Hereditary Angioedema (HAE)Phase 3NCT00456508
Safety and Efficacy Study of Repeated Doses of DX-88 (Ecallantide) to Treat Attacks of Hereditary Angioedema (HAE)
A Phase 3 study testing ecallantide in people with Hereditary Angioedema (HAE), run by Shire at 41 sites in 3 countries.
Trial summary
The purpose of this study is to evaluate the efficacy and safety of repeated doses of ecallantide in the treatment of acute attacks of hereditary angioedema and to allow HAE patients continued access to ecallantide. Read more about this study
- Who is it for?
- Ages 10 years and older
- Conditions the study is for
- Hereditary Angioedema (HAE)
- What is being tested?
- ecallantide
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 147 participants
- Where
- 41 sitesin 3 countries
- Study dates
- 1 Apr 2007to 1 Sep 2010
- Phase
- Phase 3
- 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 10 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 10 years of age or older
- Documented diagnosis of HAE (Type I or II)
- Willing and able to give informed consent
- Acute HAE attack at time of presentation
Who cannot join
- Receipt of an investigational drug or device, within 30 days prior to study treatment, other than DX-88 (ecallantide)
- Pregnancy or breastfeeding
- Receipt of non-investigational C1-INH or DX-88 within 72 hours of treatment
- Patients eligible for current, ongoing clinical trial in which DX 88 (ecallantide) is offered
Trial locations and contacts
41 sites in 3 countries.
41 sites in 3 countries
United States39 sites
Allergy Partners of Albuquerque
Albuquerque, New Mexico, 87109
Allergy Partners of Western North Carolina
Asheville, North Carolina, 28801
Family Allergy & Asthma Center, PC
Atlanta, Georgia, 30342
Alta Bates Summit Comprehensive Cancer Center
Berkeley, California, 94704
Highlands Allergy and Asthma Center, PC
Bristol, Tennessee, 37620
The Paull Allergy and Asthma Clinic, P.A
Bryan, Texas, 77802
Brigham and Women's Hospital
Chestnut Hill, Massachusetts, 02467
University Consultants in Allergy and Immunology
Chicago, Illinois, 60612
University of Cincinnati, Division of Internal Medicine
Cincinnati, Ohio, 45267
Asthma and Allergy Institutes of Michigan
Clinton Township, Michigan, 48038
Asthma and Allergy Associates, P.C.
Colorado Springs, Colorado, 80907
Allergy Center of Brookstone
Columbus, Georgia, 31904
Optimed Research, LLC
Columbus, Ohio, 43235
Pacific Coast Allergy
Crescent City, California, 95531
AARA Research Center
Dallas, Texas, 75231
Jacob Offenberger
Granada Hills, California, 91344
Penn State Milton S Hershey Medical Center
Hershey, Pennsylvania, 17033
Nevada Access to Research and Education Society
Las Vegas, Nevada, 89102
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
Little Rock Allergy & Asthma Clinic
Little Rock, Arkansas, 72205
UCLA David Geffen School of Medicine, Department of Medicine
Los Angeles, California, 90095-1680
University of Miami, General Clinical Research Center
Miami, Florida, 33136
Winthrop University Hospital
Mineola, New York, 11501
Muncie Allergy Center
Muncie, Indiana, 47304
Christiana Hospital, Christiana Care Health Services
Newark, Delaware, 19718
UMDNJ- New Jersey Medical School
Newark, New Jersey, 07103
Clinical Research Associates of Tidewater
Norfolk, Virginia, 23507
Kansas City Allergy & Asthma
Overland Park, Kansas, 66210
Asthma Allergy and Pulmonary Associates
Philadelphia, Pennsylvania, 19107
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15213
University of Nevada School of Medicine
Reno, Nevada, 89503
University of Utah Health Sciences Center
Salt Lake City, Utah, 84132-2409
Aaron Davis
Scottsdale, Arizona, 85251
Puget Sound Allergy, Asthma, & Immunology
Tacoma, Washington, 98405
University of South Florida, Asthma, Allergy and Immunology Clinical Research Unit
Tampa, Florida, 33613
Georgetown University Medical Center, Georgetown University Hospital
Washington D.C., District of Columbia, 20007
Roberson Allergy and Asthma
West Palm Beach, Florida, 33401
Institute for Asthma and Allergy
Wheaton, Maryland, 20902
Respiratory Medicine Research Institute of Michigan, PLC
Ypsilanti, Michigan, 48197
About this study
Official title: Open-label Patient Continuation of DX-88 (Ecallantide) for Acute Hereditary Angioedema Attacks
The purpose of this study is to evaluate the efficacy and safety of repeated doses of ecallantide in the treatment of acute attacks of hereditary angioedema and to allow HAE patients continued access to ecallantide. In addition, patients enrolled in DX-88/20 (EDEMA4) trial will be followed up and treated for subsequent attacks in this trial.
