Hereditary AngioedemaPhase 2NCT03712228

A Study to Investigate CSL312 in Subjects With Hereditary Angioedema (HAE)

A Phase 2 study testing Factor XIIa antagonist monoclonal antibody in people with Hereditary Angioedema, run by CSL Behring at 16 sites in 5 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

Trial summary

This is a multicenter, randomized, placebo-controlled, parallel-arm, phase 2 study to investigate the clinical efficacy, pharmacokinetics, and safety of CSL312 as prophylaxis to prevent attacks in subjects with HAE. Read more about this study

Who is it for?: Ages 18 to 65 years
Conditions the study is for: Hereditary Angioedema
What is being tested?: Factor XIIa antagonist monoclonal antibody
Type of treatment: Injection or infusion
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 44 participants
Where: 16 sitesin 5 countries
Study dates: 29 Oct 2018to 15 Oct 2021
Phase: Phase 2
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 18 to 65 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Male or female
Aged ≥ 18 to ≤ 65 years
A diagnosis of C1-INH HAE or FXII/PLG HAE;
For subjects with C1-INH HAE: ≥ 4 HAE attacks over a consecutive 2-month period during the 3 months before Screening, as documented in the subject's medical record.

Who cannot join

History of clinically significant arterial or venous thrombosis, or current clinically significant prothrombotic risk
History of an uncontrolled, abnormal bleeding event due to a coagulopathy, or a current clinically significant coagulopathy or clinically significant risks for bleeding events
Known incurable malignancies

Trial locations and contacts

16 sites in 5 countries.

Country

City, postcode or hospital

16 sites in 5 countries

United States7 sites

Immunoe Health Centers
Centennial, Colorado, 80112
Institute for Asthma and Allergy
Chevy Chase, Maryland, 20815
AARA Research Center
Dallas, Texas, 75231
Pennsylvania State University
Hershey, Pennsylvania, 17033
The Mount Sinai Hospital
New York, New York, 10029
Donald S. Levy
Orange, California, 92868
Allergy & Asthma Clinical Research
Walnut Creek, California, 94598

Other trials in United States

Germany4 sites

Charité Universitätsmedizin Berlin
Berlin, 10117
Universitätsklinikum Frankfurt Goethe-Universität
Frankfurt, 60590
Hautklinik und Poliklinik der Universitätsklinik Mainz
Mainz, 55131
HZRM Hämophilie Zentrum Rhein Main GmbH
Mörfelden-Walldorf, 64546

Other trials in Germany

Canada3 sites

University of Alberta
Edmonton, Alberta, T6G 2B7
Allergy and Clinical Immunology McMaster University
Hamilton, Ontario, L8S 4K1
Ottawa Allergy Research Corp
Ottawa, Ontario, K1G 6C6

Other trials in Canada

Australia1 site

Campbelltown Hospital
Campbelltown, New South Wales, 2560

Other trials in Australia

Israel1 site

Barzilai University Medical Center
Ashkelon, 7830604

Other trials in Israel

About this study

Official title: A Multicenter, Randomized, Placebo-controlled, Parallel-arm Study to Investigate the Efficacy, Pharmacokinetics, and Safety of CSL312 in Subjects With Hereditary Angioedema

Conditions: Hereditary Angioedema
Being tested: Factor XIIa antagonist monoclonal antibody, Placebo
Medical subject headings: Angioedemas, Hereditary

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Factor XIIa antagonist monoclonal antibodyBiological
Also called CSL312
Factor XIIa antagonist monoclonal antibody for intravenous and subcutaneous use
PlaceboDrug
Buffer without active ingredient

Study groups in NCT03712228
Study group	Role	What they receive
Placebo	Placebo comparator	Subjects with C1-INH HAE receiving buffer only Receives: Drug: Placebo
CSL312 (low)	Active comparator	Subjects with C1-INH HAE receiving low dose CSL312 Receives: Biological: Factor XIIa antagonist monoclonal antibody
CSL312 (med)	Active comparator	Subjects with C1-INH HAE receiving medium dose CSL312 Receives: Biological: Factor XIIa antagonist monoclonal antibody
CSL312 (high)	Active comparator	Subjects with C1-INH HAE receiving high dose CSL312 Receives: Biological: Factor XIIa antagonist monoclonal antibody
CSL312 (med/high)	Active comparator	Subjects with C1-INH HAE receiving medium/high dose CSL312 Receives: Biological: Factor XIIa antagonist monoclonal antibody

What is being measured

The primary outcome is the main question the study is designed to answer.

