A Phase 2 HAE Prophylaxis Study With Recombinant Human C1 Inhibitor
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)
1 other identifier
interventional
32
7 countries
10
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
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_2
Started Dec 2014
10 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
September 16, 2014
CompletedFirst Posted
Study publicly available on registry
September 25, 2014
CompletedStudy Start
First participant enrolled
December 1, 2014
CompletedPrimary Completion
Last participant's last visit for primary outcome
May 1, 2016
CompletedStudy Completion
Last participant's last visit for all outcomes
September 1, 2016
CompletedResults Posted
Study results publicly available
December 8, 2017
CompletedDecember 8, 2017
November 1, 2017
1.4 years
September 16, 2014
July 26, 2017
November 7, 2017
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Number of HAE Attacks
Average number of HAE attacks normalized to a 28 day period
28 days
Secondary Outcomes (2)
Number of Participants With Adverse Events
20 weeks
Percentage of Participants Achieving at Least 50% Reduction in Number of Attacks
28 days
Other Outcomes (1)
Immunogenicity
20 weeks
Study Arms (3)
rhC1INH twice weekly
EXPERIMENTALrhC1INH administered twice weekly
rhC1INH once weekly
EXPERIMENTALrhC1INH administered once weekly
Placebo (Saline) twice weekly
PLACEBO COMPARATORPlacebo (Saline) administered twice weekly
Interventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (10)
University of South Florida Asthma, Allergy and Immunology Clinical Research Unit
Tampa, Florida, 33613, United States
University of South Florida, Asthma, Allergy & Immunology Clinical Research Unit
Tampa, Florida, 33613, United States
Washington University Division of Allergy and Immunology
St Louis, Missouri, 63141, United States
Baker Allergy, Asthma and Dermatology Research Center
Lake Oswego, Oregon, 97035, United States
Ottawa Allergy Research Corp
Ottawa, Ontario, K1G6C6, Canada
Faculty Hospital by St. Anna Brno, Department of clinical Immunology and Allergology
Brno, 65691, Czechia
Azienda Ospedaliera Universitaria Luigi Sacco Di Milano
Milan, 20157, Italy
PHI University Clinic of Dermatology
Skopje, 1000, North Macedonia
SC Centrul Clinic Mediquest SRL
Sângeorgiu de Mureş, Mureș County, 547530, Romania
Clinical Center Serbia
Belgrade, Serbia
Related Publications (2)
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.
PMID: 36326435DERIVEDRiedl 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.
PMID: 28754491DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- VP Clinical Research & Medical Affairs
- Organization
- Pharming Group NV
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- LTE60
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- PREVENTION
- Intervention Model
- CROSSOVER
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
September 16, 2014
First Posted
September 25, 2014
Study Start
December 1, 2014
Primary Completion
May 1, 2016
Study Completion
September 1, 2016
Last Updated
December 8, 2017
Results First Posted
December 8, 2017
Record last verified: 2017-11