraception from the screening period through 30 days after the final study visit. This includes stable doses (for 3 months prior to study screening) of combined estrogen and progestin-containing hormonal contraception associated with inhibition of ovulation (oral, injectable or implantable), progestin-only hormonal contraception associated with inhibition of ovulation, intra-uterine device (IUD, all types) or intrauterine hormone releasing systems (IUS). Notes: 1) A female whose male partner has had a vasectomy must agree to use one additional form of medically acceptable contraception. 2) Use of a male condom with or without spermicide or cervical cap, diaphragm or sponge with spermicide or a combination (double barrier methods) are not considered highly effective.
    2. 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.
    3. 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 final study visit.

Exclusion Criteria:

  • Discontinued from DX-2930-03 (NCT02586805) after enrollment for any reason.
  • If rolling over from DX-2930-03 (NCT02586805), presence of important safety concerns that would preclude participation in this study.
  • Concomitant diagnosis of another form of chronic, recurrent angioedema, such as acquired angioedema (AAE), HAE with normal C1 inhibitor (also known as HAE Type III), idiopathic angioedema, or recurrent angioedema associated with urticaria.
  • Dosing with an investigational drug (not including DX-2930 or other HAE therapies) or exposure to an investigational device within 4 weeks prior to screening.
  • Exposure to angiotensin-converting enzyme (ACE) inhibitors within 4 weeks prior to screening or any newly initiated or dose modification of estrogen-containing medications with systemic absorption (such as oral contraceptives or hormonal replacement therapy) 3 months prior to the screening visit.
  • Unwilling to discontinue use of long-term prophylactic therapy for HAE (C1-INH, attenuated androgens, or anti-fibrinolytics) within 3 weeks after starting DX-2930 treatment.
  • 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.
  • Participant 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 (eg, 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).

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): NCT02741596


  Hide Study Locations
Locations
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United States, Alabama
Clinical Research Center of Alabama
Birmingham, Alabama, United States, 35209
United States, Arizona
Medical Research of Arizona
Scottsdale, Arizona, United States, 85251
United States, California
University of California San Diego
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 Research Centers
Centennial, Colorado, United States, 80112
Asthma and Allergy Associates, PC
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 Asthma and Allergy
Chevy Chase, Maryland, United States, 20815
United States, Massachusetts
Massachusetts General Hospital
Boston, Massachusetts, United States, 02421
United States, Michigan
University of Michigan
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
Icahn School of Medicine at Mount Sinai
New York, New York, United States, 10029
United States, North Carolina
Clinical Research Center of Charlotte
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 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
Premier Clinical Research
Spokane, Washington, United States, 99202
United States, Wisconsin
Medical College of Wisconsin, Childrens Hospital
Milwaukee, Wisconsin, United States, 53226
Canada, Alberta
University of Alberta Hospital
Edmonton, Alberta, Canada, T6G 2V2
Canada, Ontario
Yang Medicine
Ottawa, Ontario, Canada, K1G 6C6
Gordon Sussman Clinical Research, Inc.
Toronto, Ontario, Canada, M4V 1R2
Canada, Quebec
Clinique Spécialisée en Allergie de la Capitale
Quebec City, Quebec, Canada, G1V 4M6
Germany
Charité - Universitätsmedizin Berlin
Berlin, Germany, 10117
Universitätsklinikum Frankfurt
Frankfurt, Germany, 60596
Hautklinik der Universitätsmedizin Mainz
Mainz, Germany, 55101
HZRM Hamophilie Zentrum Rhein Main GmbH
Moerfelden-Walldorf, Germany, 64546
Israel
Triumpharma Clinical Evaluation Centre
Amman, Jordan, Israel, 11941
Italy
University of Milan Luigi Sacco Hospital
Milan, Italy, 20157
Puerto Rico
Adler Medical Plaza
San Juan, Puerto Rico, 00918
United Kingdom
Royal London Hospital
London, United Kingdom, E1 1BB
Sponsors and Collaborators
Shire
Investigators
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Study Director: Study Director Shire

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT02741596     History of Changes
Other Study ID Numbers: DX-2930-04
2015-005255-27 ( EudraCT Number )
First Posted: April 18, 2016    Key Record Dates
Last Update Posted: April 12, 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

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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 Shire:
DX-2930(SHP643)
Dyax
Hereditary Angioedema

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