Hereditary Angioedema Types I and IIPhase 3NCT01912456
A Study to Evaluate the Clinical Efficacy and Safety of Subcutaneously Administered C1-esterase Inhibitor in the Prevention of Hereditary Angioedema
A Phase 3 study testing Low-volume C1-esterase inhibitor and Higher-volume C1-esterase inhibitor in people with Hereditary Angioedema Types I and II, run by CSL Behring at 39 sites in 10 countries.
Trial summary
The aim of this study is to assess the efficacy of C1-esterase inhibitor in preventing hereditary angioedema attacks when it is administered under the skin of subjects with hereditary angioedema. The safety of C1-esterase inhibitor will also be assessed. Each subject will enter a run-in period of up to 8-weeks. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Hereditary Angioedema Types I and II
- What is being tested?
- Low-volume C1-esterase inhibitor and Higher-volume C1-esterase inhibitor
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 90 participants
- Where
- 39 sitesin 10 countries
- Study dates
- Jan 2014to Oct 2015
- 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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Run-In Period Inclusion Criteria:
- Males or females aged 12 years or older.
- A clinical diagnosis of hereditary angioedema type I or II.
- Hereditary angioedema attacks over a consecutive 2-month period that required acute treatment, medical attention, or caused significant functional impairment.
- For subjects who have used oral therapy for prophylaxis against HAE attacks within 3 months of Screening: use of a stable regimen within 3 months of Screening, with no plans to change.
- Eligibility Criteria for Entering Treatment Period 1:
Show all 9Show fewer
- Laboratory confirmation of type I or type II hereditary angioedema, including C1-esterase inhibitor functional activity less than 50% AND C4 antigen level below the laboratory reference range.
- No clinically significant abnormalities as assessed using laboratory parameters.
- During participation in the run-in period, subjects must have experienced hereditary angioedema attacks that required acute treatment, required medical attention, or caused significant functional impairment.
Who cannot join
- Run-In Period Exclusion Criteria:
- History of clinical significant arterial or venous thrombosis, or current history of a clinically significant prothrombotic risk.
- Incurable malignancies at screening.
- Any clinical condition that will interfere with the evaluation of C1-esterase inhibitor therapy.
- Clinically significant history of poor response to C1-esterase therapy for the management of hereditary angioedema.
- Receiving therapy prohibited by the protocol, including medications for hereditary angioedema prophylaxis.
- Female subjects who started taking or changed dose of any hormonal contraceptive regimen or hormone replacement therapy (i.e., estrogen/progesterone-containing products) within 3 months prior to the screening visit.
Trial locations and contacts
39 sites in 10 countries.
39 sites in 10 countries
United States19 sites
Study Site
Bell Gardens, California, 90201
Study Site
Birmingham, Alabama, 35209
Study Site
Boston, Massachusetts, 02114
Study Site
Chevy Chase, Maryland, 20815
Study Site
Cincinnati, Ohio, 45267-0563
Study Site
Colorado Springs, Colorado, 80907
Study Site
Columbus, Ohio, 43235
Study Site
Dallas, Texas, 75231
Study Site
Hershey, Pennsylvania, 17033
Study Site
La Jolla, California, 92093
Study Site
Lake Oswego, Oregon, 97035
Study Site
Orange, California, 92868
Study Site
Richmond, Virginia, 23298
Study Site
Scottsdale, Arizona, 85251
Study Site
Spokane, Washington, 99204
Study Site
Toledo, Ohio, 43617
Study Site
Tulsa, Oklahoma, 74136
Study Site
Virginia Beach, Virginia, 23452
Study Site
Walnut Creek, California, 94598
Canada4 sites
Study Site
Hamilton, Ontario, L8N 3Z5
Study Site
Ottawa, Ontario, K1Y 4G2
Study Site
Québec, G1V 4M6
Study Site
Toronto, Ontario, M4V 1R2
Spain4 sites
Study Site
Barcelona, 08035
Study Site
Madrid, 28007
Study Site
Madrid, 28046
Study Site
Valencia, 46026
United Kingdom2 sites
Study Site
Brighton, BN2 5BE
Study Site
London, E1 2ES
About this study
Official title: A Double-blind, Randomized, Placebo-controlled, Cross-over Study to Evaluate the Clinical Efficacy and Safety of Subcutaneous Administration of Human Plasma-derived C1-esterase Inhibitor in the Prophylactic Treatment of Hereditary Angioedema
The aim of this study is to assess the efficacy of C1-esterase inhibitor in preventing hereditary angioedema attacks when it is administered under the skin of subjects with hereditary angioedema. The safety of C1-esterase inhibitor will also be assessed. Each subject will enter a run-in period of up to 8-weeks. Subjects who complete the run-in period and who are eligible will then enter the treatment phase which comprises two sequential treatment periods. In the treatment phase, subjects will be randomized to one of four arms consisting of treatment with low- or higher-volume C1-esterase inhibitor in one treatment period and treatment with low- or higher-volume placebo in the other treatment period. The study will measure the number of hereditary angioedema attacks that subjects experience while receiving each treatment.
