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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

Primary Purpose

Hereditary Angioedema (HAE)

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
DX-2930 - 300mg/2wk
DX-2930 - 300mg/4wk
DX-2930 - 150mg/4wk
Placebo
Sponsored by
Shire
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional prevention trial for Hereditary Angioedema (HAE) focused on measuring DX-2930, Hereditary Angioedema, Dyax

Eligibility Criteria

12 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Clinical Research Center of Alabama, LLC
  • Medical Research of Arizona
  • UC San Diego School of Medicine
  • AIRE Medical of Los Angeles
  • Allergy & Asthma Clinical Research
  • IMMUNOe International Health & Research Centers
  • Asthma and Allergy Associates, P.C.
  • University of South Florida
  • University of Kansas Medical Center
  • Institute of Asthma & Allergy, P.C.
  • Massachusetts General Hospital
  • University of Michigan Hospital and Health System
  • Midwest Immunology Clinic
  • Washington University School of Medicine
  • Hudson-Essex Allergy, LLC
  • Atlantic Research Center, LLC
  • Winthrop University Hospital
  • The Mount Sinai Medical Center
  • American Health Research
  • Duke Asthma, Allergy and Airway Center
  • Bernstein Clinical Research Center, LLC
  • Optimed Research, LTD
  • Toledo Institute of Clinical Research
  • Penn State Milton S. Hershey Medical Center
  • Austin Regional Clinic
  • AARA Research Center
  • Intermountain Clinical Research
  • Allergy Associates of Utah
  • Virginia Commonwealth University
  • Marycliff Allergy Specialists
  • Children's Hospital of Wisconsin
  • Ottawa Allergy Research Corporation
  • Gordon Sussman Clinical Research Inc.
  • Clinique Specialisee en Allergie de la Capitale
  • Charité - University of Berlin
  • Hautklinik und Poliklinik der Universitätsmedizin
  • HZRM Hamophilie-Zentrum Rhein Main
  • Hospital L. Sacco, Milan University
  • Triumpharma
  • Sociedad Alergologica
  • Barts Health NHS Trust Clinical Research Centre

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Experimental

Experimental

Placebo Comparator

Arm Label

DX-2930 300 mg every 2 weeks

DX-2930 300 mg every 4 weeks

DX-2930 150 mg every 4 weeks

Placebo

Arm Description

300 mg DX-2930 administered every 2 weeks by subcutaneous injection.

300 mg DX-2930 administered every 4 weeks by subcutaneous injection

150 mg DX-2930 administered every 4 weeks by subcutaneous injection

Placebo administered every 2 weeks by subcutaneous injection.

Outcomes

Primary Outcome Measures

Rate of Investigator Confirmed Hereditary Angioedema (HAE) Attacks During Treatment Period
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

Rate of Investigator Confirmed Hereditary Angioedema (HAE) Attack Requiring Acute Treatment
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.
Rate of Moderate or Severe Investigator Confirmed Hereditary Angioedema (HAE) Attacks
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.
Rate of Investigator Confirmed Hereditary Angioedema (HAE) Attacks During Day 14 Through 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.

Full Information

First Posted
October 23, 2015
Last Updated
May 13, 2021
Sponsor
Shire
Collaborators
Dyax Corp.
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1. Study Identification

Unique Protocol Identification Number
NCT02586805
Brief Title
Efficacy and Safety Study of DX-2930 to Prevent Acute Angioedema Attacks in Patients With Type I and Type II HAE
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)
Study Type
Interventional

2. Study Status

Record Verification Date
May 2021
Overall Recruitment Status
Completed
Study Start Date
March 3, 2016 (Actual)
Primary Completion Date
April 13, 2017 (Actual)
Study Completion Date
April 13, 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Shire
Collaborators
Dyax Corp.

