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Completed

NCT Number: NCT02586805

Efficacy and Safety Study of DX-2930 to Prevent Acute Angioedema Attacks in Patients With Type I and Type II HAE

This is a phase 3, multicenter, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of DX-2930 in preventing acute angioedema attacks in patients with Type I and Type II HAE.

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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

12 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Ottawa Allergy Research Corporation, Ottawa, Ontario, Canada

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Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Males and females 12 years of age or older at time of screening
  • Documented diagnosis of HAE, Type I or II
  • Baseline rate of at least 1 Investigator-confirmed HAE attack per 4 weeks
  • Adult subjects and caregivers of subjects under the age of 18 are willing and able to read, understand, and sign an informed consent form. Subjects age 12 to 17, whose caregiver provides informed consent, are willing and able to read, understand an dsign an assent form.
  • Males and femailes who are fertile and sexually active must adhere to contraception requirements.

Exclusion criteria

  • Concomitant diagnosis of another form of chronic, recurrent angioedema, such as acquired angioedema, idiopathic angioedema, or recurrent angioedema associated with urticaria.
  • Participation in a prior DX-2930 study
  • Treatment with any other investigational drug or exposure to an investigational device within 4 weeks prior screening
  • Exposure to angiotensin-converting enzyme (ACE) inhibitors or any estrogen-containing medications within 4 weeks prior to screening.
  • Exposure to androgens within 2 weeks prior to entering the run-in period.
  • Use of long-term prophylactic therapy for HAE within 2 weeks prior to entering the run-in period.
  • Use of short-term prophylaxis for HAE within 7 days prior to entering the run-in period.
  • Any of the following liver function test abnormalities: alanine aminotransferase (ALT) > 3x upper limit of normal, or aspartate aminotransferase (AST) > 3x upper limit of normal, or total bilirubin > 2x upper limit of normal (unless the bilirubin elevation is a result of Gilbert's syndrome).
  • Pregnancy or breastfeeding.

Treatment and study plan

DX-2930 - 300mg/2wk

Drug

300 mg DX-2930 administered every 2 weeks by subcutaneous injection.

DX-2930 - 300mg/4wk

Drug

300 mg DX-2930 administered every 4 weeks by subcutaneous injection. To maintain the study blind, subjects will be given placebo injections every other 2 weeks when they are not receiving drug.

DX-2930 - 150mg/4wk

Drug

150 mg DX-2930 administered every 4 weeks by subcutaneous injection. To maintain the study blind, subjects will be given placebo injections every other 2 weeks when they are not receiving drug.

Placebo

Drug

Placebo administered every 2 weeks by subcutaneous injection.

Primary outcomes

  1. Rate of Investigator Confirmed Hereditary Angioedema (HAE) Attacks During Treatment Period

    Time frame: From Day 0 to Day 182

    HAE attack was defined as a discrete episode during which the participant progressed from no angioedema to symptoms of angioedema. Rate of investigator confirmed HAE attacks was analyzed using a generalized linear model (GLM) for count data assuming a poisson distribution with a log link function and Pearson chi-square scaling of standard errors to account for potential overdispersion. The logarithm of time in days each subject was observed during the treatment period was used as an offset variable in the model.

Secondary outcomes

  1. Rate of Investigator Confirmed Hereditary Angioedema (HAE) Attack Requiring Acute Treatment

    Time frame: From Day 0 to Day 182

    HAE attack was defined as a discrete episode during which the participant progressed from no angioedema to symptoms of angioedema. Rate of investigator confirmed HAE attack was analyzed using the GLM for count data assuming a poisson distribution with a log link function and Pearson chi-square scaling of standard errors to account for potential overdispersion. The logarithm of time in days each subject was observed during the treatment period was used as an offset variable in the model.

  2. Rate of Moderate or Severe Investigator Confirmed Hereditary Angioedema (HAE) Attacks

    Time frame: From Day 0 to Day 182

    HAE attack was defined as a discrete episode during which the participant progressed from no angioedema to symptoms of angioedema. Moderate and severe investigator-confirmed HAE attacks were the attacks that were moderate or severe as per the HAE attack assessment and reporting procedures (HAARP) defined severity. The overall severity of attack was determined by the investigator using following definitions: mild (transient or mild discomfort), moderate (mild to moderate limitation in activity), severe (marked limitation in activity). Rate of moderate or severe investigator confirmed HAE attack was analyzed using the GLM for count data assuming a poisson distribution with a log link function and Pearson chi-square scaling of standard errors to account for potential overdispersion. The logarithm of time in days each subject was observed during the treatment period was used as an offset variable in the model.

  3. Rate of Investigator Confirmed Hereditary Angioedema (HAE) Attacks During Day 14 Through Day 182

    Time frame: From Day 14 to Day 182

    HAE attack was defined as a discrete episode during which the participant progressed from no angioedema to symptoms of angioedema. Rate of investigator confirmed HAE attacks during day 14 after study drug administration through day 182 was analyzed by the same poisson regression model as in the primary endpoint analysis.

Sponsors and collaborators

Lead sponsor

Shire

Industry

Collaborators

  • Dyax Corp.

Registry information

Official study title

HELP Study: A Multicenter, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study to Evaluate DX-2930 For Long-Term Prophylaxis Against Acute Attacks of Hereditary Angioedema (HAE)

Important dates

Study start
2016
Primary completion
2017
Study completion
2017
First posted
Oct 26, 2015
Registry last updated
Jun 2, 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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