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

Primary Purpose

Hereditary Angioedema

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
rhC1INH
Placebo (Saline)
Sponsored by
Pharming Technologies B.V.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hereditary Angioedema focused on measuring Hereditary Angioedema, HAE, Angioedema, Recombinant C1 Inhibitor, rhC1INH

Eligibility Criteria

13 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Allergy, Asthma & Immunology, Assoc, Ltd.
  • Allergy and Asthma Institute of the Valley
  • UCLA Department of Medicine Division of Clinical Immunology, David Geffen School of Medicine
  • USF Asthma, Allergy and Immunology Clinical Research Unit
  • Family Allergy and Asthma Center
  • Institute for Asthma and Allergy, P.C.
  • Asthma & Allergy Center - Washington University School of Medicine
  • University of Cincinnati Physicians, Inc.
  • Optimed Research, LTD
  • Baker Allergy, Asthma and Dermatology Research Center, LLC
  • Pennsylvania State- Milton S. Hershey Medical Center
  • AARA Research Center
  • University of Texas - Medical Branch
  • Allergy, Asthma & Immunology Clinic, P.A.
  • Marycliff Allergy Specialists
  • UMHAT "Tsaritsa Yoanna - ISUL"; Clinic of Ear-Nose-Throat Diseases
  • Ottawa Allergy Research Corp.
  • Semmelweis University Faculty of Medicine, III Department of Internal Medicine
  • Allergy, Immunology & Angioedema Center,
  • Bnei-Zion Medical Centre, Clinical Immunology and Allergy Division
  • Ospedale Luigi Sacco, Azienda Ospedaliera - Polo Universitario II Divisione di Medicina Interna
  • P.H.U. Clinic for Dermatology, Medical University Skopje, Unit of Allergology and Clinical Immunology
  • Szpital Uniwersytecki w Krakowie, Oddział Kliniczny Klinik Chorób Wewnętrznych, Poradnia Alergologiczna
  • Spitalul Clinic Judeţean Mureş Secţia Clinică Medicină Internă, Compartimentul de Alergologie şi Imunologie
  • Clinic for Immunology and Allergology
  • Allergy Diagnostic & Clinical Research Unit University of Cape Town Lung Institute
  • Wits Health Consortium (Pty) Ltd - Wits Donald Gordon Medical Centre

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

rhC1INH

Placebo (Saline)

Arm Description

Outcomes

Primary Outcome Measures

Time to Beginning of Relief of Symptoms
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 Outcome Measures

Time to Minimal Symptoms
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.

Full Information

First Posted
August 24, 2010
Last Updated
August 3, 2015
Sponsor
Pharming Technologies B.V.
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1. Study Identification

Unique Protocol Identification Number
NCT01188564
Brief Title
Efficacy, Safety and Immunogenicity Study of Recombinant Human C1 Inhibitor for the Treatment of Acute HAE Attacks
Official 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
Study Type
Interventional

2. Study Status

Record Verification Date
July 2015
Overall Recruitment Status
Completed
Study Start Date
January 2011 (undefined)
Primary Completion Date
December 2012 (Actual)
Study Completion Date
March 2013 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Pharming Technologies B.V.

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
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.
Detailed Description
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hereditary Angioedema
Keywords
Hereditary Angioedema, HAE, Angioedema, Recombinant C1 Inhibitor, rhC1INH

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
75 (Actual)

8. Arms, Groups, and Interventions

Arm Title
rhC1INH
Arm Type
Experimental
Arm Title
Placebo (Saline)
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
rhC1INH
Intervention Description
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.
Intervention Type
Drug
Intervention Name(s)
Placebo (Saline)
Intervention Description
One i.v. injection of saline (NaCl 0.9% w/v), equivalent in volume to the active treatment
Primary Outcome Measure Information:
Title
Time to Beginning of Relief of Symptoms
Description
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.
Time Frame
Patients observed for 24 hours
Secondary Outcome Measure Information:
Title
Time to Minimal Symptoms
Description
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.
Time Frame
24 hours