- Conditions
- Hereditary Angioedema Types I and II
- Being tested
- Low-volume C1-esterase inhibitor, Higher-volume C1-esterase inhibitor, Low-volume placebo, Higher-volume placebo
- Keywords
- Hereditary Angioedema
- Medical subject headings
- Hereditary Angioedema Types I and II, Angioedemas, Hereditary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Low-volume C1-esterase inhibitorBiological
Higher-volume C1-esterase inhibitorBiological
Low-volume placeboBiological
Higher-volume placeboBiological
| Study group | Role | What they receive |
|---|---|---|
| Higher-volume placebo, then low-volume C1-esterase inhibitor | Experimental | A higher-volume dose of placebo will be administered subcutaneously twice a week for up to 16 weeks, then a low-volume dose of C1-esterase inhibitor will be administered subcutaneously twice a week for up to 16 weeks. Receives: Biological: Low-volume C1-esterase inhibitor; Biological: Higher-volume placebo |
| Low-volume C1-esterase inhibitor, then higher-volume placebo | Experimental | A low-volume dose of C1-esterase inhibitor will be administered subcutaneously twice a week for up to 16 weeks, then a higher-volume dose of placebo will be administered subcutaneously twice a week for up to 16 weeks. Receives: Biological: Low-volume C1-esterase inhibitor; Biological: Higher-volume placebo |
| Low-volume placebo, then higher-volume C1-esterase inhibitor | Experimental | A low-volume dose of placebo will be administered subcutaneously twice a week for up to 16 weeks then a higher-volume dose of C1-esterase inhibitor will be administered subcutaneously twice a week for up to 16 weeks. Receives: Biological: Higher-volume C1-esterase inhibitor; Biological: Low-volume placebo |
| Higher-volume C1-esterase inhibitor, then low-volume placebo | Experimental | A higher-volume dose of C1-esterase inhibitor will be administered subcutaneously twice a week for up to 16 weeks, then a low-volume dose of placebo will be administered subcutaneously twice a week for up to 16 weeks. Receives: Biological: Higher-volume C1-esterase inhibitor; Biological: Low-volume placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
The Time-normalized Number of Hereditary Angioedema Attacks
The time normalized number of HAE attacks as reported by the investigator per subject was calculated as: The total number of HAE attacks per subject and per treatment period / length of stay of subject in treatment period (days), Where length of stay of subject in treatment period was calculated as: Date of last day of subject in treatment period - date of first day of Week 3 of subject in treatment period + 1.
Measured: During the treatment phase, up to 28 weeks.
Secondary outcomes (6)
Percentage of Subjects With a ≥ 50% Reduction in the Number of Hereditary Angioedema Attacks by CSL830 Treatment
The percentage reduction (%) in the time normalized number of HAE attacks was calculated as: 100 x [1 - (the time normalized number of HAE attacks when treated with CSL830) / (the time normalized number of HAE attacks when treated with placebo)]. A subject is classed as a responder if the percentage reduction is >= 50%.
Measured: During the treatment phase, up to 28 weeks.
Time-Normalized Number of Uses of Rescue Medication
The time-normalized number of uses of rescue medication during treatment with C1-esterase inhibitor or placebo
Measured: During the treatment phase, up to 28 weeks.
Percentage of Subjects With Adverse Events (AEs) Within 24 Hours of C1-esterase Inhibitor or Placebo Administration
Measured: Within 24 hours of C1-esterase inhibitor or placebo administration.
