Hereditary Angioedema (HAE)NCT01059526
Observational Safety Study for KALBITOR (Ecallantide) in the Treatment of Acute Attacks of Hereditary Angioedema
An observational study of Hereditary Angioedema (HAE), run by Shire at 40 sites in United States.
Trial summary
The objective of this study is to evaluate the formation of antibodies, the occurence of allergic reactions, and the risk of hypercoagulability and hypocoagulability in patients treated with KALBITOR (ecallantide). Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Hereditary Angioedema (HAE)
- What is being tested?
- ecallantide
- How many people
- 81 participants
- Where
- 40 sitesUnited States
- Study dates
- 1 Feb 2010to 1 Jun 2014
- Phase
- Observational
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: patients with hereditary angioedema, either naive or non-naive to KALBITOR (ecallantide) prior to enrollment in the study
Who may be able to join
- Patients indicated per the approved product label for KALBITOR
- Patient or guardian is able to understand and sign the informed consent form
- Patient is willing and able to undergo a skin test procedure at screening (baseline)
Who cannot join
- Patient contraindicated per the approved product label for KALBITOR
- Patient confirmed pregnancy or active breastfeeding
- Any other condition that, in the opinion of the Investigator, may compromise the safety or compliance of the patient or would preclude the patient from successful completion of the study
Trial locations and contacts
40 sites in 1 country.
40 sites in 1 country
United States40 sites
University of Michigan Health System Allergy Specialty Clinic
Ann Arbor, Michigan, 48105
Allergy Partners of Western North Carolina
Asheville, North Carolina, 28801
Sunrise Clinical Research
Bell Gardens, California, 90201
Brigham and Women's Hospital
Chestnut Hill, Massachusetts, 02467
Institute for Asthma and Allergy, P.C.
Chevy Chase, Maryland, 20815
University Consultants in Allergry and Immunology
Chicago, Illinois, 60612
Department of Internal Medicine, University of Cincinnati -MSB
Cincinnati, Ohio, 45267-0563
Asthma and Allergy Institute of Michigan
Clinton Township, Michigan, 48038
Brookstone Clinical Research Center
Columbus, Georgia, 31904
Optimed Research, LTD
Columbus, Ohio, 43235
AARA Research Center
Dallas, Texas, 75231
Deaconess Clinic Downtown
Evansville, Indiana, 47713
Allergy & Asthma Institute of the Valley
Granada Hills, California, 91344
Penn State University - Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033-0850
Allergy Treatment Center of New Jersey
Iselin, New Jersey, 08830
Ochsner Health System - Allergy, Asthma and Immunology Department
Jefferson, Louisiana, 70121
Little Rock Allergry and Asthma Clinical Research Center
Little Rock, Arkansas, 72205
Unidversity of California, Los Angeles David Geffen School of Medicine
Los Angeles, California, 90095-1680
Clinical Research Specialists
Metairie, Louisiana, 70006
Winthrop University Hospital
Mineola, New York, 11501
Muncie Allergy Ctr
Muncie, Indiana, 47304
Children's Hospital of the King's Daughters
Norfolk, Virginia, 23507
Medical Research Associates of CNY
North Syracuse, New York, 13212
705 West LaVeta
Orange, California, 92868
Kansas City Allergy and Asthma Associates
Overland Park, Kansas, 66210
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224
University of Nevada School of Medicine - Department of Pediatrics
Reno, Nevada, 89503
University of Utah, Department of Dermatology
Salt Lake City, Utah, 84132
Specialty Medical Clinic And Research Center
Sanford, North Carolina, 27330
Saint Louis University School of Medicine
St Louis, Missouri, 63104
Washington University School of Medicine
St Louis, Missouri, 63110
Puget Sound Allergy, Asthma and Immunology
Tacoma, Washington, 98405
University of South Florida Asthma
Tampa, Florida, 33613
Study site
The Bronx, New York, 10465
Reynolds Clinic
Toledo, Ohio, 43615
Toledo Institute of Clinical Research
Toledo, Ohio, 43617
Allergy Clinic of the Tulsa, Inc.
Tulsa, Oklahoma, 74133
Study site
Virginia Beach, Virginia, 23452
Allergy & Asthma Clinical Research Inc.
Walnut Creek, California, 94598
Center for Allergy, Asthma & Immunology
Waterbury, Connecticut, 06708
About this study
Official title: A Phase 4, Long-Term Observational Safety Study to Evaluate Immunogenicity and Hypersensitivity With Exposure to KALBITOR (Ecallantide) for the Treatment of Acute Attacks of HAE
The objective of this study is to evaluate the formation of antibodies, the occurence of allergic reactions, and the risk of hypercoagulability and hypocoagulability in patients treated with KALBITOR (ecallantide).
Read the detailed description
The objective of this study is to evaluate immunogenicity and hypersensitivity upon exposure to KALBITOR, in particular:
- Determine the rate of anaphylaxis and Type I hypersensitivity reactions upon exposure to KALBITOR.
