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Completed

NCT Number: NCT00456508

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

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.

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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 Centre, Ottawa, Ontario, Canada

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About this study

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.

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
  • 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

Treatment and study plan

ecallantide

Drug

solution for SC injection, one 30 mg dose per HAE attack

Other names: DX-88

Primary outcomes

  1. 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 outcomes

  1. 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.

  2. 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.

Sponsors and collaborators

Lead sponsor

Shire

Industry

Registry information

Official study title

Open-label Patient Continuation of DX-88 (Ecallantide) for Acute Hereditary Angioedema Attacks

Important dates

Study start
2007
Primary completion
2010
Study completion
2010
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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