Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema

September 27, 2012 updated by: Pharming Technologies B.V.

A Randomized, Placebo-controlled, Double-blind Phase III Study of the Efficacy and Safety of Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema

Hereditary angioedema ("HAE") is a genetic disorder characterized by sudden recurrent attacks of local swelling (angioedema). These attacks are often painful and disabling, and, in some cases, life-threatening. "HAE" is caused by mutations in the "C1INH" gene that leads to a decrease in the blood level of functional "C1INH". This multi-center study was designed to assess the safety and tolerability, efficacy and pharmacodynamics/ pharmacokinetics of recombinant human C1 inhibitor ("rhC1INH") in the treatment of acute hereditary angioedema attacks.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

A prospectively planned interim analysis will be performed on the double-blind data.

Study Type

Interventional

Enrollment (Actual)

75

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Netherlands
      • Leiden, Netherlands, 2300 AL
        • For information on sites, please contact Pharming Medical Affairs Deparment
  • Romania
      • Tirgu Mures, Romania, 541103
        • Emergency County Hospital, Internal Medicin Clinica, Allergology-Immunology Department

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

16 years and older (ADULT, OLDER_ADULT, CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Clear clinical and laboratory diagnosis of HAE
  • Baseline plasma level of functional C1INH of less than 50% of normal
  • Evidence for exacerbation or development of a severe abdominal, oro-facial/ pharyngeal/ laryngeal, genito-urinary and/or peripheral HAE attack

Exclusion Criteria:

  • Acquired angioedema
  • Pregnancy or breastfeeding
  • Participation in another clinical study within prior 3 months

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: RANDOMIZED
  • Interventional Model: PARALLEL
  • Masking: QUADRUPLE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
EXPERIMENTAL: 100 IU/kg "rhC1INH"
100 IU/kg recombinant human C1 inhibitor
Drug: recombinant human C1 inhibitor
IV
Other Names:
  • Ruconest
  • "rhC1INH"
  • conestat alfa
PLACEBO_COMPARATOR: Saline
Saline solution
Drug: Placebo
IV
Other Names:
  • saline
  • physiological salt solution

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Time to Beginning of Relief of Symptoms
Time Frame: up to 48 hours after study drug administration
The time to beginning of relief of symptoms has been assessed by using a patient-reported visual analogue scale ("VAS") ranging from 0 mm (no symptoms at all) to 100 mm (extremely disabling). Time to beginning of relief of symptoms at the location that showed first "VAS" score decrease of at least 20 mm from baseline score (t= 0 min) to the next assessment time-point). Assessment time-points were taken on pre-scheduled time-points after drug administration: baseline (0 minutes), 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 8 hours, 12 hours, 16 hours, 24 hours, 48 hours. Time to beginning of relief has been calculated as median time, by using the exact time-points on which each assessment was performed.
up to 48 hours after study drug administration

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Time to Minimal Symptoms
Time Frame: up to 48 hours after study drug administration
the time to minimal symptoms was the time to minimal symptoms for an attack, assessed using the Visual Analogue Scale ("VAS") score. Symptoms were said to be minimal when the "VAS" score at all locations was below 20 mm. Assessment time-points were: baseline (0 minutes), 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 8 hours, 12 hours, 16 hours, 24 hours, 48 hours. Time to minimal symptoms has been calculated by using the exact time-points on which each assessment was performed.
up to 48 hours after study drug administration

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Pharming Technologies B.V.

Investigators

  • Study Chair: Jan Nuijens, MD, PhD, Pharming Group N.V.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

June 1, 2004

Primary Completion (ACTUAL)

July 1, 2009

Study Completion (ACTUAL)

October 1, 2009

Study Registration Dates

First Submitted

December 1, 2005

First Submitted That Met QC Criteria

December 5, 2005

First Posted (ESTIMATE)

December 6, 2005

Study Record Updates

Last Update Posted (ESTIMATE)

October 2, 2012

Last Update Submitted That Met QC Criteria

September 27, 2012

Last Verified

September 1, 2012

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • C1 1304-01

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Hereditary Angioedema

Clinical Trials on Placebo

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Lebanon

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe