Efficacy and Safety Study of DX-2930 to Prevent Acute Angioedema Attacks in Patients With Type I and Type II HAE
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)
2 other identifiers
interventional
125
7 countries
41
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
Started Mar 2016
Shorter than P25 for phase_3
41 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
October 23, 2015
CompletedFirst Posted
Study publicly available on registry
October 26, 2015
CompletedStudy Start
First participant enrolled
March 3, 2016
CompletedPrimary Completion
Last participant's last visit for primary outcome
April 13, 2017
CompletedStudy Completion
Last participant's last visit for all outcomes
April 13, 2017
CompletedResults Posted
Study results publicly available
April 24, 2018
CompletedJune 2, 2021
May 1, 2021
1.1 years
October 23, 2015
March 20, 2018
May 13, 2021
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
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.
From Day 0 to Day 182
Secondary Outcomes (3)
Rate of Investigator Confirmed Hereditary Angioedema (HAE) Attack Requiring Acute Treatment
From Day 0 to Day 182
Rate of Moderate or Severe Investigator Confirmed Hereditary Angioedema (HAE) Attacks
From Day 0 to Day 182
Rate of Investigator Confirmed Hereditary Angioedema (HAE) Attacks During Day 14 Through Day 182
From Day 14 to Day 182
Study Arms (4)
DX-2930 300 mg every 2 weeks
EXPERIMENTAL300 mg DX-2930 administered every 2 weeks by subcutaneous injection.
DX-2930 300 mg every 4 weeks
EXPERIMENTAL300 mg DX-2930 administered every 4 weeks by subcutaneous injection
DX-2930 150 mg every 4 weeks
EXPERIMENTAL150 mg DX-2930 administered every 4 weeks by subcutaneous injection
Placebo
PLACEBO COMPARATORPlacebo administered every 2 weeks by subcutaneous injection.
Interventions
300 mg DX-2930 administered every 2 weeks by subcutaneous injection.
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.
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.
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Shirelead
- Dyax Corp.collaborator
Study Sites (41)
Clinical Research Center of Alabama, LLC
Birmingham, Alabama, 35209, United States
Medical Research of Arizona
Scottsdale, Arizona, 85251, United States
UC San Diego School of Medicine
San Diego, California, 92122, United States
AIRE Medical of Los Angeles
Santa Monica, California, 90404, United States
Allergy & Asthma Clinical Research
Walnut Creek, California, 94598, United States
IMMUNOe International Health & Research Centers
Centennial, Colorado, 80112, United States
Asthma and Allergy Associates, P.C.
Colorado Springs, Colorado, 80907, United States
University of South Florida
Tampa, Florida, 33613, United States
University of Kansas Medical Center
Kansas City, Kansas, 66160, United States
Institute of Asthma & Allergy, P.C.
Chevy Chase, Maryland, 20815, United States
Massachusetts General Hospital
Boston, Massachusetts, 02421, United States
University of Michigan Hospital and Health System
Ann Arbor, Michigan, 48106, United States
Midwest Immunology Clinic
Plymouth, Minnesota, 55446, United States
Washington University School of Medicine
St Louis, Missouri, 63110, United States
Hudson-Essex Allergy, LLC
Belleville, New Jersey, 07109, United States
Atlantic Research Center, LLC
Ocean City, New Jersey, 07712, United States
Winthrop University Hospital
Mineola, New York, 11501, United States
The Mount Sinai Medical Center
New York, New York, 10029, United States
American Health Research
Charlotte, North Carolina, 28277, United States
Duke Asthma, Allergy and Airway Center
Durham, North Carolina, 27705, United States
Bernstein Clinical Research Center, LLC
Cincinnati, Ohio, 45231, United States
Optimed Research, LTD
Columbus, Ohio, 43235, United States
Toledo Institute of Clinical Research
Toledo, Ohio, 43617, United States
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033, United States
Austin Regional Clinic
Austin, Texas, 78731, United States
AARA Research Center
Dallas, Texas, 75231, United States
Intermountain Clinical Research
Draper, Utah, 84020, United States
Allergy Associates of Utah
Murray, Utah, 84107, United States
Virginia Commonwealth University
Richmond, Virginia, 23219, United States
Marycliff Allergy Specialists
Spokane, Washington, 99202, United States
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53226, United States
Ottawa Allergy Research Corporation
Ottawa, Ontario, K1G 6C6, Canada
Gordon Sussman Clinical Research Inc.
Toronto, Ontario, M4V 1R2, Canada
Clinique Specialisee en Allergie de la Capitale
Québec, Quebec, G1V 4M6, Canada
Charité - University of Berlin
Berlin, 10117, Germany
Hautklinik und Poliklinik der Universitätsmedizin
Mainz, 55131, Germany
HZRM Hamophilie-Zentrum Rhein Main
Mörfelden-Walldorf, 64546, Germany
Hospital L. Sacco, Milan University
Milan, 20157, Italy
Triumpharma
Amman, 11941, Jordan
Sociedad Alergologica
San Juan, PR, 00918, Puerto Rico
Barts Health NHS Trust Clinical Research Centre
London, E1 2ES, United Kingdom
Related Publications (4)
Lumry WR, Maurer M, Weller K, Riedl MA, Watt M, Yu M, Devercelli G, Meunier J, Banerji A; HELP OLE Study Group. Long-term lanadelumab treatment improves health-related quality of life in patients with hereditary angioedema. Ann Allergy Asthma Immunol. 2023 Jul;131(1):101-108.e3. doi: 10.1016/j.anai.2023.03.028. Epub 2023 Apr 5.
PMID: 37028510DERIVEDBeard 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.
PMID: 36326435DERIVEDCraig 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.
PMID: 36153561DERIVEDBanerji 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.
PMID: 30480729DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Study Director
- Organization
- Shire
Study Officials
- STUDY DIRECTOR
Shire Physician
Shire
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- PREVENTION
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 23, 2015
First Posted
October 26, 2015
Study Start
March 3, 2016
Primary Completion
April 13, 2017
Study Completion
April 13, 2017
Last Updated
June 2, 2021
Results First Posted
April 24, 2018
Record last verified: 2021-05
Data Sharing
- IPD Sharing
- Will share
- Shared Documents
- STUDY PROTOCOL, SAP, ICF, 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.
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.