10. Eligibility

Sex
All
Minimum Age & Unit of Time
13 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Facility Information:
Facility Name
Allergy, Asthma & Immunology, Assoc, Ltd.
City
Scottsdale
State/Province
Arizona
ZIP/Postal Code
85251
Country
United States
Facility Name
Allergy and Asthma Institute of the Valley
City
Granada Hills
State/Province
California
ZIP/Postal Code
91344
Country
United States
Facility Name
UCLA Department of Medicine Division of Clinical Immunology, David Geffen School of Medicine
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
USF Asthma, Allergy and Immunology Clinical Research Unit
City
Tampa
State/Province
Florida
ZIP/Postal Code
33613
Country
United States
Facility Name
Family Allergy and Asthma Center
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30342
Country
United States
Facility Name
Institute for Asthma and Allergy, P.C.
City
Chevy Chase
State/Province
Maryland
ZIP/Postal Code
20815
Country
United States
Facility Name
Asthma & Allergy Center - Washington University School of Medicine
City
St. Louis
State/Province
Missouri
ZIP/Postal Code
63141
Country
United States
Facility Name
University of Cincinnati Physicians, Inc.
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45267
Country
United States
Facility Name
Optimed Research, LTD
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43235
Country
United States
Facility Name
Baker Allergy, Asthma and Dermatology Research Center, LLC
City
Lake Oswego
State/Province
Oregon
ZIP/Postal Code
97035
Country
United States
Facility Name
Pennsylvania State- Milton S. Hershey Medical Center
City
Hershey
State/Province
Pennsylvania
ZIP/Postal Code
17033
Country
United States
Facility Name
AARA Research Center
City
Dallas
State/Province
Texas
ZIP/Postal Code
75231
Country
United States
Facility Name
University of Texas - Medical Branch
City
Galveston
State/Province
Texas
ZIP/Postal Code
77555
Country
United States
Facility Name
Allergy, Asthma & Immunology Clinic, P.A.
City
Irving
State/Province
Texas
ZIP/Postal Code
75063
Country
United States
Facility Name
Marycliff Allergy Specialists
City
Spokane
State/Province
Washington
ZIP/Postal Code
99204
Country
United States
Facility Name
UMHAT "Tsaritsa Yoanna - ISUL"; Clinic of Ear-Nose-Throat Diseases
City
Sofia
ZIP/Postal Code
1527
Country
Bulgaria
Facility Name
Ottawa Allergy Research Corp.
City
Ottawa
State/Province
Ontario
ZIP/Postal Code
KIY 4G2
Country
Canada
Facility Name
Semmelweis University Faculty of Medicine, III Department of Internal Medicine
City
Budapest
ZIP/Postal Code
H-1125
Country
Hungary
Facility Name
Allergy, Immunology & Angioedema Center,
City
Tel Hashomer
State/Province
Ramat Gan
ZIP/Postal Code
52621
Country
Israel
Facility Name
Bnei-Zion Medical Centre, Clinical Immunology and Allergy Division
City
Haifa
ZIP/Postal Code
31048
Country
Israel
Facility Name
Ospedale Luigi Sacco, Azienda Ospedaliera - Polo Universitario II Divisione di Medicina Interna
City
Milan
ZIP/Postal Code
20157
Country
Italy
Facility Name
P.H.U. Clinic for Dermatology, Medical University Skopje, Unit of Allergology and Clinical Immunology
City
Skopje
ZIP/Postal Code
1000
Country
Macedonia, The Former Yugoslav Republic of
Facility Name
Szpital Uniwersytecki w Krakowie, Oddział Kliniczny Klinik Chorób Wewnętrznych, Poradnia Alergologiczna
City
Krakow
ZIP/Postal Code
31-531
Country
Poland
Facility Name
Spitalul Clinic Judeţean Mureş Secţia Clinică Medicină Internă, Compartimentul de Alergologie şi Imunologie
City
Târgu-Mureş
ZIP/Postal Code
540103
Country
Romania
Facility Name
Clinic for Immunology and Allergology
City
Belgrade
ZIP/Postal Code
11000
Country
Serbia
Facility Name
Allergy Diagnostic & Clinical Research Unit University of Cape Town Lung Institute
City
Mowbray
ZIP/Postal Code
7700
Country
South Africa
Facility Name
Wits Health Consortium (Pty) Ltd - Wits Donald Gordon Medical Centre
City
Parktown
ZIP/Postal Code
2193
Country
South Africa

12. IPD Sharing Statement

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

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