Percentage of Subjects With AEs or Other Specified Safety Events.
The percentage of subjects experiencing the following during treatment with CSL830 and placebo: unsolicited AEs, serious AEs, suspected adverse drug reactions, increased risk scores for deep vein thrombosis and pulmonary embolism, thromboembolic events, inhibitory anti C1 INH antibodies, or clinically significant abnormalities in laboratory assessments.
Measured: During the treatment phase, up to 32 weeks.
Percentage of Subjects Experiencing Solicited AEs (Injection Site Reactions)
The percentage of subjects experiencing solicited local AEs (discomfort [eg, pain, burning], swelling, bruising, or itching at the investigational product injection site) during treatment with CSL830 and placebo.
Measured: During the treatment phase, up to 32 weeks.
Injections Resulting in Solicited AEs (Injection Site Reactions)
The rate/injection of injections of C1-esterase inhibitor or placebo that were followed by solicited local AEs (discomfort [eg, pain, burning], swelling, bruising, or itching at the investigational product injection site) during treatment with CSL830 and placebo. Rate/Injection = Number of events/number of injections.
Measured: During the treatment phase, up to 32 weeks.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Jul 2013 (estimated)
- Study startJan 2014
- Primary completionOct 2015
- Study completionOct 2015
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 (3)Papers the sponsor has linked to this study.
- Other trials for Hereditary Angioedema Types I and IIStudies of the same condition, open and under way.
- More trials run by CSL BehringThe sponsor's other registered studies.
- 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
- Prevention
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 90 participants (actual)
Sponsor and investigators
- Lead sponsor
- CSL Behring
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Global Clinical Program Director, Study director, CSL Behring
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01912456
- Sponsor's protocol ID
- CSL830_3001
- Other IDs
- 2013-000916-10
- Status last verified
- Jan 2021
- First submitted
- 29 Jul 2013
- Last updated
- 29 Jan 2021
- Results posted
- Yes, on ClinicalTrials.gov
Publications
Longhurst H, Cicardi M, Craig T, Bork K, Grattan C, Baker J, Li HH, Reshef A, Bonner J, Bernstein JA, Anderson J, Lumry WR, Farkas H, Katelaris CH, Sussman GL, Jacobs J, Riedl M, Manning ME, Hebert J, Keith PK, Kivity S, Neri S, Levy DS, Baeza ML, Nathan R, Schwartz LB, Caballero T, Yang W, Crisan I, Hernandez MD, Hussain I, Tarzi M, Ritchie B, Kralickova P, Guilarte M, Rehman SM, Banerji A, Gower RG, Bensen-Kennedy D, Edelman J, Feuersenger H, Lawo JP, Machnig T, Pawaskar D, Pragst I, Zuraw BL; COMPACT Investigators. Prevention of Hereditary Angioedema Attacks with a Subcutaneous C1 Inhibitor. N Engl J Med. 2017 Mar 23;376(12):1131-1140. doi: 10.1056/NEJMoa1613627.
PubMed 28328347Beard 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.
PubMed 36326435Li HH, Zuraw B, Longhurst HJ, Cicardi M, Bork K, Baker J, Lumry W, Bernstein J, Manning M, Levy D, Riedl MA, Feuersenger H, Prusty S, Pragst I, Machnig T, Craig T; COMPACT Investigators. Subcutaneous C1 inhibitor for prevention of attacks of hereditary angioedema: additional outcomes and subgroup analysis of a placebo-controlled randomized study. Allergy Asthma Clin Immunol. 2019 Aug 28;15:49. doi: 10.1186/s13223-019-0362-1. eCollection 2019.