Read the detailed description
This is an open label trial.
The study is designed to assess the efficacy and safety of 30 mg subcutaneous ecallantide in the treatment of acute attacks of hereditary angioedema. This study is designed to provide efficacy and safety data on repeated use of ecallantide. These data are intended to support the marketing authorization of ecallantide in the treatment of acute attacks of hereditary angioedema. Efficacy and safety of ecallantide will be evaluated in this study.
- Conditions
- Hereditary Angioedema (HAE)
- Being tested
- ecallantide
- 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 DX-88
solution for SC injection, one 30 mg dose per HAE attack
| Study group | Role | What they receive |
|---|---|---|
| DX-88 (ecallantide) | Experimental | DX-88 (ecallantide) Patients were treated with DX-88 (ecallantide) when they experienced an HAE attack. 30 mg dose of ecallantide given via 3 SC injections; a second 30 mg dose can be administered if needed. Patients were to be assessed until 4 hrs post-dose. Patients were asked to return for 3 follow-up visits: 7 days, 28 days and 90 days post-dose. Receives: Drug: ecallantide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hrs Post Dosing
Mean Symptom Complex Severity (MSCS) score is a validated point-in-time measure of symptom severity. At baseline and 4 hrs, patients rated the severity on a categorical scale (0=normal, 1=mild, 2=moderate, 3=severe) for symptoms at each affected anatomical location. Ratings were averaged to obtain the MSCS score. A decrease in MSCS score reflected an improvement in symptoms; clinically meaningful improvement was indicated by a reduction in the score of 0.30 or more.
Measured: 4 hrs post dose after every episode
Secondary outcomes (2)
Treatment Outcome Score (TOS) at 4 Hrs Post Dosing, Based on the Patient Assessment of Baseline Severity of Symptoms
The Treatment Outcome Score (TOS)is a validated measure of response to therapy. Response assessment for each symptom complex (internal head/neck, stomach/GI, genital/buttocks, external head/neck or cutaneous) was to be weighted based on the severity of symptom complexes at baseline. Severity assessment at baseline was rated on a categorical scale (1=mild, 2=moderate, 3=severe) for symptoms at each affected symptom complex. Response assessment of each symptom complex post-dosing relative to baseline used a scale (100=significant improvement, 50=improvement, 0=same). The weighted values were used to calculate the composite TOS. A TOS greater than 0 denotes an improvement in symptoms compared with baseline severity.
Measured: 4 hrs post dose after every episode
Time to Significant Improvement
Time to significant improvement in overall response based on the period from 15 minutes after dosing through 4 hrs post dosing. Significant improvement was defined as a response of "a lot better or resolved" in the overall response assessment.
Measured: 15 min - 4 hrs post dose after every episode
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Apr 2007 (estimated)
- Study start1 Apr 2007
- Primary completion1 Jun 2010
- Study completion1 Sep 2010
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.
- Publications (4)Papers the sponsor has linked to this study.
- 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
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 147 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
- NCT00456508
- Sponsor's protocol ID
- DX-88/19
- Status last verified
- May 2021
- First submitted
- 4 Apr 2007
- Last updated
- 8 Jun 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Rubinstein E, Stolz LE, Sheffer AL, Stevens C, Bousvaros A. Abdominal attacks and treatment in hereditary angioedema with C1-inhibitor deficiency. BMC Gastroenterol. 2014 Apr 9;14:71. doi: 10.1186/1471-230X-14-71.
PubMed 24712435MacGinnitie AJ, Davis-Lorton M, Stolz LE, Tachdjian R. Use of ecallantide in pediatric hereditary angioedema. Pediatrics. 2013 Aug;132(2):e490-7. doi: 10.1542/peds.2013-0646. Epub 2013 Jul 22.
PubMed 23878046Sheffer AL, MacGinnitie AJ, Campion M, Stolz LE, Pullman WE. Outcomes after ecallantide treatment of laryngeal hereditary angioedema attacks. Ann Allergy Asthma Immunol. 2013 Mar;110(3):184-188.e2. doi: 10.1016/j.anai.2012.12.007. Epub 2013 Jan 5.
PubMed 23548529Li HH, Campion M, Craig TJ, Soteres DF, Riedl M, Lumry WR, MacGinnitie AJ, Shea EP, Bernstein JA. Analysis of hereditary angioedema attacks requiring a second dose of ecallantide. Ann Allergy Asthma Immunol. 2013 Mar;110(3):168-72. doi: 10.1016/j.anai.2012.12.004. Epub 2013 Jan 8.