- Determine the rate of seroconversion to anti-ecallantide antibodies upon exposure to KALBITOR.
- Determine the rate of adverse events related to disordered coagulation (hypercoagulability and hypocoagulability) upon exposure to KALBITOR.
- Conditions
- Hereditary Angioedema (HAE)
- Being tested
- ecallantide
- Keywords
- HAE
- Medical subject headings
- Angioedemas, Hereditary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ecallantideDrug
Also called Kalbitor
30 mg SC
| Study group | Role | What they receive |
|---|---|---|
| Patients naive to KALBITOR | HAE patients that have not been treated with KALBITOR (ecallantide) prior to enrollment in the study Receives: Drug: ecallantide | |
| Patients non- naive to KALBITOR | HAE patients that have been treated with KALBITOR prior to enrollment in the study Receives: Drug: ecallantide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Occurrence of Anaphylaxis or Other Adverse Events Suggestive of Hypersensitivity
Based on medical review of multiple preferred terms for treatment emergent adverse events (TEAEs) suggestive of Type 1 hypersensitivity; terms included adverse drug reaction, anaphylaxis, anaphylactic reaction, anaphylactoid reaction, hypersensitivity, erythema, flushing, hot flush, pharyngeal edema, laryngeal edema, pruritus, pruritus generalized, rash, rash erythematous, rhinitis allergic, rhinorrhea, throat irritation, urticaria, urticaria localized, dyspnea, and wheezing. Records of patients with any of these TEAE referred terms were reviewed further to assess potential hypersensitivity reactions, considering factors such as timing of TEAEs in relationship to dose (ie, occurred within 24 hours after start of KALBITOR treatment), accompanying symptoms, Investigator causality assessment (ie, reported as possibly, probably, or definitely related to study drug), and any other available clinical information. Anaphylaxis subset determined based on criteria established by the NIAID.
Measured: 12 months after first treatment
Occurrence of Seroconversion to Anti-ecallantide Antibodies Upon Exposure to KALBITOR.
Seroconversion is the development of detectable specific antibodies in the blood serum. Serum was tested for development of antibodies (irrespective of immunoglobulin class) against ecallantide at screening and at all safety evaluations. Positive results were to undergo a confirmatory test. Confirmed positive samples were further titered. Patients who developed an antibody response were evaluated for the development of neutralizing antibodies. Patients also had their serum analyzed for IgE-specific antibodies to ecallantide at screening and during safety evaluations. Positive results underwent a confirmatory test. Confirmed positive samples were further titered.
Measured: 12 months after first treatment
Occurrence of Adverse Events Related to Disordered Coagulation (Hypercoagulability and Hypocoagulability) Upon Exposure to KALBITOR
Events of ecchymosis, hemorrhage, petechiae, spontaneous hemorrhage, hematoma, gastrointestinal bleeding, hemorrhagic stroke and any other term indicative of a bleeding event or increased tendency for bleeding were reviewed to determine the occurrence of hypocoagulability. Events of clotting, thrombosis, pulmonary embolism, vaso-occlusive stroke, myocardial infarction, and any other term indicative of a clotting event or increased risk of clotting were reviewed to determine the occurrence of hypercoagulability.
Measured: 12 months after first treatment
Secondary outcomes (1)
Overall Patient Response Assessment
The Overall Response Assessment was to be completed every 30 minutes after treatment until patient discharge. Patients evaluated their response to treatment as "a lot better or resolved," "a little better," "the same," "a little worse," or "a lot worse." The data presented is based on the best response achieved following a single dose of KALBITOR (Dose A) for the first HAE treatment episode. Responses of "a lot better or resolved" and "a little better" were combined to form a category of "Better." Similarly, "a little worse" and "a lot worse" were combined to form a category of "Worse." Patients treated in a clinic (study site) could have been discharged after an hour and hence may have only had 2 post-treatment evaluations (30 and 60 minutes); response assessments may not have been consistently provided when patients were treated at an alternate site outside of the study site.
Measured: within 4 hours post dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Feb 2010 (estimated)
- Study start1 Feb 2010
- Primary completion1 Sep 2013
- Study completion1 Jun 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Hereditary Angioedema (HAE)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 81 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01059526
- Sponsor's protocol ID
- DX-88/24
- Status last verified
- May 2021
- First submitted
- 28 Jan 2010
- Last updated
- 8 Jun 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 8 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 4, Long-Term Observational Safety Study to Evaluate Immunogenicity and Hypersensitivity With Exposure to KALBITOR (Ecallantide) for the Treatment of Acute Attacks of HAE
- ClinicalTrials.gov ID
- NCT01059526
- Acronym / sponsor ID
- DX-88/24
- Conditions
- Hereditary Angioedema (HAE)
- Keywords
- HAE
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 81 participants (actual)
- Lead sponsor
- ShireIndustry (pharma, biotech, devices)
- Study start
- 1 Feb 2010 (actual)
- Primary completion
- 1 Sep 2013 (actual)
- Study completion
- 1 Jun 2014 (actual)
- First posted
- 1 Feb 2010 (estimated)
- Results
- Posted 6 Oct 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The objective of this study is to evaluate the formation of antibodies, the occurence of allergic reactions, and the risk of hypercoagulability and hypocoagulability in patients treated with KALBITOR (ecallantide).