PubMed 31485239
Study at a glance
From the registry record, last updated by the sponsor on 29 Jan 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Double-blind, Randomized, Placebo-controlled, Cross-over Study to Evaluate the Clinical Efficacy and Safety of Subcutaneous Administration of Human Plasma-derived C1-esterase Inhibitor in the Prophylactic Treatment of Hereditary Angioedema
- ClinicalTrials.gov ID
- NCT01912456
- Acronym / sponsor ID
- CSL830_3001
- Conditions
- Hereditary Angioedema Types I and II
- Keywords
- Hereditary Angioedema
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 90 participants (actual)
- Lead sponsor
- CSL BehringIndustry (pharma, biotech, devices)
- Study start
- Jan 2014
- Primary completion
- Oct 2015 (actual)
- Study completion
- Oct 2015 (actual)
- First posted
- 31 Jul 2013 (estimated)
- Results
- Posted 29 Jan 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The aim of this study is to assess the efficacy of C1-esterase inhibitor in preventing hereditary angioedema attacks when it is administered under the skin of subjects with hereditary angioedema. The safety of C1-esterase inhibitor will also be assessed. Each subject will enter a run-in period of up to 8-weeks. Subjects who complete the run-in period and who are eligible will then enter the treatment phase which comprises two sequential treatment periods. In the treatment phase, subjects will be randomized to one of four arms consisting of treatment with low- or higher-volume C1-esterase inhibitor in one treatment period and treatment with low- or higher-volume placebo in the other treatment period. The study will measure the number of hereditary angioedema attacks that subjects experience while receiving each treatment.
Study design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Higher-volume placebo, then low-volume C1-esterase inhibitor | Experimental | Biological: Low-volume C1-esterase inhibitor, Biological: Higher-volume placeboA higher-volume dose of placebo will be administered subcutaneously twice a week for up to 16 weeks, then a low-volume dose of C1-esterase inhibitor will be administered subcutaneously twice a week for up to 16 weeks. |
| Low-volume C1-esterase inhibitor, then higher-volume placebo | Experimental | Biological: Low-volume C1-esterase inhibitor, Biological: Higher-volume placeboA low-volume dose of C1-esterase inhibitor will be administered subcutaneously twice a week for up to 16 weeks, then a higher-volume dose of placebo will be administered subcutaneously twice a week for up to 16 weeks. |
| Low-volume placebo, then higher-volume C1-esterase inhibitor | Experimental | Biological: Higher-volume C1-esterase inhibitor, Biological: Low-volume placeboA low-volume dose of placebo will be administered subcutaneously twice a week for up to 16 weeks then a higher-volume dose of C1-esterase inhibitor will be administered subcutaneously twice a week for up to 16 weeks. |
| Higher-volume C1-esterase inhibitor, then low-volume placebo | Experimental | Biological: Higher-volume C1-esterase inhibitor, Biological: Low-volume placeboA higher-volume dose of C1-esterase inhibitor will be administered subcutaneously twice a week for up to 16 weeks, then a low-volume dose of placebo will be administered subcutaneously twice a week for up to 16 weeks. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- Males or females aged 12 years or older.
- A clinical diagnosis of hereditary angioedema type I or II.
- Hereditary angioedema attacks over a consecutive 2-month period that required acute treatment, medical attention, or caused significant functional impairment.
- For subjects who have used oral therapy for prophylaxis against HAE attacks within 3 months of Screening: use of a stable regimen within 3 months of Screening, with no plans to change.
- Laboratory confirmation of type I or type II hereditary angioedema, including C1-esterase inhibitor functional activity less than 50% AND C4 antigen level below the laboratory reference range.
Key exclusion criteria (7 listed)
- History of clinical significant arterial or venous thrombosis, or current history of a clinically significant prothrombotic risk.
- Incurable malignancies at screening.
- Any clinical condition that will interfere with the evaluation of C1-esterase inhibitor therapy.
- Clinically significant history of poor response to C1-esterase therapy for the management of hereditary angioedema.
- Receiving therapy prohibited by the protocol, including medications for hereditary angioedema prophylaxis.
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 |
|---|---|---|
| Low-volume C1-esterase inhibitor | Biological | Higher-volume placebo, then low-volume C1-esterase inhibitor, Low-volume C1-esterase inhibitor, then higher-volume placebo |
| Higher-volume C1-esterase inhibitor | Biological | Higher-volume C1-esterase inhibitor, then low-volume placebo, Low-volume placebo, then higher-volume C1-esterase inhibitor |
| Low-volume placebo | Biological | Higher-volume C1-esterase inhibitor, then low-volume placebo, Low-volume placebo, then higher-volume C1-esterase inhibitor |
| Higher-volume placebo | Biological | Higher-volume placebo, then low-volume C1-esterase inhibitor, Low-volume C1-esterase inhibitor, then higher-volume placebo |
Primary outcome
The Time-normalized Number of Hereditary Angioedema Attacks
Time frame: During the treatment phase, up to 28 weeks.
The time normalized number of HAE attacks as reported by the investigator per subject was calculated as: The total number of HAE attacks per subject and per treatment period / length of stay of subject in treatment period (days), Where length of stay of subject in treatment period was calculated as: Date of last day of subject in treatment period - date of first day of Week 3 of subject in treatment period + 1.
6 secondary outcomes
Percentage of Subjects With a ≥ 50% Reduction in the Number of Hereditary Angioedema Attacks by CSL830 Treatment
Time frame: During the treatment phase, up to 28 weeks.
Time-Normalized Number of Uses of Rescue Medication
Time frame: During the treatment phase, up to 28 weeks.
Percentage of Subjects With Adverse Events (AEs) Within 24 Hours of C1-esterase Inhibitor or Placebo Administration
Time frame: Within 24 hours of C1-esterase inhibitor or placebo administration.
Percentage of Subjects With AEs or Other Specified Safety Events.
Time frame: During the treatment phase, up to 32 weeks.
Percentage of Subjects Experiencing Solicited AEs (Injection Site Reactions)
Time frame: During the treatment phase, up to 32 weeks.
Injections Resulting in Solicited AEs (Injection Site Reactions)
Time frame: During the treatment phase, up to 32 weeks.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Jan 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 28328347Longhurst H, Cicardi M, Craig T, Bork K, Grattan C, Baker J, Li HH, Reshef A, Bonner J, Bernstein JA, Anderson J, Lumry WR, Farkas H, Katelaris CH, Sussman GL, Jacobs J, Riedl M, Manning ME, Hebert J, Keith PK, Kivity S, Neri S, Levy DS, Baeza ML, Nathan R, Schwartz LB, Caballero T, Yang W, Crisan I, Hernandez MD, Hussain I, Tarzi M, Ritchie B, Kralickova P, Guilarte M, Rehman SM, Banerji A, Gower RG, Bensen-Kennedy D, Edelman J, Feuersenger H, Lawo JP, Machnig T, Pawaskar D, Pragst I, Zuraw BL; COMPACT Investigators. Prevention of Hereditary Angioedema Attacks with a Subcutaneous C1 Inhibitor. N Engl J Med. 2017 Mar 23;376(12):1131-1140. doi: 10.1056/NEJMoa1613627.
- Derived from this study: PubMed 36326435Beard 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.
- Derived from this study: PubMed 31485239Li HH, Zuraw B, Longhurst HJ, Cicardi M, Bork K, Baker J, Lumry W, Bernstein J, Manning M, Levy D, Riedl MA, Feuersenger H, Prusty S, Pragst I, Machnig T, Craig T; COMPACT Investigators. Subcutaneous C1 inhibitor for prevention of attacks of hereditary angioedema: additional outcomes and subgroup analysis of a placebo-controlled randomized study. Allergy Asthma Clin Immunol. 2019 Aug 28;15:49. doi: 10.1186/s13223-019-0362-1. eCollection 2019.
Related trials for Hereditary Angioedema Types I and II
Enrolling by invitationPhase 2/3
Extension Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema
Hereditary Angioedema, Hereditary Angioedema Type I
Not yet recruitingObservational study
A Study About the Diagnosis and Management of Hereditary Angioedema (HAE) in Egypt
Hereditary Angioedema (HAE)
Active, not recruitingObservational study
A Study of Lanadelumab in Teenagers and Adults With Hereditary Angioedema (HAE)
Hereditary Angioedema (HAE)
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.
Global Clinical Program Director study director
CSL Behring
39 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Low-volume C1-esterase inhibitor and Higher-volume C1-esterase inhibitor in people with Hereditary Angioedema Types I and II, run by CSL Behring at 39 sites in 10 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 9 inclusion and 7 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 29 Jan 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 39 sites in United States, Canada, Spain, Czechia, Israel and 5 more countries. 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 29 Jan 2021). 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?
The Time-normalized Number of Hereditary Angioedema Attacks (time frame: During the treatment phase, up to 28 weeks.). 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 NCT01912456, which also lists 3 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 29 Jan 2021.