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Completed

NCT Number: NCT00457015

Efficacy Study of DX-88 (Ecallantide) to Treat Acute Attacks of Hereditary Angioedema (HAE)

The purpose of this study is to evaluate the efficacy and safety of DX-88 (ecallantide) versus placebo in the treatment of moderate to severe acute attacks of hereditary angioedema.

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Key information

Conditions

Hereditary Angioedema (HAE) Angioedema Angioedemas, Hereditary Cardiovascular Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hereditary Complement Deficiency Diseases Hypersensitivity Hypersensitivity, Immediate Immune System Diseases Immunologic Deficiency Syndromes Primary Immunodeficiency Diseases Skin Diseases Skin Diseases, Vascular Skin and Connective Tissue Diseases Urticaria Vascular Diseases

Age range

10 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Allergy and Asthma Research Center, Ottawa, Ontario, Canada

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About this study

This is a randomized placebo-controlled trial.

The study is designed to assess the efficacy and safety of 30 mg subcutaneous ecallantide versus placebo in the treatment of moderate to severe acute attacks of hereditary angioedema. This study is conducted under Special Protocol Assessment with the FDA and is designed to provide pivotal efficacy data on 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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 10 years of age or older
  • Executed informed consent
  • Documented diagnosis of HAE (Type I or II)
  • Presentation at the site within 8 hours of patient recognition of an moderate to severe HAE acute attack

Exclusion criteria

  • Receipt of an investigational drug or device, within 30 days prior to study treatment
  • Receipt of non-investigational C1-INH within 7 days of treatment
  • Receipt of DX-88 (ecallantide) within 3 days prior to study treatment
  • Diagnosis of acquired angioedema (AAE), estrogen-dependent angioedema or drug-induced angioedema (including angiotensin-converting enzyme inhibitor induced angioedema)
  • Pregnancy or breastfeeding

Treatment and study plan

ecallantide

Drug

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

Other names: DX-88

Phosphate Buffer Saline (PBS), pH 7.0

Drug

given as three 1mL subcutaneous injections.

Primary outcomes

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

    Time frame: baseline, 4 hours post-dose

    The Mean Symptom Complex Severity (MSCS) score is a validated, comprehensive point-in-time measure of symptom severity. At baseline and 4 hours, 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 outcomes

  1. Treatment Outcome Score at 4 Hours Post-Dose

    Time frame: 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; best value]to significant worsening [-100; worst value]). Clinically meaningful improvement was indicated by a TOS of 30 or higher.

  2. Patients With Significant Improvement in Overall Response

    Time frame: 4 hours post-dose

    Patients were to be asked to perform an overall response assessment at intervals during the first 4 hours post-dose. Assessments were to be made relative to baseline (ie, immediately before initial dosing) using a 5-category scale. Categories were: significant improvement = "a lot better or resolved"; improvement = "a little better"; same = response unchanged; worsening = "a little worse"; significant worsening = "a lot worse". Significant improvement is the first time that a patient responded to the overall response assessment as "a lot better or resolved."

  3. Patients With a Successful Response at 4 Hours Post-dosing, Based on the Change From Baseline in the MSCS Score

    Time frame: baseline, 4 hours post-dosing

    A successful response was defined as improvement in existing laryngeal symptom complex,stabilization of an existing peripheral symptom complex, or a change from baseline in the MSCS score at 4 hours of at least -1.0.

  4. Proportion of Patients Maintaining a Significant Improvement in Overall Response Through 24 Hours

    Time frame: 24 hours post-dosing

    Maintenance of significant improvement was defined as achieving and maintaining a significant improvement in overall response through 24 hours after dosing. Patient response categories were: significant improvement = "a lot better or resolved"; improvement = "a little better"; same = response unchanged; worsening = "a little worse"; significant worsening = "a lot worse".

Sponsors and collaborators

Lead sponsor

Shire

Industry

Registry information

Official study title

EDEMA4: A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Assess the Efficacy and Safety of DX-88 (Ecallantide) for the Treatment of Acute Attacks of Hereditary Angioedema

Important dates

Study start
2007
Primary completion
2008
Study completion
2008
First posted
Apr 5, 2007
Registry last updated
Jun 8, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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