Detailed description
The objective of this study is to evaluate immunogenicity and hypersensitivity upon exposure to KALBITOR, in particular:
- Determine the rate of anaphylaxis and Type I hypersensitivity reactions upon exposure to KALBITOR.
- Determine the rate of seroconversion to anti-ecallantide antibodies upon exposure to KALBITOR.
- Determine the rate of adverse events related to disordered coagulation (hypercoagulability and hypocoagulability) upon exposure to KALBITOR.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Patients naive to KALBITOR | – | Drug: ecallantideHAE patients that have not been treated with KALBITOR (ecallantide) prior to enrollment in the study |
| Patients non- naive to KALBITOR | – | Drug: ecallantideHAE patients that have been treated with KALBITOR prior to enrollment in the study |
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- patients with hereditary angioedema, either naive or non-naive to KALBITOR (ecallantide) prior to enrollment in the study
Key inclusion criteria (3 listed)
- Patients indicated per the approved product label for KALBITOR
- Patient or guardian is able to understand and sign the informed consent form
- Patient is willing and able to undergo a skin test procedure at screening (baseline)
Key exclusion criteria (3 listed)
- Patient contraindicated per the approved product label for KALBITOR
- Patient confirmed pregnancy or active breastfeeding
- Any other condition that, in the opinion of the Investigator, may compromise the safety or compliance of the patient or would preclude the patient from successful completion of the study
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ecallantideAlso: Kalbitor30 mg SC | Drug | Patients naive to KALBITOR, Patients non- naive to KALBITOR |
Primary outcome
Occurrence of Anaphylaxis or Other Adverse Events Suggestive of Hypersensitivity
Time frame: 12 months after first treatment
Based on medical review of multiple preferred terms for treatment emergent adverse events (TEAEs) suggestive of Type 1 hypersensitivity; terms included adverse drug reaction, anaphylaxis, anaphylactic reaction, anaphylactoid reaction, hypersensitivity, erythema, flushing, hot flush, pharyngeal edema, laryngeal edema, pruritus, pruritus generalized, rash, rash erythematous, rhinitis allergic, rhinorrhea, throat irritation, urticaria, urticaria localized, dyspnea, and wheezing. Records of patients with any of these TEAE referred terms were reviewed further to assess potential hypersensitivity reactions, considering factors such as timing of TEAEs in relationship to dose (ie, occurred within 24 hours after start of KALBITOR treatment), accompanying symptoms, Investigator causality assessment (ie, reported as possibly, probably, or definitely related to study drug), and any other available clinical information. Anaphylaxis subset determined based on criteria established by the NIAID.
Occurrence of Seroconversion to Anti-ecallantide Antibodies Upon Exposure to KALBITOR.
Time frame: 12 months after first treatment
Seroconversion is the development of detectable specific antibodies in the blood serum. Serum was tested for development of antibodies (irrespective of immunoglobulin class) against ecallantide at screening and at all safety evaluations. Positive results were to undergo a confirmatory test. Confirmed positive samples were further titered. Patients who developed an antibody response were evaluated for the development of neutralizing antibodies. Patients also had their serum analyzed for IgE-specific antibodies to ecallantide at screening and during safety evaluations. Positive results underwent a confirmatory test. Confirmed positive samples were further titered.
Occurrence of Adverse Events Related to Disordered Coagulation (Hypercoagulability and Hypocoagulability) Upon Exposure to KALBITOR
Time frame: 12 months after first treatment
Events of ecchymosis, hemorrhage, petechiae, spontaneous hemorrhage, hematoma, gastrointestinal bleeding, hemorrhagic stroke and any other term indicative of a bleeding event or increased tendency for bleeding were reviewed to determine the occurrence of hypocoagulability. Events of clotting, thrombosis, pulmonary embolism, vaso-occlusive stroke, myocardial infarction, and any other term indicative of a clotting event or increased risk of clotting were reviewed to determine the occurrence of hypercoagulability.
1 secondary outcome
Overall Patient Response Assessment
Time frame: within 4 hours post dose
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jun 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Study Director study director
Takeda
40 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hereditary Angioedema (HAE), run by Shire at 40 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 Jun 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 40 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 6 Oct 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Occurrence of Anaphylaxis or Other Adverse Events Suggestive of Hypersensitivity (time frame: 12 months after first treatment). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Jun 2021.