NCT00262080

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
91

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Dec 2005

Shorter than P25 for phase_3

Geographic Reach
1 country

1 active site

Status
completed

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

December 5, 2005

Completed
1 day until next milestone

First Posted

Study publicly available on registry

December 6, 2005

Completed
25 days until next milestone

Study Start

First participant enrolled

December 31, 2005

Completed
Same day until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 31, 2005

Completed
1.2 years until next milestone

Study Completion

Last participant's last visit for all outcomes

February 28, 2007

Completed
3.2 years until next milestone

Results Posted

Study results publicly available

May 11, 2010

Completed
Last Updated

June 11, 2021

Status Verified

June 1, 2021

Enrollment Period

Same day

First QC Date

December 5, 2005

Results QC Date

December 30, 2009

Last Update Submit

June 3, 2021

Conditions

Hereditary Angioedema (HAE)

Outcome Measures

Primary Outcomes (1)

  • Treatment Outcome Score at 4 Hours Post-Dose

    Treatment Outcome Score (TOS) is a validated, comprehensive measure of symptom response to treatment. At 4 hours , patient assessment of response characterized by their change from baseline in symptom severity and collected by anatomic site of attack involvement, was recorded on a categorical scale (significant improvement \[100\] to significant worsening \[-100\]). The response at each anatomic site was weighted by baseline severity and then the weighted scores across all involved sites were averaged to calculate the TOS. Clinically meaningful improvement was indicated by a TOS of 30 or higher.

    4 hours post-dose (DOUBLE-BLIND PART)

Secondary Outcomes (2)

  • Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hours Post-dose

    baseline, 4 hours post-dose (DOUBLE-BLIND PART)

  • Time to Significant Improvement in Overall Response

    4 hours post-dose (DOUBLE-BLIND PART)

Other Outcomes (4)

  • Number of Patients With Symptom Complexes of Treated Attack at Baseline(DOUBLE-BLIND PART)

    Baseline

  • Treatment Outcome Score at 4 Hours Post-Dose Over Multiple Treatment Episodes

    4 hours post-dose (REPEAT-DOSING PART)

  • Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hours Post-dose Over Multiple Treatment Episodes

    baseline, 4 hours post-dose (REPEAT-DOSING PART)

  • +1 more other outcomes

Study Arms (2)

DX-88 (ecallantide)

EXPERIMENTAL

DX-88 (ecallantide) 30 mg given as three 10 mg/mL subcutaneous injections.

Drug: ecallantide

Placebo

PLACEBO COMPARATOR

Phosphate Buffer Saline (PBS), pH 7.0 given as 3 subcutaneous injections.

Drug: Phosphate Buffer Saline (PBS),

Interventions

ecallantideDRUG

dose of 30 mg (10 mg/ml) given as 3 subcutaneous injections.

Also known as: DX-88
DX-88 (ecallantide)
Phosphate Buffer Saline (PBS),DRUG

given as three 1mL subcutaneous injections.

Placebo

Eligibility Criteria

Age10 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Institute for Asthma and Allergy

Wheaton, Maryland, 20902, United States

Location

Related Publications (6)

  • 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.

    PMID: 24712435DERIVED

  • 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.

    PMID: 23878046DERIVED

  • 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.

    PMID: 23548529DERIVED

  • 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.

    PMID: 22765833DERIVED

  • 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.

    PMID: 21130380DERIVED

  • 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.

    PMID: 20818887DERIVED

MeSH Terms

Conditions

Angioedemas, Hereditary

Interventions

ecallantide

Condition Hierarchy (Ancestors)

AngioedemaVascular DiseasesCardiovascular DiseasesHereditary Complement Deficiency DiseasesPrimary Immunodeficiency DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesUrticariaSkin Diseases, VascularSkin DiseasesSkin and Connective Tissue DiseasesHypersensitivity, ImmediateHypersensitivityImmune System DiseasesImmunologic Deficiency Syndromes

Results Point of Contact

Title
Study Director
Organization
Shire
ClinicalTransparency@shire.com
+1 866 842 5335

Study Officials

  • Study Director

    Takeda

    STUDY DIRECTOR

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
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 5, 2005

First Posted

December 6, 2005

Study Start

December 31, 2005

Primary Completion

December 31, 2005

Study Completion

February 28, 2007

Last Updated

June 11, 2021

Results First Posted

May 11, 2010

Record last verified: 2021-06

Locations

US(1)