Clinical trial NCT00262080: Efficacy and Safety Study of DX-88 to Treat Acute Attacks of Hereditary Angioedema (HAE)
Official title: A Double-blind, Placebo-controlled Study (72 Patients, Randomized 1:1) Followed by a Repeat-dosing Phase to Assess the Efficacy and Safety of DX-88 (Ecallantide; Recombinant Plasma Kallikrein Inhibitor) for the Treatment of Acute Attacks of Hereditary Angioedema
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Hereditary Angioedema (HAE)
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2005-12-06
Enrollment: 91
Sex: ALL
Minimum age (years): 10
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to determine if a subcutaneous dose of DX-88 (ecallantide; an investigational product) is safe and relieves symptoms of HAE in patients suffering from moderate to severe acute attacks of HAE.


Eligibility criteria: Inclusion Criteria:

* Age 10 and older
* Documented diagnosis of HAE, Type I or II
* Executed informed consent
* Presentation for treatment within 8 hours of patient recognition of moderate to severe HAE attack

Exclusion Criteria:

* Receipt of investigational drug or device, other than DX-88, within 30 days of treatment
* Receipt of non-investigational C1-INH (C1 esterase inhibitor) within 7 days of treatment
* Diagnostic of acquired angioedema, estrogen-dependent angioedema or drug induced angioedema
* Pregnancy or breastfeeding
* Patients who have received DX-88 within 7 days of presentation for dosing in the Double-blind Phase

Primary outcomes:
measure: Treatment Outcome Score at 4 Hours Post-Dose
timeFrame: 4 hours post-dose (DOUBLE-BLIND PART)
Secondary outcomes:
measure: Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hours Post-dose
timeFrame: baseline, 4 hours post-dose (DOUBLE-BLIND PART)
measure: Time to Significant Improvement in Overall Response
timeFrame: 4 hours post-dose (DOUBLE-BLIND PART)
Other outcomes:
measure: Number of Patients With Symptom Complexes of Treated Attack at Baseline(DOUBLE-BLIND PART)
timeFrame: Baseline
measure: Treatment Outcome Score at 4 Hours Post-Dose Over Multiple Treatment Episodes
timeFrame: 4 hours post-dose (REPEAT-DOSING PART)
measure: Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hours Post-dose Over Multiple Treatment Episodes
timeFrame: baseline, 4 hours post-dose (REPEAT-DOSING PART)
measure: Time to Significant Improvement in Overall Response Over Multiple Treatment Episodes
timeFrame: 4 hours post-dose (REPEAT-DOSING PART)
Locations:
city: Wheaton
country: United States
facility: Institute for Asthma and Allergy
state: Maryland
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: Bernstein JA, Shea EP, Koester J, Iarrobino R, Pullman WE. Assessment of rebound and relapse following ecallantide treatment for acute attacks of hereditary angioedema. Allergy. 2012 Sep;67(9):1173-80. doi: 10.1111/j.1398-9995.2012.02864.x. Epub 2012 Jul 5.
citation: Riedl M, Campion M, Horn PT, Pullman WE. Response time for ecallantide treatment of acute hereditary angioedema attacks. Ann Allergy Asthma Immunol. 2010 Dec;105(6):430-436.e2. doi: 10.1016/j.anai.2010.09.005. Epub 2010 Oct 25.
citation: Cicardi M, Levy RJ, McNeil DL, Li HH, Sheffer AL, Campion M, Horn PT, Pullman WE. Ecallantide for the treatment of acute attacks in hereditary angioedema. N Engl J Med. 2010 Aug 5;363(6):523-31. doi: 10.1056/NEJMoa0905079.