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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 : June 2, 2021
Sponsor:
Collaborator:
Dyax Corp.
Information provided by (Responsible Party):
Takeda ( Shire )

Study Description
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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

Study Design
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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


Arms and Interventions
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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.


Outcome Measures
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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.


Eligibility Criteria
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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.
Contacts and Locations
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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


Locations
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Sponsors and Collaborators
Shire
Dyax Corp.
Investigators
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Study Director: Shire Physician Shire
  Study Documents (Full-Text)

Documents provided by Takeda ( 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
Statistical Analysis Plan: Original  [PDF] May 24, 2016
Statistical Analysis Plan: Amendment 1  [PDF] May 3, 2017

More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT02586805    
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: June 2, 2021
Last Verified: May 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment 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 on https://vivli.org/ourmember/takeda/. 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://vivli.org/ourmember/takeda/
Keywords provided by Takeda ( 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
Hereditary Complement Deficiency Diseases
Primary Immunodeficiency Diseases
Genetic Diseases, Inborn
Immunologic Deficiency Syndromes
Antibodies, Monoclonal
Immunologic Factors
Physiological Effects of Drugs