Clinical trial NCT00456508: Safety and Efficacy Study of Repeated Doses of DX-88 (Ecallantide) to Treat Attacks of Hereditary Angioedema (HAE)
Official title: Open-label Patient Continuation of DX-88 (Ecallantide) for Acute Hereditary Angioedema Attacks
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Hereditary Angioedema (HAE)
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2007-04-05
Enrollment: 147
Sex: ALL
Minimum age (years): 10
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to evaluate the efficacy and safety of repeated doses of ecallantide in the treatment of acute attacks of hereditary angioedema and to allow HAE patients continued access to ecallantide. In addition, patients enrolled in DX-88/20 (EDEMA4) trial will be followed up and treated for subsequent attacks in this trial.

Detailed description: This is an open label trial.

The study is designed to assess the efficacy and safety of 30 mg subcutaneous ecallantide in the treatment of acute attacks of hereditary angioedema. This study is designed to provide efficacy and safety data on repeated use of ecallantide. These data are intended to support the marketing authorization of ecallantide in the treatment of acute attacks of hereditary angioedema. Efficacy and safety of ecallantide will be evaluated in this study.

Eligibility criteria: Inclusion Criteria:

* 10 years of age or older
* Documented diagnosis of HAE (Type I or II)
* Willing and able to give informed consent
* Acute HAE attack at time of presentation

Exclusion Criteria:

* Receipt of an investigational drug or device, within 30 days prior to study treatment, other than DX-88 (ecallantide)
* Pregnancy or breastfeeding
* Receipt of non-investigational C1-INH or DX-88 within 72 hours of treatment
* Patients eligible for current, ongoing clinical trial in which DX 88 (ecallantide) is offered

Primary outcomes:
measure: Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hrs Post Dosing
timeFrame: 4 hrs post dose after every episode
Secondary outcomes:
measure: Treatment Outcome Score (TOS) at 4 Hrs Post Dosing, Based on the Patient Assessment of Baseline Severity of Symptoms
timeFrame: 4 hrs post dose after every episode
measure: Time to Significant Improvement
timeFrame: 15 min - 4 hrs post dose after every episode
Other outcomes:
Locations:
city: Scottsdale
country: United States
facility: Aaron Davis
state: Arizona
city: Little Rock
country: United States
facility: Arkansas Children's Hospital
state: Arkansas
city: Little Rock
country: United States
facility: Little Rock Allergy & Asthma Clinic
state: Arkansas
city: Berkeley
country: United States
facility: Alta Bates Summit Comprehensive Cancer Center
state: California
city: Crescent City
country: United States
facility: Pacific Coast Allergy
state: California
city: Granada Hills
country: United States
facility: Jacob Offenberger
state: California
city: Los Angeles
country: United States
facility: UCLA David Geffen School of Medicine, Department of Medicine
state: California
city: Colorado Springs
country: United States
facility: Asthma and Allergy Associates, P.C.
state: Colorado
city: Newark
country: United States
facility: Christiana Hospital, Christiana Care Health Services
state: Delaware
city: Washington
country: United States
facility: Georgetown University Medical Center, Georgetown University Hospital
state: District of Columbia
city: Miami
country: United States
facility: University of Miami, General Clinical Research Center
state: Florida
city: Tampa
country: United States
facility: University of South Florida, Asthma, Allergy and Immunology Clinical Research Unit
state: Florida
city: West Palm Beach
country: United States
facility: Roberson Allergy and Asthma
state: Florida
city: Atlanta
country: United States
facility: Family Allergy & Asthma Center, PC
state: Georgia
city: Columbus
country: United States
facility: Allergy Center of Brookstone
state: Georgia
city: Chicago
country: United States
facility: University Consultants in Allergy and Immunology
state: Illinois
city: Muncie
country: United States
facility: Muncie Allergy Center
state: Indiana
city: Overland Park
country: United States
facility: Kansas City Allergy & Asthma
state: Kansas
city: Wheaton
country: United States
facility: Institute for Asthma and Allergy
state: Maryland
city: Chestnut Hill
country: United States
facility: Brigham and Women's Hospital
state: Massachusetts
city: Clinton Township
country: United States
facility: Asthma and Allergy Institutes of Michigan
state: Michigan
city: Ypsilanti
country: United States
facility: Respiratory Medicine Research Institute of Michigan, PLC
state: Michigan
city: Las Vegas
country: United States
facility: Nevada Access to Research and Education Society
state: Nevada
city: Reno
country: United States
facility: University of Nevada School of Medicine
state: Nevada
city: Newark
country: United States
facility: UMDNJ- New Jersey Medical School
state: New Jersey
city: Albuquerque
country: United States
facility: Allergy Partners of Albuquerque
state: New Mexico
city: Mineola
country: United States
facility: Winthrop University Hospital
state: New York
city: Asheville
country: United States
facility: Allergy Partners of Western North Carolina
state: North Carolina
city: Cincinnati
country: United States
facility: University of Cincinnati, Division of Internal Medicine
state: Ohio
city: Columbus
country: United States
facility: Optimed Research, LLC
state: Ohio
city: Hershey
country: United States
facility: Penn State Milton S Hershey Medical Center
state: Pennsylvania
city: Philadelphia
country: United States
facility: Asthma Allergy and Pulmonary Associates
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Children's Hospital of Pittsburgh
state: Pennsylvania
city: Bristol
country: United States
facility: Highlands Allergy and Asthma Center, PC
state: Tennessee
city: Bryan
country: United States
facility: The Paull Allergy and Asthma Clinic, P.A
state: Texas
city: Dallas
country: United States
facility: AARA Research Center
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah Health Sciences Center
state: Utah
city: Norfolk
country: United States
facility: Clinical Research Associates of Tidewater
state: Virginia
city: Tacoma
country: United States
facility: Puget Sound Allergy, Asthma, & Immunology
state: Washington
city: Ottawa
country: Canada
facility: Allergy and Asthma Research Centre
state: Ontario
city: Amman
country: Jordan
facility: Jordan University Hospital
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References:
citation: Rubinstein E, Stolz LE, Sheffer AL, Stevens C, Bousvaros A. Abdominal attacks and treatment in hereditary angioedema with C1-inhibitor deficiency. BMC Gastroenterol. 2014 Apr 9;14:71. doi: 10.1186/1471-230X-14-71.
citation: MacGinnitie AJ, Davis-Lorton M, Stolz LE, Tachdjian R. Use of ecallantide in pediatric hereditary angioedema. Pediatrics. 2013 Aug;132(2):e490-7. doi: 10.1542/peds.2013-0646. Epub 2013 Jul 22.
citation: Sheffer AL, MacGinnitie AJ, Campion M, Stolz LE, Pullman WE. Outcomes after ecallantide treatment of laryngeal hereditary angioedema attacks. Ann Allergy Asthma Immunol. 2013 Mar;110(3):184-188.e2. doi: 10.1016/j.anai.2012.12.007. Epub 2013 Jan 5.
citation: Li HH, Campion M, Craig TJ, Soteres DF, Riedl M, Lumry WR, MacGinnitie AJ, Shea EP, Bernstein JA. Analysis of hereditary angioedema attacks requiring a second dose of ecallantide. Ann Allergy Asthma Immunol. 2013 Mar;110(3):168-72. doi: 10.1016/j.anai.2012.12.004. Epub 2013 Jan 8.