The Mean Time Normalized Number of HAE Attacks Per Month in Subjects With C1-INH HAE During Treatment Period 1
The time-normalized number of HAE attacks per month during Treatment Period 1 for a subject was calculated as the (number of HAE attacks / length of subject's evaluation period in days) * 30.4375
Measured: 13 weeks

Secondary outcomes (16)

The Number of Responder Subjects With C1-INH HAE During Treatment Period 1
Response is defined as a ≥ 50% relative reduction in the time-normalized number of HAE attacks (per month) during Treatment Period 1 compared to each subject's time-normalized number of HAE attacks (per month) during the Run-in Period
Measured: 13 weeks
The Percentage of Responder Subjects With C1-INH HAE During Treatment Period 1
Response is defined as a ≥ 50% relative reduction in the time-normalized number of HAE attacks (per month) during Treatment Period 1 compared to each subject's time-normalized number of HAE attacks (per month) during the Run-in Period.
Measured: 13 weeks
The Number of HAE Attack-free Subjects With C1-INH HAE During Treatment Period 1
Measured: 13 weeks
The Percentage of HAE Attack-free Subjects With C1-INH HAE During Treatment Period 1
Measured: 13 weeks
The Number of Mild, Moderate or Severe HAE Attacks in Subjects With C1-INH HAE During Treatment Period 1
Measured: 13 weeks
The Percentage of Mild, Moderate or Severe HAE Attacks in Subjects With C1-INH HAE During Treatment Period 1
Measured: 13 weeks
The Mean Time-normalized Number of Mild, Moderate or Severe HAE Attacks Per Month in Subjects With C1-INH HAE During Treatment Period 1
The time-normalized number of HAE attacks per month during Treatment Period 1 for a subject was calculated as the (number of HAE attacks / length of subject's evaluation period in days) * 30.4375
Measured: 13 weeks
The Number of Subjects With at Least One (1) HAE Attack Treated With On-demand HAE Medication, in Subjects With C1-INH HAE During Treatment Period 1
Measured: 13 weeks
The Percentage of Subjects With at Least One (1) HAE Attack Treated With On-demand HAE Medication, in Subjects With C1-INH HAE During Treatment Period 1
Measured: 13 weeks
Maximum Concentration (Cmax) of CSL312 in Subjects With C1-INH HAE During Treatment Period 1
Measured: 13 weeks
Area Under the Concentration-time Curve in 1 Dosing Interval (AUC0-tau) of CSL312 in Subjects With C1-INH HAE During Treatment Period 1
Measured: 13 weeks
Time of Maximum Concentration (Tmax) of CSL312 in Subjects With C1-INH HAE During Treatment Period 1
Measured: 13 weeks
Terminal Elimination Half-life (T1/2) of CSL312 in Subjects With C1-INH HAE During Treatment Period 1
Measured: 13 weeks
Clearance (CL/F) of CSL312 in Subjects With C1-INH HAE During Treatment Period 1
Measured: 13 weeks
Volume of Distribution During the Elimination Phase (Vz/F) of CSL312 in Subjects With C1-INH HAE During Treatment Period 1
Measured: 13 weeks
The Number of Subjects With C1-INH HAE With Adverse Events (AEs), Serious Adverse Events (SAEs), Adverse Events of Special Interest (AESI), Injection Site Reactions (ISRs), Binding Antibodies to CSL312 During Treatment Period 1
Adverse events of special interest is defined as anaphylaxis, thromboembolic events, and bleeding events.
Measured: 13 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

29 Oct 201815 Oct 2021

First posted19 Oct 2018
Study start29 Oct 2018
Primary completion15 Oct 2021
Study completion15 Oct 2021

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 AngioedemaStudies 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 2
Primary purpose: Prevention
Allocation: Randomised
Intervention model: Parallel groups
Model description: Subjects are assigned to 1 of 2 or more groups in parallel for the duration of the study
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 44 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03712228
Sponsor's protocol ID: CSL312_2001
Other IDs: 2018-000605-24, 2018
Status last verified: Oct 2022
First submitted: 17 Oct 2018
Last updated: 8 Nov 2022
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Publications

