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Safety and Efficacy Study of Repeated Doses of DX-88 (Ecallantide) to Treat Attacks of Hereditary Angioedema (HAE)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00456508
Recruitment Status : Completed
First Posted : April 5, 2007
Results First Posted : December 28, 2012
Last Update Posted : February 20, 2018
Sponsor:
Information provided by (Responsible Party):
Shire

Tracking Information
First Submitted Date  ICMJE April 4, 2007
First Posted Date  ICMJE April 5, 2007
Results First Submitted Date  ICMJE June 29, 2011
Results First Posted Date  ICMJE December 28, 2012
Last Update Posted Date February 20, 2018
Study Start Date  ICMJE April 1, 2007
Actual Primary Completion Date June 1, 2010   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 27, 2012)
Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hrs Post Dosing [ Time Frame: 4 hrs post dose after every episode ]
Mean Symptom Complex Severity (MSCS) score is a validated point-in-time measure of symptom severity. At baseline and 4 hrs, patients rated the severity on a categorical scale (0=normal, 1=mild, 2=moderate, 3=severe) for symptoms at each affected anatomical location. Ratings were averaged to obtain the MSCS score. A decrease in MSCS score reflected an improvement in symptoms; clinically meaningful improvement was indicated by a reduction in the score of 0.30 or more.
Original Primary Outcome Measures  ICMJE
 (submitted: April 4, 2007)
Change in symptom severity as reported by the patient
Change History Complete list of historical versions of study NCT00456508 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: November 27, 2012)
  • Treatment Outcome Score (TOS) at 4 Hrs Post Dosing, Based on the Patient Assessment of Baseline Severity of Symptoms [ Time Frame: 4 hrs post dose after every episode ]
    The Treatment Outcome Score (TOS)is a validated measure of response to therapy. Response assessment for each symptom complex (internal head/neck, stomach/GI, genital/buttocks, external head/neck or cutaneous) was to be weighted based on the severity of symptom complexes at baseline. Severity assessment at baseline was rated on a categorical scale (1=mild, 2=moderate, 3=severe) for symptoms at each affected symptom complex. Response assessment of each symptom complex post-dosing relative to baseline used a scale (100=significant improvement, 50=improvement, 0=same). The weighted values were used to calculate the composite TOS. A TOS greater than 0 denotes an improvement in symptoms compared with baseline severity.
  • Time to Significant Improvement [ Time Frame: 15 min - 4 hrs post dose after every episode ]
    Time to significant improvement in overall response based on the period from 15 minutes after dosing through 4 hrs post dosing. Significant improvement was defined as a response of "a lot better or resolved" in the overall response assessment.
Original Secondary Outcome Measures  ICMJE
 (submitted: April 4, 2007)
  • Patient reported symptom change
  • Time to improvement
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety and Efficacy Study of Repeated Doses of DX-88 (Ecallantide) to Treat Attacks of Hereditary Angioedema (HAE)
Official Title  ICMJE Open-label Patient Continuation of DX-88 (Ecallantide) for Acute Hereditary Angioedema Attacks
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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Hereditary Angioedema (HAE)
Intervention  ICMJE Drug: ecallantide
solution for SC injection, one 30 mg dose per HAE attack
Other Name: DX-88
Study Arms  ICMJE Experimental: DX-88 (ecallantide)
DX-88 (ecallantide) Patients were treated with DX-88 (ecallantide) when they experienced an HAE attack. 30 mg dose of ecallantide given via 3 SC injections; a second 30 mg dose can be administered if needed. Patients were to be assessed until 4 hrs post-dose. Patients were asked to return for 3 follow-up visits: 7 days, 28 days and 90 days post-dose.
Intervention: Drug: ecallantide
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: November 27, 2012)
147
Original Enrollment  ICMJE
 (submitted: April 4, 2007)
150
Actual Study Completion Date  ICMJE September 1, 2010
Actual Primary Completion Date June 1, 2010   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 10 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Canada,   Jordan,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00456508
Other Study ID Numbers  ICMJE DX-88/19
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Shire
Study Sponsor  ICMJE Shire
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Bill Pullman, MD, PhD Dyax Corp.
PRS Account Shire
Verification Date January 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP