Study to Evaluate the Clinical Efficacy and Safety of Subcutaneously Administered C1 Esterase Inhibitor for the Prevention of Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema
A Phase 3, Randomized, Double-blind, Placebo-controlled, Two-period, Three-sequence, Partial Crossover Study to Evaluate the Efficacy and Safety of Subcutaneous Administration of 2000 IU of C1 Esterase Inhibitor [Human] Liquid for Injection for the Prevention of Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema
1 other identifier
interventional
75
7 countries
27
Brief Summary
The purpose of this study is to assess the efficacy and safety of subcutaneous administration of a liquid formulation of C1 esterase inhibitor for the prevention of angioedema attacks in adolescent and adult subjects with hereditary angioedema.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Nov 2015
27 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
October 20, 2015
CompletedFirst Posted
Study publicly available on registry
October 23, 2015
CompletedStudy Start
First participant enrolled
November 1, 2015
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 24, 2017
CompletedStudy Completion
Last participant's last visit for all outcomes
July 24, 2017
CompletedResults Posted
Study results publicly available
November 28, 2018
CompletedJune 8, 2021
May 1, 2021
1.7 years
October 20, 2015
July 17, 2018
May 14, 2021
Conditions
Outcome Measures
Primary Outcomes (1)
Time-Normalized Number of Attacks (NNA) for Participants During a Treatment Period
The angioedema attacks were recorded in the electronic patient diary. The investigator completed a separate angioedema eCRF for each attack based on the review of the patients diary. Time-normalized number of angioedema attacks was expressed as the number of attacks per month (ie, 30.4 days) of exposure. NNA=30.4 \* (number of attacks during treatment period) / (days of treatment period).
Weeks 1 to 14 for treatment period 1 and 2
Secondary Outcomes (26)
Proportion of Participants Meeting at Least a 50% Reduction in NNA (Normalized Number of Angioedema Attacks) During the Experimental Injection Treatment Period Relative to the Placebo Period.
Weeks 1 to 14 for treatment period 1 and 2
Time-Normalized Number of Attacks (NNA) for Participants During Each Treatment Period Excluding the First 2 Weeks.
Weeks 3 to 14 for treatment period 1 and 2
Proportion of Participants Meeting at Least a 50% Reduction in NNA (Normalized Number of Angioedema Attacks) During the Experimental Injection Treatment Period Relative to the Placebo Period Excluding the First 2 Weeks of Each Treatment Period.
Weeks 3 to 14 for treatment period 1 and 2
Proportion of Participants Meeting at Least a 50% Reduction in NNA (Normalized Number of Angioedema Attacks) During the Experimental Injection Treatment Period Relative to the Pre-treatment Assessment.
Weeks 1 to 14 for treatment period 1 and 2
Cumulative Attack Severity
Weeks 1 to 14 for treatment period 1 and 2
- +21 more secondary outcomes
Study Arms (3)
Experimental/Placebo
EXPERIMENTALSubjects will be randomized to receive C1 Esterase Inhibitor in the 1st Treatment period and then switch to Placebo in the 2nd treatment period.
Placebo/Experimental
EXPERIMENTALSubjects will be randomized to receive a placebo treatment in the 1st Treatment period and then switch to receive C1 Esterase Inhibitor in the 2nd treatment period.
Experimental/ Experimental
EXPERIMENTALSubjects will be randomized and receive C1 Esterase Inhibitor in both 1st as well as the 2nd treatment period
Interventions
C1 Esterase Inhibitor \[Human\] Liquid administered Subcutaneously as specified on specified days
Eligibility Criteria
Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.
Sponsors & Collaborators
- Shirelead
Study Sites (27)
Medical Research of Arizona
Scottsdale, Arizona, 85251, United States
AIRE Medical of Los Angeles
Santa Monica, California, 90404, United States
Bay Area Allergy
Walnut Creek, California, 94598, United States
IMMUNOe Research Centers
Centennial, Colorado, 80112, United States
Asthma and Allergy Associates PC
Colorado Springs, Colorado, 80907, United States
Atlanta Allergy and Asthma Clinic
Marietta, Georgia, 30060, United States
Institute For Asthma and Allergy
Chevy Chase, Maryland, 20815, United States
Washington University
St Louis, Missouri, 63141, United States
Allergy Asthma and Immunology
Fair Lawn, New Jersey, 07410, United States
Allergy Treatment Center of New Jersey
Iselin, New Jersey, 08830, United States
Clinical Research of Charlotte
Charlotte, North Carolina, 28277, United States
Bernstein Clinical Research Center Inc
Cincinnati, Ohio, 45231, United States
Clinical Research Solutions
Middleburg Heights, Ohio, 44130, United States
Allergy Clinic of Tulsa
Tulsa, Oklahoma, 74133, United States
AARA Research Center
Dallas, Texas, 75231, United States
Premier Clinical Research
Spokane, Washington, 99202, United States
McMaster University Medical Center
Hamilton, Ontario, L8N 325, Canada
Ottawa Allergy Research Corporation
Ottawa, Ontario, K1Y 4G2, Canada
Hämophilie Zentrum Rhein Main GmbH
Mörfelden-Walldorf, 64546, Germany
Semmelweis Egyetem
Budapest, 1125, Hungary
Chaim Sheba Medical Center
Ramat Gan, 52621, Israel
Tel Aviv Sourasky Medical Center
Tel Aviv, 64239, Israel
MediQuest Clinical Research Center
Târgu Mureş, 540072, Romania
Hospital Universitario Vall d'Hebron
Barcelona, 8035, Spain
Hospital Universitario La Paz
Madrid, 28046, Spain
Hospital Universitario Virgen del Rocio
Seville, 41013, Spain
Hospital Universitari i Politecnic La Fe de Valencia
Valencia, 46026, Spain
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: 36326435DERIVEDLumry WR, Martinez-Saguer I, Yang WH, Bernstein JA, Jacobs J, Moldovan D, Riedl MA, Johnston DT, Li HH, Tang Y, Schranz J, Lu P, Vardi M, Farkas H; SAHARA study group. Fixed-Dose Subcutaneous C1-Inhibitor Liquid for Prophylactic Treatment of C1-INH-HAE: SAHARA Randomized Study. J Allergy Clin Immunol Pract. 2019 May-Jun;7(5):1610-1618.e4. doi: 10.1016/j.jaip.2019.01.021. Epub 2019 Jan 23.
PMID: 30682573DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Study Director
- Organization
- Shire
Study Officials
- STUDY DIRECTOR
Study Director
Takeda
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- DOUBLE
- Who Masked
- PARTICIPANT, INVESTIGATOR
- Purpose
- PREVENTION
- Intervention Model
- CROSSOVER
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 20, 2015
First Posted
October 23, 2015
Study Start
November 1, 2015
Primary Completion
July 24, 2017
Study Completion
July 24, 2017
Last Updated
June 8, 2021
Results First Posted
November 28, 2018
Record last verified: 2021-05
Data Sharing
- IPD Sharing
- Will share
- Shared Documents
- STUDY PROTOCOL, SAP, ICF, CSR
- Access Criteria
- IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.