Craig TJ, Guilarte M, Li HH, Anderson J, Martinez Saguer I, Jacobs JS, Tachdjian R, Farkas H, Yang WH, Ohsawa I, Hakl R, Pollen M, Pragst I, Lawo JP, Shetty H, Nenci C, Magerl M. Long-Term Integrated Safety and Efficacy of Garadacimab for Hereditary Angioedema Prophylaxis. Adv Ther. 2026 Jun 10. doi: 10.1007/s12325-026-03628-x. Online ahead of print.
PubMed 42268494
Craig TJ, Levy DS, Reshef A, Lumry WR, Martinez-Saguer I, Jacobs JS, Yang WH, Ritchie B, Aygoren-Pursun E, Keith PK, Busse P, Feuersenger H, Alexandru Bica M, Jacobs I, Pragst I, Magerl M. Garadacimab for hereditary angioedema attack prevention: long-term efficacy, quality of life, and safety data from a phase 2, randomised, open-label extension study. Lancet Haematol. 2024 Jun;11(6):e436-e447. doi: 10.1016/S2352-3026(24)00081-4. Epub 2024 May 3.
PubMed 38710185
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.
PubMed 36326435
Craig T, Magerl M, Levy DS, Reshef A, Lumry WR, Martinez-Saguer I, Jacobs JS, Yang WH, Ritchie B, Aygoren-Pursun E, Keith PK, Busse P, Feuersenger H, Pawaskar D, Jacobs I, Pragst I, Doyle MK. Prophylactic use of an anti-activated factor XII monoclonal antibody, garadacimab, for patients with C1-esterase inhibitor-deficient hereditary angioedema: a randomised, double-blind, placebo-controlled, phase 2 trial. Lancet. 2022 Mar 5;399(10328):945-955. doi: 10.1016/S0140-6736(21)02225-X. Epub 2022 Feb 24.
PubMed 35219377

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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A Study of Takhzyro in Teenagers and Adults With Hereditary Angioedema (HAE) in South Korea
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RecruitingPhase 3
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Acute Hereditary Angio Edema
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Hereditary Angioedema

All Hereditary Angioedema trials

Study at a glance

From the registry record, last updated by the sponsor on 8 Nov 2022. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Multicenter, Randomized, Placebo-controlled, Parallel-arm Study to Investigate the Efficacy, Pharmacokinetics, and Safety of CSL312 in Subjects With Hereditary Angioedema
ClinicalTrials.gov ID: NCT03712228
Acronym / sponsor ID: CSL312_2001
Conditions: Hereditary Angioedema
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 44 participants (actual)
Lead sponsor: CSL BehringIndustry (pharma, biotech, devices)
Study start: 29 Oct 2018 (actual)
Primary completion: 15 Oct 2021 (actual)
Study completion: 15 Oct 2021 (actual)
First posted: 19 Oct 2018 (actual)
Results: Posted 8 Nov 2022

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Prevention
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 5
Model description: Subjects are assigned to 1 of 2 or more groups in parallel for the duration of the study

Arm	Type	Receives
Placebo	Placebo comparator	Drug: PlaceboSubjects with C1-INH HAE receiving buffer only
CSL312 (low)	Active comparator	Biological: Factor XIIa antagonist monoclonal antibodySubjects with C1-INH HAE receiving low dose CSL312
CSL312 (med)	Active comparator	Biological: Factor XIIa antagonist monoclonal antibodySubjects with C1-INH HAE receiving medium dose CSL312
CSL312 (high)	Active comparator	Biological: Factor XIIa antagonist monoclonal antibodySubjects with C1-INH HAE receiving high dose CSL312
CSL312 (med/high)	Active comparator	Biological: Factor XIIa antagonist monoclonal antibodySubjects with C1-INH HAE receiving medium/high dose CSL312

Who may qualify

Ages: 18 to 65 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (4 listed)

Male or female
Aged ≥ 18 to ≤ 65 years
A diagnosis of C1-INH HAE or FXII/PLG HAE;
For subjects with C1-INH HAE: ≥ 4 HAE attacks over a consecutive 2-month period during the 3 months before Screening, as documented in the subject's medical record.

