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Completed

NCT Number: NCT01188564

Efficacy, Safety and Immunogenicity Study of Recombinant Human C1 Inhibitor for the Treatment of Acute HAE Attacks

This study is being conducted to confirm the efficacy, safety, and immunogenicity of recombinant human C1 inhibitor (rhC1INH) at a dose of 50 U/kg when used for the treatment of acute angioedema attacks in Hereditary Angioedema (HAE) patients.

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Key information

Conditions

Hereditary Angioedema 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

13 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

UMHAT "Tsaritsa Yoanna - ISUL"; Clinic of Ear-Nose-Throat Diseases, Sofia, Bulgaria

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About this study

HAE is characterized by recurrent localized angioedema caused by uncontrolled activation of the complement and contact systems due to a congenital deficiency of functional C1 inhibitor.

rhC1INH has been developed to offer a more widely available therapeutic alternative to the existing plasma-derived C1INH (pdC1INH) products that have been used in the treatment of acute angioedema attacks patients with HAE.

Patients who have qualified for enrollment in advance and who present to a study center within 5 hours of onset of an attack will be evaluated for eligibility. 75 eligible patients will be randomized (3:2) to receive an intravenous infusion of rhC1INH or saline in a double-blind fashion. Open-label rhC1INH may be provided as rescue medication to patients who do not experience the beginning of relief within 4 hours or who experience life-threatening oropharyngeal-laryngeal angioedema symptoms.

Any patient having received a randomized treatment will be allowed to receive treatment with rhC1INH in an open-label fashion for subsequent eligible attacks.

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Aged at least 13 years
  • Signed written informed consent
  • Clear clinical and laboratory diagnosis of HAE with baseline plasma level of functional C1INH of less than 50% of normal
  • Willingness and ability to comply with all protocol procedures
  • Clinical symptoms of an eligible HAE attack with onset less than 5 hours before the time of initial evaluation

Exclusion criteria

  • Medical history of allergy to rabbits or rabbit-derived products (including rhC1INH), or positive anti-rabbit dander IgE test (cut off >0.35 kU/L; ImmunoCap® assay; Phadia or equivalent).
  • A diagnosis of acquired C1INH deficiency (AAE)
  • Pregnancy, or breastfeeding, or current intention to become pregnant
  • Treatment with any investigational drug in the past 30 days
  • Known or suspected addiction to drug and/or alcohol abuse
  • Suspicion for an alternate explanation of the symptoms other than acute HAE attack

Treatment and study plan

rhC1INH

Drug

One i.v. injection of rhC1INH at the dose of 50 U/kg, for patients up to 84 kg; one i.v. injection of rhC1INH at the dose of 4200U (2 vials) for patients of 84 kg body weight or greater.

Placebo (saline)

Drug

One i.v. injection of saline (NaCl 0.9% w/v), equivalent in volume to the active treatment

Primary outcomes

  1. Time to Beginning of Relief of Symptoms

    Time frame: Patients observed for 24 hours

    Time to beginning of relief is the time lapsed from the beginning of the infusion of study medication to the beginning of a beneficial effect based on patient's responses to the Treatmetn Effect Questionnaire (TEQ) for the primary attack location. The beginning of relief is defined as the first timepoint at which

    • The patient reports any of the following answers for TEQ question 1: "A little better", "Better" or "Much better"; and;
    • The patient reports the following answer for TEQ question 2: "Yes"; and,
    • There is persistence in improvement at the next assessment time, i.e.either the same or a better response to Question 1 and "Yes" to Question 2.

Secondary outcomes

  1. Time to Minimal Symptoms

    Time frame: 24 hours

    The key secondary efficacy endpoint was the time to minimal symptoms at all locations. The time to achieving minimal symptoms was defined as an answer of "Yes" to TEQ question 3.

Sponsors and collaborators

Lead sponsor

Pharming Technologies B.V.

Industry

Registry information

Official study title

A Phase III Randomized, Double-blind, Placebo-controlled Study With an Open-label Extension Evaluating the Efficacy, Safety and Immunogenicity of Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks of Angioedema in Patients With HAE

Important dates

Study start
2011
Primary completion
2012
Study completion
2013
First posted
Aug 25, 2010
Registry last updated
Aug 7, 2015

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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