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A Phase 2 HAE Prophylaxis Study With Recombinant Human C1 Inhibitor

Primary Purpose

Hereditary Angioedema

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Recombinant human C1 inhibitor
Placebo
Sponsored by
Pharming Technologies B.V.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional prevention trial for Hereditary Angioedema focused on measuring HAE, Hereditary Angioedema, Prophylaxis

Eligibility Criteria

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

Inclusion Criteria:

Patients fulfilling the following criteria at Screening are eligible for participation in the study:

  1. Age 13 years or older
  2. Laboratory confirmed diagnosis of HAE
  3. A history of frequent HAE attacks (at least 4 attacks per month across a minimum of 3 consecutive months).
  4. Female patients of childbearing potential who are sexually active must be willing to use an acceptable form of contraception.
  5. Provided written informed consent (and written assent for minors)
  6. Willingness and ability to comply with all protocol procedures

Exclusion Criteria:

Patients who meet any of the following criteria at Screening are to be excluded from study participation:

  1. Patients with medical history of allergy to rabbits or rabbit-derived products (including rhC1INH)
  2. Diagnosis of acquired angioedema (AAE)
  3. Patients who are pregnant, or breastfeeding, or are currently intending to become pregnant
  4. Treatment with any investigational drug in the past 30 days
  5. Patients with any condition or treatment that, in the opinion of the Investigator, might interfere with the evaluation of study objectives
  6. Patients currently treated with angiotensin-converting enzyme (ACE) inhibitors

Sites / Locations

  • University of South Florida Asthma, Allergy and Immunology Clinical Research Unit
  • University of South Florida, Asthma, Allergy & Immunology Clinical Research Unit
  • Washington University Division of Allergy and Immunology
  • Baker Allergy, Asthma and Dermatology Research Center
  • Ottawa Allergy Research Corp
  • Faculty Hospital by St. Anna Brno, Department of clinical Immunology and Allergology
  • Azienda Ospedaliera Universitaria Luigi Sacco Di Milano
  • PHI University Clinic of Dermatology
  • SC Centrul Clinic Mediquest SRL
  • Clinical Center Serbia

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Placebo Comparator

Arm Label

rhC1INH twice weekly

rhC1INH once weekly

Placebo (Saline) twice weekly

Arm Description

rhC1INH administered twice weekly

rhC1INH administered once weekly

Placebo (Saline) administered twice weekly

Outcomes

Primary Outcome Measures

Number of HAE Attacks
Average number of HAE attacks normalized to a 28 day period

Secondary Outcome Measures

Number of Participants With Adverse Events
Number of participants that experienced Treatment Emergent Adverse Events observed in safety population
Percentage of Participants Achieving at Least 50% Reduction in Number of Attacks
Percentage of participants achieving at least 50% reduction in the number of attacks normalized to a 28-day period as compared to the placebo treatment period

Full Information

First Posted
September 16, 2014
Last Updated
November 7, 2017
Sponsor
Pharming Technologies B.V.
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1. Study Identification

Unique Protocol Identification Number
NCT02247739
Brief Title
A Phase 2 HAE Prophylaxis Study With Recombinant Human C1 Inhibitor
Official Title
A Phase 2 Multicenter, Randomized, Double-Blind, Placebo-Controlled, 3-Period Crossover Study to Evaluate the Efficacy and Safety of Recombinant Human C1 Inhibitor in the Prophylaxis of Angioedema Attacks in Patients With Hereditary Angioedema (HAE)
Study Type
Interventional

2. Study Status

Record Verification Date
November 2017
Overall Recruitment Status
Completed
Study Start Date
December 2014 (undefined)
Primary Completion Date
May 2016 (Actual)
Study Completion Date
September 2016 (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
Primary Objective: To evaluate the efficacy of recombinant human C1 inhibitor (rhC1INH) in the prophylaxis of angioedema attacks in patients with HAE Secondary Objective: To evaluate the safety and immunogenicity of recombinant human C1 inhibitor (rhC1INH) in the prophylaxis of angioedema attacks in patients with HAE
Detailed Description
Study Design: This is a multi-center, randomized, double-blind, placebo-controlled, 3-period crossover study of rhC1INH in prophylaxis of angioedema attacks in patients with HAE. Medical screening (clinical and laboratory parameters) will be performed and patient medical history specific to HAE attacks will be collected to assess eligibility. Each patient will receive three 4 week periods of treatment twice weekly.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hereditary Angioedema
Keywords
HAE, Hereditary Angioedema, Prophylaxis

7. Study Design

Primary Purpose
Prevention
Study Phase
Phase 2
Interventional Study Model
Crossover Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
32 (Actual)