Key exclusion criteria (3 listed)

History of clinically significant arterial or venous thrombosis, or current clinically significant prothrombotic risk
History of an uncontrolled, abnormal bleeding event due to a coagulopathy, or a current clinically significant coagulopathy or clinically significant risks for bleeding events
Known incurable malignancies

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
Factor XIIa antagonist monoclonal antibodyAlso: CSL312Factor XIIa antagonist monoclonal antibody for intravenous and subcutaneous use	Biological	CSL312 (high), CSL312 (low), CSL312 (med), CSL312 (med/high)
PlaceboBuffer without active ingredient	Drug	Placebo

Primary outcome

The Mean Time Normalized Number of HAE Attacks Per Month in Subjects With C1-INH HAE During Treatment Period 1
Time frame: 13 weeks
The time-normalized number of HAE attacks per month during Treatment Period 1 for a subject was calculated as the (number of HAE attacks / length of subject's evaluation period in days) * 30.4375

16 secondary outcomes

The Number of Responder Subjects With C1-INH HAE During Treatment Period 1
Time frame: 13 weeks
The Percentage of Responder Subjects With C1-INH HAE During Treatment Period 1
Time frame: 13 weeks
The Number of HAE Attack-free Subjects With C1-INH HAE During Treatment Period 1
Time frame: 13 weeks
The Percentage of HAE Attack-free Subjects With C1-INH HAE During Treatment Period 1
Time frame: 13 weeks
The Number of Mild, Moderate or Severe HAE Attacks in Subjects With C1-INH HAE During Treatment Period 1
Time frame: 13 weeks
The Percentage of Mild, Moderate or Severe HAE Attacks in Subjects With C1-INH HAE During Treatment Period 1
Time frame: 13 weeks
The Mean Time-normalized Number of Mild, Moderate or Severe HAE Attacks Per Month in Subjects With C1-INH HAE During Treatment Period 1
Time frame: 13 weeks
The Number of Subjects With at Least One (1) HAE Attack Treated With On-demand HAE Medication, in Subjects With C1-INH HAE During Treatment Period 1
Time frame: 13 weeks
The Percentage of Subjects With at Least One (1) HAE Attack Treated With On-demand HAE Medication, in Subjects With C1-INH HAE During Treatment Period 1
Time frame: 13 weeks
Maximum Concentration (Cmax) of CSL312 in Subjects With C1-INH HAE During Treatment Period 1
Time frame: 13 weeks
Area Under the Concentration-time Curve in 1 Dosing Interval (AUC0-tau) of CSL312 in Subjects With C1-INH HAE During Treatment Period 1
Time frame: 13 weeks
Time of Maximum Concentration (Tmax) of CSL312 in Subjects With C1-INH HAE During Treatment Period 1
Time frame: 13 weeks
Terminal Elimination Half-life (T1/2) of CSL312 in Subjects With C1-INH HAE During Treatment Period 1
Time frame: 13 weeks
Clearance (CL/F) of CSL312 in Subjects With C1-INH HAE During Treatment Period 1
Time frame: 13 weeks
Volume of Distribution During the Elimination Phase (Vz/F) of CSL312 in Subjects With C1-INH HAE During Treatment Period 1
Time frame: 13 weeks
The Number of Subjects With C1-INH HAE With Adverse Events (AEs), Serious Adverse Events (SAEs), Adverse Events of Special Interest (AESI), Injection Site Reactions (ISRs), Binding Antibodies to CSL312 During Treatment Period 1
Time frame: 13 weeks

Results and safety

Results posted to the registry on 8 Nov 2022, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Placebo	8	8
CSL312 (Low)	9	9
CSL312 (Med)	8	8
CSL312 (High)	7	7
CSL312 (Med/High)	6	6
CSL312 (FXII/PLG HAE)	6	5

Primary outcome: The Mean Time Normalized Number of HAE Attacks Per Month in Subjects With C1-INH HAE During Treatment Period 1

Time frame: 13 weeks. Mean (Standard Deviation). Unit: Number of HAE attacks per month

Measure	Placebo n = 8	CSL312 (Low) n = 9	CSL312 (Med) n = 8	CSL312 (High) n = 7	CSL312 (Med/High) n = 6
Value	4.24 (1.801)	0.48 (1.057)	0.05 (0.127)	0.35 (0.407)	0.14 (0.222)

Population analysed: The Intent-to-Treat (ITT) population consisted of all subjects who provided informed consent, underwent any study screening procedure, and who were assigned to treatment in Treatment Period 1 or to treatment in Treatment Period 2, regardless of whether they received investigational product.