PubMed 23548526
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
- Open-label Patient Continuation of DX-88 (Ecallantide) for Acute Hereditary Angioedema Attacks
- ClinicalTrials.gov ID
- NCT00456508
- Acronym / sponsor ID
- DX-88/19
- Conditions
- Hereditary Angioedema (HAE)
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 147 participants (actual)
- Lead sponsor
- ShireIndustry (pharma, biotech, devices)
- Study start
- 1 Apr 2007 (actual)
- Primary completion
- 1 Jun 2010 (actual)
- Study completion
- 1 Sep 2010 (actual)
- First posted
- 5 Apr 2007 (estimated)
- Results
- Posted 28 Dec 2012
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to evaluate the efficacy and safety of repeated doses of ecallantide in the treatment of acute attacks of hereditary angioedema and to allow HAE patients continued access to ecallantide. In addition, patients enrolled in DX-88/20 (EDEMA4) trial will be followed up and treated for subsequent attacks in this trial.
Detailed description
This is an open label trial.
The study is designed to assess the efficacy and safety of 30 mg subcutaneous ecallantide in the treatment of acute attacks of hereditary angioedema. This study is designed to provide efficacy and safety data on repeated use of ecallantide. These data are intended to support the marketing authorization of ecallantide in the treatment of acute attacks of hereditary angioedema. Efficacy and safety of ecallantide will be evaluated in this study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| DX-88 (ecallantide) | Experimental | Drug: ecallantideDX-88 (ecallantide) Patients were treated with DX-88 (ecallantide) when they experienced an HAE attack. 30 mg dose of ecallantide given via 3 SC injections; a second 30 mg dose can be administered if needed. Patients were to be assessed until 4 hrs post-dose. Patients were asked to return for 3 follow-up visits: 7 days, 28 days and 90 days post-dose. |
Who may qualify
- Ages
- 10 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 10 years of age or older
- Documented diagnosis of HAE (Type I or II)
- Willing and able to give informed consent
- Acute HAE attack at time of presentation
Key exclusion criteria (4 listed)
- Receipt of an investigational drug or device, within 30 days prior to study treatment, other than DX-88 (ecallantide)
- Pregnancy or breastfeeding
- Receipt of non-investigational C1-INH or DX-88 within 72 hours of treatment
- Patients eligible for current, ongoing clinical trial in which DX 88 (ecallantide) is offered
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: DX-88solution for SC injection, one 30 mg dose per HAE attack | Drug | DX-88 (ecallantide) |
Primary outcome
Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hrs Post Dosing
Time frame: 4 hrs post dose after every episode
Mean Symptom Complex Severity (MSCS) score is a validated point-in-time measure of symptom severity. At baseline and 4 hrs, patients rated the severity on a categorical scale (0=normal, 1=mild, 2=moderate, 3=severe) for symptoms at each affected anatomical location. Ratings were averaged to obtain the MSCS score. A decrease in MSCS score reflected an improvement in symptoms; clinically meaningful improvement was indicated by a reduction in the score of 0.30 or more.
2 secondary outcomes
Treatment Outcome Score (TOS) at 4 Hrs Post Dosing, Based on the Patient Assessment of Baseline Severity of Symptoms
Time frame: 4 hrs post dose after every episode
Time to Significant Improvement
Time frame: 15 min - 4 hrs post dose after every episode
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.
- Derived from this study: PubMed 24712435Rubinstein E, Stolz LE, Sheffer AL, Stevens C, Bousvaros A. Abdominal attacks and treatment in hereditary angioedema with C1-inhibitor deficiency. BMC Gastroenterol. 2014 Apr 9;14:71. doi: 10.1186/1471-230X-14-71.
- Derived from this study: PubMed 23878046MacGinnitie AJ, Davis-Lorton M, Stolz LE, Tachdjian R. Use of ecallantide in pediatric hereditary angioedema. Pediatrics. 2013 Aug;132(2):e490-7. doi: 10.1542/peds.2013-0646. Epub 2013 Jul 22.
- Derived from this study: PubMed 23548529Sheffer AL, MacGinnitie AJ, Campion M, Stolz LE, Pullman WE. Outcomes after ecallantide treatment of laryngeal hereditary angioedema attacks. Ann Allergy Asthma Immunol. 2013 Mar;110(3):184-188.e2. doi: 10.1016/j.anai.2012.12.007. Epub 2013 Jan 5.
- Derived from this study: PubMed 23548526Li HH, Campion M, Craig TJ, Soteres DF, Riedl M, Lumry WR, MacGinnitie AJ, Shea EP, Bernstein JA. Analysis of hereditary angioedema attacks requiring a second dose of ecallantide. Ann Allergy Asthma Immunol. 2013 Mar;110(3):168-72. doi: 10.1016/j.anai.2012.12.004. Epub 2013 Jan 8.
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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
41 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing ecallantide in people with Hereditary Angioedema (HAE), run by Shire at 41 sites in 3 countries.
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 4 inclusion and 4 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 41 sites in United States, Canada, Jordan. 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.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 28 Dec 2012). 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?
Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hrs Post Dosing (time frame: 4 hrs post dose after every episode). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00456508, which also lists 4 linked publications.
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.