Clinical trial NCT02586805: 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
Overall status: COMPLETED
Phase: PHASE3
Conditions: Hereditary Angioedema (HAE)
Keywords: DX-2930
Hereditary Angioedema
Dyax
Lead sponsor: Takeda
Sponsor class: INDUSTRY
Collaborators: name: Dyax Corp.
First posted: 2015-10-26
Enrollment: 125
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: PREVENTION

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.


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.

Primary outcomes:
measure: Rate of Investigator Confirmed Hereditary Angioedema (HAE) Attacks During Treatment Period
timeFrame: From Day 0 to Day 182
Secondary outcomes:
measure: Rate of Investigator Confirmed Hereditary Angioedema (HAE) Attack Requiring Acute Treatment
timeFrame: From Day 0 to Day 182
measure: Rate of Moderate or Severe Investigator Confirmed Hereditary Angioedema (HAE) Attacks
timeFrame: From Day 0 to Day 182
measure: Rate of Investigator Confirmed Hereditary Angioedema (HAE) Attacks During Day 14 Through Day 182
timeFrame: From Day 14 to Day 182
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Clinical Research Center of Alabama, LLC
state: Alabama
city: Scottsdale
country: United States
facility: Medical Research of Arizona
state: Arizona
city: San Diego
country: United States
facility: UC San Diego School of Medicine
state: California
city: Santa Monica
country: United States
facility: AIRE Medical of Los Angeles
state: California
city: Walnut Creek
country: United States
facility: Allergy & Asthma Clinical Research
state: California
city: Centennial
country: United States
facility: IMMUNOe International Health & Research Centers
state: Colorado
city: Colorado Springs
country: United States
facility: Asthma and Allergy Associates, P.C.
state: Colorado
city: Tampa
country: United States
facility: University of South Florida
state: Florida
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Chevy Chase
country: United States
facility: Institute of Asthma & Allergy, P.C.
state: Maryland
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan Hospital and Health System
state: Michigan
city: Plymouth
country: United States
facility: Midwest Immunology Clinic
state: Minnesota
city: Saint Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
city: Belleville
country: United States
facility: Hudson-Essex Allergy, LLC
state: New Jersey
city: Ocean City
country: United States
facility: Atlantic Research Center, LLC
state: New Jersey
city: Mineola
country: United States
facility: Winthrop University Hospital
state: New York
city: New York
country: United States
facility: The Mount Sinai Medical Center
state: New York
city: Charlotte
country: United States
facility: American Health Research
state: North Carolina
city: Durham
country: United States
facility: Duke Asthma, Allergy and Airway Center
state: North Carolina
city: Cincinnati
country: United States
facility: Bernstein Clinical Research Center, LLC
state: Ohio
city: Columbus
country: United States
facility: Optimed Research, LTD
state: Ohio
city: Toledo
country: United States
facility: Toledo Institute of Clinical Research
state: Ohio
city: Hershey
country: United States
facility: Penn State Milton S. Hershey Medical Center
state: Pennsylvania
city: Austin
country: United States
facility: Austin Regional Clinic
state: Texas
city: Dallas
country: United States
facility: AARA Research Center
state: Texas
city: Draper
country: United States
facility: Intermountain Clinical Research
state: Utah
city: Murray
country: United States
facility: Allergy Associates of Utah
state: Utah
city: Richmond
country: United States
facility: Virginia Commonwealth University
state: Virginia
city: Spokane
country: United States
facility: Marycliff Allergy Specialists
state: Washington
city: Milwaukee
country: United States
facility: Children's Hospital of Wisconsin
state: Wisconsin
city: Ottawa
country: Canada
facility: Ottawa Allergy Research Corporation
state: Ontario
city: Toronto
country: Canada
facility: Gordon Sussman Clinical Research Inc.
state: Ontario
city: Quebec City
country: Canada
facility: Clinique Specialisee en Allergie de la Capitale
state: Quebec
city: Berlin
country: Germany
facility: Charité - University of Berlin
city: Mainz
country: Germany
facility: Hautklinik und Poliklinik der Universitätsmedizin
city: Morfelden-Walldorf
country: Germany
facility: HZRM Hamophilie-Zentrum Rhein Main
city: Milan
country: Italy
facility: Hospital L. Sacco, Milan University
city: Amman
country: Jordan
facility: Triumpharma
city: San Juan
country: Puerto Rico
facility: Sociedad Alergologica
city: London
country: United Kingdom
facility: Barts Health NHS Trust Clinical Research Centre
Study officials:
affiliation: Shire
name: Shire Physician
role: STUDY_DIRECTOR
References:
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.
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.
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.