Safety of Ruconest in 2-13 Year Old Hereditary Angioedema (HAE) Patients

March 28, 2024 updated by: Pharming Technologies B.V.

Open-label, Phase II, Single Arm Study to Evaluate the Safety, Immunogenicity, Pharmacokinetics and Efficacy of rhC1INH for the Treatment of Acute Attacks in Pediatric Patients With Hereditary Angioedema, From 2-13 Years of Age

This open-label study is being conducted to confirm the safety, pharmacokinetic profile and efficacy of Ruconest at a dose of 50 U/kg when used for the treatment of acute angioedema attacks in patients, from 2 up to and including 13 years of age.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

This study was an open-label, Phase 2, non-comparative, multinational, multicenter clinical study in pediatric patients from 2 up to and including 13 years of age, with a confirmed diagnosis of HAE. Patients were eligible for treatment with recombinant human C1-inhibitor (rhC1INH) if they presented to the clinic within 5 hours of onset with an acute attack of at least moderate severity without signs of spontaneous regression. Patients received rhC1INH at a dose of 50 U/kg body weight up to a maximum of 4200 U. The reconstituted solution was administered as a slow intravenous (iv) injection over approximately 5 minutes. The patients remained in hospital and were closely monitored in the study center for at least 4 hours after study medication administration.

Study Type

Interventional

Enrollment (Actual)

57

Phase

  • Phase 2

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

  • Czechia
    • Alej Svobody 80
      • Plzen, Alej Svobody 80, Czechia, 304 60 Plzen
        • UIA FN Plzen ( Institute of Immunology and Allergology), Faculty Hospital Plzen
    • V Úvalu 84
      • Prague, V Úvalu 84, Czechia, 150 06 Prague
        • University Hospital Motol, Institute of Immunology
  • Germany
      • Munich, Germany
        • Klinikum Rechts der Isar, Technical University Munich
    • Charitéplatz 1
      • Berlin, Charitéplatz 1, Germany, 10117
        • Charité - Universitätsmedizin Berlin
  • Hungary
    • Madarász Utca 22-24
      • Budapest, Madarász Utca 22-24, Hungary, 1131
        • Heim Pál Gyermekkórház, II. számú Gyermek Belgyógyászati Osztály
  • Israel
      • Haifa, Israel
        • Bnei Zion Hospital
      • Tel Hashomer, Israel
        • Sheba Medical Center
      • Tel-Aviv, Israel
        • Souraski Medical Center
  • Italy
      • Milan, Italy
        • Hospital Luigi Sacco
      • Salerno, Italy, 84131
        • Azienda Ospedaliera Universitaria S. Giovanni di Dio e Ruggi d'Aragona
  • North Macedonia
      • Skopje, North Macedonia, 1000
        • University Clinic Of Dermatology Skopje
  • Poland
      • Krakow, Poland
        • Pediatric Hospital
      • Lublin, Poland
        • Pediatric Hospital
  • Romania
      • Targu Mures, Romania
        • Mures County Clinical Hospital
  • Slovakia
    • Kollárova 2
      • Martin, Kollárova 2, Slovakia, 036 59 Martin
        • Klinika detí a dorastu, Univerzitna nemocnica Martin
  • United States
    • Oregon
      • Portland, Oregon, United States, 97015
        • Portland Clinical Research/AAIM Care, LLC

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

2 years to 13 years (Child)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • From 2 up to and including 13 years of age
  • Clinical and laboratory confirmed diagnosis of HAE (baseline C1INH activity <50% of normal)
  • Signed written Informed Consent Form (ICF)(parental permission) signed by the legal guardian(s)
  • Clinical symptoms of an acute HAE attack
  • Onset of eligible symptoms within 5 hours from the moment at which medical evaluation to determine eligibility has occurred
  • Attack severity moderate or greater, as rated by the investigator

Exclusion Criteria:

  • A diagnosis of acquired C1INH deficiency (AAE)
  • A medical history of allergy to rabbits or rabbit-derived products or positive anti-rabbit epithelium (dander) immunoglobuline E (IgE) test