8. Arms, Groups, and Interventions

Arm Title
rhC1INH twice weekly
Arm Type
Experimental
Arm Description
rhC1INH administered twice weekly
Arm Title
rhC1INH once weekly
Arm Type
Experimental
Arm Description
rhC1INH administered once weekly
Arm Title
Placebo (Saline) twice weekly
Arm Type
Placebo Comparator
Arm Description
Placebo (Saline) administered twice weekly
Intervention Type
Biological
Intervention Name(s)
Recombinant human C1 inhibitor
Other Intervention Name(s)
rhC1INH, Ruconest, Conestat alfa
Intervention Type
Other
Intervention Name(s)
Placebo
Other Intervention Name(s)
Saline
Primary Outcome Measure Information:
Title
Number of HAE Attacks
Description
Average number of HAE attacks normalized to a 28 day period
Time Frame
28 days
Secondary Outcome Measure Information:
Title
Number of Participants With Adverse Events
Description
Number of participants that experienced Treatment Emergent Adverse Events observed in safety population
Time Frame
20 weeks
Title
Percentage of Participants Achieving at Least 50% Reduction in Number of Attacks
Description
Percentage of participants achieving at least 50% reduction in the number of attacks normalized to a 28-day period as compared to the placebo treatment period
Time Frame
28 days
Other Pre-specified Outcome Measures:
Title
Immunogenicity
Description
Number of participants analyzed for neutralizing C1INH-specific antibodies and neutralizing rhC1INH-specific antibodies after confirmed anti-C1INH and anti rhC1INH IgM or IgG antibodies
Time Frame
20 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
13 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Patients fulfilling the following criteria at Screening are eligible for participation in the study: Age 13 years or older Laboratory confirmed diagnosis of HAE A history of frequent HAE attacks (at least 4 attacks per month across a minimum of 3 consecutive months). Female patients of childbearing potential who are sexually active must be willing to use an acceptable form of contraception. Provided written informed consent (and written assent for minors) Willingness and ability to comply with all protocol procedures Exclusion Criteria: Patients who meet any of the following criteria at Screening are to be excluded from study participation: Patients with medical history of allergy to rabbits or rabbit-derived products (including rhC1INH) Diagnosis of acquired angioedema (AAE) Patients who are pregnant, or breastfeeding, or are currently intending to become pregnant Treatment with any investigational drug in the past 30 days Patients with any condition or treatment that, in the opinion of the Investigator, might interfere with the evaluation of study objectives Patients currently treated with angiotensin-converting enzyme (ACE) inhibitors
Facility Information:
Facility Name
University of South Florida Asthma, Allergy and Immunology Clinical Research Unit
City
Tampa
State/Province
Florida
ZIP/Postal Code
33613
Country
United States
Facility Name
University of South Florida, Asthma, Allergy & Immunology Clinical Research Unit
City
Tampa
State/Province
Florida
ZIP/Postal Code
33613
Country
United States
Facility Name
Washington University Division of Allergy and Immunology
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63141
Country
United States
Facility Name
Baker Allergy, Asthma and Dermatology Research Center
City
Lake Oswego
State/Province
Oregon
ZIP/Postal Code
97035
Country
United States
Facility Name
Ottawa Allergy Research Corp
City
Ottawa
State/Province
Ontario
ZIP/Postal Code
K1G6C6
Country
Canada
Facility Name
Faculty Hospital by St. Anna Brno, Department of clinical Immunology and Allergology
City
Brno
ZIP/Postal Code
65691
Country
Czechia
Facility Name
Azienda Ospedaliera Universitaria Luigi Sacco Di Milano
City
Milan
ZIP/Postal Code
20157
Country
Italy
Facility Name
PHI University Clinic of Dermatology
City
Skopje
ZIP/Postal Code
1000
Country
Macedonia, The Former Yugoslav Republic of
Facility Name
SC Centrul Clinic Mediquest SRL
City
Sângeorgiu de Mureş
State/Province
Mures
ZIP/Postal Code
547530
Country
Romania
Facility Name
Clinical Center Serbia
City
Belgrade
Country
Serbia

12. IPD Sharing Statement

Citations:
PubMed Identifier
36326435
Citation
Beard N, Frese M, Smertina E, Mere P, Katelaris C, Mills K. Interventions for the long-term prevention of hereditary angioedema attacks. Cochrane Database Syst Rev. 2022 Nov 3;11(11):CD013403. doi: 10.1002/14651858.CD013403.pub2.
Results Reference
derived
PubMed Identifier
28754491
Citation
Riedl MA, Grivcheva-Panovska V, Moldovan D, Baker J, Yang WH, Giannetti BM, Reshef A, Andrejevic S, Lockey RF, Hakl R, Kivity S, Harper JR, Relan A, Cicardi M. Recombinant human C1 esterase inhibitor for prophylaxis of hereditary angio-oedema: a phase 2, multicentre, randomised, double-blind, placebo-controlled crossover trial. Lancet. 2017 Sep 30;390(10102):1595-1602. doi: 10.1016/S0140-6736(17)31963-3. Epub 2017 Jul 25.
Results Reference
derived

Learn more about this trial

A Phase 2 HAE Prophylaxis Study With Recombinant Human C1 Inhibitor

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