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hereditary Angioedema (HAE)
Keywords
DX-2930, Hereditary Angioedema, Dyax

7. Study Design

Primary Purpose
Prevention
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
125 (Actual)

8. Arms, Groups, and Interventions

Arm Title
DX-2930 300 mg every 2 weeks
Arm Type
Experimental
Arm Description
300 mg DX-2930 administered every 2 weeks by subcutaneous injection.
Arm Title
DX-2930 300 mg every 4 weeks
Arm Type
Experimental
Arm Description
300 mg DX-2930 administered every 4 weeks by subcutaneous injection
Arm Title
DX-2930 150 mg every 4 weeks
Arm Type
Experimental
Arm Description
150 mg DX-2930 administered every 4 weeks by subcutaneous injection
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo administered every 2 weeks by subcutaneous injection.
Intervention Type
Drug
Intervention Name(s)
DX-2930 - 300mg/2wk
Intervention Description
300 mg DX-2930 administered every 2 weeks by subcutaneous injection.
Intervention Type
Drug
Intervention Name(s)
DX-2930 - 300mg/4wk
Intervention Description
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.
Intervention Type
Drug
Intervention Name(s)
DX-2930 - 150mg/4wk
Intervention Description
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.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo administered every 2 weeks by subcutaneous injection.
Primary Outcome Measure Information:
Title
Rate of Investigator Confirmed Hereditary Angioedema (HAE) Attacks During Treatment Period
Description
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.
Time Frame
From Day 0 to Day 182
Secondary Outcome Measure Information:
Title
Rate of Investigator Confirmed Hereditary Angioedema (HAE) Attack Requiring Acute Treatment
Description
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.
Time Frame
From Day 0 to Day 182
Title
Rate of Moderate or Severe Investigator Confirmed Hereditary Angioedema (HAE) Attacks
Description
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.
Time Frame
From Day 0 to Day 182
Title
Rate of Investigator Confirmed Hereditary Angioedema (HAE) Attacks During Day 14 Through Day 182
Description
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.
Time Frame
From Day 14 to Day 182

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Shire Physician
Organizational Affiliation
Shire
Official's Role
Study Director
Facility Information:
Facility Name
Clinical Research Center of Alabama, LLC
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35209
Country
United States
Facility Name
Medical Research of Arizona
City
Scottsdale
State/Province
Arizona
ZIP/Postal Code
85251
Country
United States
Facility Name
UC San Diego School of Medicine
City
San Diego
State/Province
California
ZIP/Postal Code
92122
Country
United States
Facility Name
AIRE Medical of Los Angeles
City
Santa Monica
State/Province
California
ZIP/Postal Code
90404
Country
United States
Facility Name
Allergy & Asthma Clinical Research
City
Walnut Creek
State/Province
California
ZIP/Postal Code
94598
Country
United States
Facility Name
IMMUNOe International Health & Research Centers
City
Centennial
State/Province
Colorado
ZIP/Postal Code
80112
Country
United States
Facility Name
Asthma and Allergy Associates, P.C.
City
Colorado Springs
State/Province
Colorado
ZIP/Postal Code
80907
Country
United States
Facility Name
University of South Florida
City
Tampa
State/Province
Florida
ZIP/Postal Code
33613
Country
United States
Facility Name
University of Kansas Medical Center
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
Institute of Asthma & Allergy, P.C.
City
Chevy Chase
State/Province
Maryland
ZIP/Postal Code
20815
Country
United States
Facility Name
Massachusetts General Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02421
Country
United States
Facility Name
University of Michigan Hospital and Health System
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48106
Country
United States
Facility Name
Midwest Immunology Clinic
City
Plymouth
State/Province
Minnesota
ZIP/Postal Code
55446
Country
United States
Facility Name
Washington University School of Medicine
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
Hudson-Essex Allergy, LLC
City
Belleville
State/Province
New Jersey
ZIP/Postal Code
07109
Country
United States
Facility Name
Atlantic Research Center, LLC
City
Ocean City
State/Province
New Jersey
ZIP/Postal Code
07712
Country
United States
Facility Name
Winthrop University Hospital
City
Mineola
State/Province
New York
ZIP/Postal Code
11501
Country
United States
Facility Name
The Mount Sinai Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Facility Name
American Health Research
City
Charlotte
State/Province
North Carolina
ZIP/Postal Code
28277
Country
United States
Facility Name
Duke Asthma, Allergy and Airway Center
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27705
Country
United States
Facility Name
Bernstein Clinical Research Center, LLC
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45231
Country
United States
Facility Name
Optimed Research, LTD
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43235
Country
United States
Facility Name
Toledo Institute of Clinical Research
City
Toledo
State/Province
Ohio
ZIP/Postal Code
43617
Country
United States
Facility Name
Penn State Milton S. Hershey Medical Center
City
Hershey
State/Province
Pennsylvania
ZIP/Postal Code
17033
Country
United States
Facility Name
Austin Regional Clinic
City
Austin
State/Province
Texas
ZIP/Postal Code
78731
Country
United States
Facility Name
AARA Research Center
City
Dallas
State/Province
Texas
ZIP/Postal Code
75231
Country
United States
Facility Name
Intermountain Clinical Research
City
Draper
State/Province
Utah
ZIP/Postal Code
84020
Country
United States
Facility Name
Allergy Associates of Utah
City
Murray
State/Province
Utah
ZIP/Postal Code
84107
Country
United States
Facility Name
Virginia Commonwealth University
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23219
Country
United States
Facility Name
Marycliff Allergy Specialists
City
Spokane
State/Province
Washington
ZIP/Postal Code
99202
Country
United States
Facility Name
Children's Hospital of Wisconsin
City
Milwaukee
State/Province
Wisconsin
ZIP/Postal Code
53226
Country
United States
Facility Name
Ottawa Allergy Research Corporation
City
Ottawa
State/Province
Ontario
ZIP/Postal Code
K1G 6C6
Country
Canada
Facility Name
Gordon Sussman Clinical Research Inc.
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M4V 1R2
Country
Canada
Facility Name
Clinique Specialisee en Allergie de la Capitale
City
Quebec City
State/Province
Quebec
ZIP/Postal Code
G1V 4M6
Country
Canada
Facility Name
Charité - University of Berlin
City
Berlin
ZIP/Postal Code
10117
Country
Germany
Facility Name
Hautklinik und Poliklinik der Universitätsmedizin
City
Mainz
ZIP/Postal Code
55131
Country
Germany
Facility Name
HZRM Hamophilie-Zentrum Rhein Main
City
Morfelden-Walldorf
ZIP/Postal Code
64546
Country
Germany
Facility Name
Hospital L. Sacco, Milan University
City
Milan
ZIP/Postal Code
20157
Country
Italy
Facility Name
Triumpharma
City
Amman
ZIP/Postal Code
11941
Country
Jordan
Facility Name
Sociedad Alergologica
City
San Juan
ZIP/Postal Code
00918
Country
Puerto Rico
Facility Name
Barts Health NHS Trust Clinical Research Centre
City
London
ZIP/Postal Code
E1 2ES
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing 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.
IPD Sharing 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.
IPD Sharing URL
https://vivli.org/ourmember/takeda/
Citations:
PubMed Identifier
36326435
Citation
Beard N, Frese M, Smertina E, Mere P, Katelaris C, Mills K. Interventions for the long-term prevention of hereditary angioedema attacks. Cochrane Database Syst Rev. 2022 Nov 3;11(11):CD013403. doi: 10.1002/14651858.CD013403.pub2.
Results Reference
derived
PubMed Identifier
36153561
Citation
Craig TJ, Zaragoza-Urdaz RH, Li HH, Yu M, Ren H, Juethner S, Anderson J; HELP and HELP OLE Study Investigators. Effectiveness and safety of lanadelumab in ethnic and racial minority subgroups of patients with hereditary angioedema: results from phase 3 studies. Allergy Asthma Clin Immunol. 2022 Sep 24;18(1):85. doi: 10.1186/s13223-022-00721-y.
Results Reference
derived
PubMed Identifier
30480729
Citation
Banerji A, Riedl MA, Bernstein JA, Cicardi M, Longhurst HJ, Zuraw BL, Busse PJ, Anderson J, Magerl M, Martinez-Saguer I, Davis-Lorton M, Zanichelli A, Li HH, Craig T, Jacobs J, Johnston DT, Shapiro R, Yang WH, Lumry WR, Manning ME, Schwartz LB, Shennak M, Soteres D, Zaragoza-Urdaz RH, Gierer S, Smith AM, Tachdjian R, Wedner HJ, Hebert J, Rehman SM, Staubach P, Schranz J, Baptista J, Nothaft W, Maurer M; HELP Investigators. Effect of Lanadelumab Compared With Placebo on Prevention of Hereditary Angioedema Attacks: A Randomized Clinical Trial. JAMA. 2018 Nov 27;320(20):2108-2121. doi: 10.1001/jama.2018.16773. Erratum In: JAMA. 2019 Apr 23;321(16):1636.
Results Reference
derived

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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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