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: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Recombinant Human C1 Inhibitor
Patients presented to the clinic within 5 hours of onset received rhC1INH 50 U/kg body weight up to a maximum of 4200 U.
Drug: rhC1INH
Patients up to 84 kg will receive one i.v. injection of Ruconest at a dose of 50 U/kg. The reconstituted solution should be administered as a slow i.v. injection over approximately 5 minutes. Patients of 84 kg body weight or greater will receive one i.v. injection of Ruconest at the dose of 4200 U (2 vials).
Other Names:
  • Ruconest

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Time to Beginning of Relief Based on Visual Analogue Scale (VAS) Was Defined as the Time, in Minutes, From Time of Infusion to the Beginning of Relief.
Time Frame: The assessment of the angioedema signs by the VAS and TEQ will be performed just before start of infusion, and at T30m, T1h, T2h, T4h, T8h and T24h after study medication infusion VAS score decrease of ≥ 20 mm from baseline.
Time to beginning of relief of symptoms that showed the response to treatment based on the overall VAS score decrease of ≥ 20 mm from baseline. Separate VAS forms will be given to express the current feelings considering the severity of angioedema symptoms for five possible anatomical locations. The form will be completed by placing vertical marks on each of the 100 mm horizontal lines. The Abdominal VAS measures the patient's perceptions relating to illness, pain, bloodedness, and nausea; the oro-pharyngeal-laryngeal (OPL) VAS measures illness, pain, swelling, breathing, speech, and swallowing; the facial VAS instrument measure illness, pain and swelling; the Peripheral VAS measures swelling, pain, and use of extremity; and the Urogenital VAS measures illness, pain, swelling, nausea, and urination. Time to beginning of relief will also be calculated based on the Investigator Score (IS) and Treatment Effect Questionnaire (TEQ).
The assessment of the angioedema signs by the VAS and TEQ will be performed just before start of infusion, and at T30m, T1h, T2h, T4h, T8h and T24h after study medication infusion VAS score decrease of ≥ 20 mm from baseline.

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Time to Minimal Symptoms Based on Patient's VAS Scores; Time From the Start of the Infusion of Study Medication to the First Assessment Time at Which the Overall Severity VAS Reaches a Value of Less Than 20 mm for All Locations
Time Frame: The assessment of the angioedema signs by the VAS and TEQ will be performed just before start of infusion, and at T30m, T1h, T2h, T4h, T8h and T24h after study medication infusion all locations where VAS Scores were recorded.
Time to minimal symptoms was defined as the time at which the Overall VAS score fell below 20 mm for all locations where VAS Scores were recorded. Separate VAS forms will be given to express the current feelings considering the severity of angioedema symptoms for five possible anatomical locations. The form will be completed by placing vertical marks on each of the 100 mm horizontal lines provided to answer each question related to the severity of symptoms and the patient's condition. The Abdominal VAS measures the patient's perceptions relating to illness, pain, bloodedness, and nausea; the OPL VAS measures illness, pain, swelling, breathing, speech, and swallowing; the facial VAS instrument measure illness, pain and swelling; the Peripheral VAS measures swelling, pain, and use of extremity; and the Urogenital VAS measures illness, pain, swelling, nausea, and urination.
The assessment of the angioedema signs by the VAS and TEQ will be performed just before start of infusion, and at T30m, T1h, T2h, T4h, T8h and T24h after study medication infusion all locations where VAS Scores were recorded.

Collaborators and Investigators

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

Sponsor

Pharming Technologies B.V.

Investigators

  • Study Director: Anurag Relan, MD, VP Clinical Research & Medical Affairs at Pharming

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 (Actual)

January 17, 2012

Primary Completion (Actual)

July 17, 2017

Study Completion (Actual)

July 17, 2017

Study Registration Dates

First Submitted

May 19, 2011

First Submitted That Met QC Criteria

May 24, 2011

First Posted (Estimated)

May 25, 2011

Study Record Updates

Last Update Posted (Actual)

March 29, 2024

Last Update Submitted That Met QC Criteria

March 28, 2024

Last Verified

March 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • C1 1209
  • 2011-000987-92 (EudraCT Number)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

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.

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