16 secondary outcomes are reported in the full results.

Adverse events

Time frame: Approximately 129 weeks per participant. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Placebo	0 of 8 (0%)	7 of 8 (87.5%)	0 of 8
CSL312 (Low)	0 of 9 (0%)	7 of 9 (77.8%)	0 of 9
CSL312 (Med)	0 of 8 (0%)	7 of 8 (87.5%)	0 of 8
CSL312 (High)	0 of 7 (0%)	7 of 7 (100%)	0 of 7
CSL312 (Med/High)	0 of 6 (0%)	4 of 6 (66.7%)	0 of 6
CSL312 (Med-Period 2)	1 of 36 (2.8%)	25 of 36 (69.4%)	0 of 36
CSL312 (Med/High-Period 2)	0 of 3 (0%)	2 of 3 (66.7%)	0 of 3
CSL312 (High-Period 2)	1 of 18 (5.6%)	17 of 18 (94.4%)	0 of 18
CSL312 (FXII/PLG HAE-Period 1)	1 of 6 (16.7%)	1 of 6 (16.7%)	0 of 6
CSL312 (FXII/PLG HAE-Period 2)	0 of 2 (0%)	2 of 2 (100%)	0 of 2

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 8 Nov 2022.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 42268494Craig TJ, Guilarte M, Li HH, Anderson J, Martinez Saguer I, Jacobs JS, Tachdjian R, Farkas H, Yang WH, Ohsawa I, Hakl R, Pollen M, Pragst I, Lawo JP, Shetty H, Nenci C, Magerl M. Long-Term Integrated Safety and Efficacy of Garadacimab for Hereditary Angioedema Prophylaxis. Adv Ther. 2026 Jun 10. doi: 10.1007/s12325-026-03628-x. Online ahead of print.
Derived from this study: PubMed 38710185Craig TJ, Levy DS, Reshef A, Lumry WR, Martinez-Saguer I, Jacobs JS, Yang WH, Ritchie B, Aygoren-Pursun E, Keith PK, Busse P, Feuersenger H, Alexandru Bica M, Jacobs I, Pragst I, Magerl M. Garadacimab for hereditary angioedema attack prevention: long-term efficacy, quality of life, and safety data from a phase 2, randomised, open-label extension study. Lancet Haematol. 2024 Jun;11(6):e436-e447. doi: 10.1016/S2352-3026(24)00081-4. Epub 2024 May 3.
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 35219377Craig T, Magerl M, Levy DS, Reshef A, Lumry WR, Martinez-Saguer I, Jacobs JS, Yang WH, Ritchie B, Aygoren-Pursun E, Keith PK, Busse P, Feuersenger H, Pawaskar D, Jacobs I, Pragst I, Doyle MK. Prophylactic use of an anti-activated factor XII monoclonal antibody, garadacimab, for patients with C1-esterase inhibitor-deficient hereditary angioedema: a randomised, double-blind, placebo-controlled, phase 2 trial. Lancet. 2022 Mar 5;399(10328):945-955. doi: 10.1016/S0140-6736(21)02225-X. Epub 2022 Feb 24.
Individual participant dataSharing planned: No

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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

CSL Behring LLC

16 sites in 5 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Factor XIIa antagonist monoclonal antibody in people with Hereditary Angioedema, run by CSL Behring at 16 sites in 5 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 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 Nov 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 16 sites in United States, Germany, Canada, Australia, Israel. 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. Some participants in this study may receive a placebo. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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 8 Nov 2022). 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 Mean Time Normalized Number of HAE Attacks Per Month in Subjects With C1-INH HAE During Treatment Period 1 (time frame: 13 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 NCT03712228, 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 Nov 2022.