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

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00456508
First Posted: April 5, 2007
Last Update Posted: July 27, 2016
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.
Information provided by (Responsible Party):
Shire
  Purpose
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.

Condition Intervention Phase
Hereditary Angioedema Drug: ecallantide Phase 3

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Open-label Patient Continuation of DX-88 (Ecallantide) for Acute Hereditary Angioedema Attacks

Resource links provided by NLM:


Further study details as provided by Shire:

Primary Outcome Measures:
  • 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.


Secondary Outcome Measures:
  • 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.


Enrollment: 147
Study Start Date: April 2007
Study Completion Date: September 2010
Primary Completion Date: June 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.
Drug: ecallantide
solution for SC injection, one 30 mg dose per HAE attack
Other Name: DX-88

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

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   10 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00456508


  Show 41 Study Locations
Sponsors and Collaborators
Shire
Investigators
Study Director: Bill Pullman, MD, PhD Dyax Corp.
  More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT00456508     History of Changes
Other Study ID Numbers: DX-88/19
First Submitted: April 4, 2007
First Posted: April 5, 2007
Results First Submitted: June 29, 2011
Results First Posted: December 28, 2012
Last Update Posted: July 27, 2016
Last Verified: June 2016

Additional relevant MeSH terms:
Angioedema
Angioedemas, Hereditary
Vascular Diseases
Cardiovascular Diseases
Urticaria
Skin Diseases, Vascular
Skin Diseases
Hypersensitivity, Immediate
Hypersensitivity
Immune System Diseases
Genetic Diseases, Inborn