Hereditary AngioedemaPhase 2NCT02247739
A Phase 2 HAE Prophylaxis Study With Recombinant Human C1 Inhibitor
A Phase 2 study testing Recombinant human C1 inhibitor in people with Hereditary Angioedema, run by Pharming Technologies B.V. at 10 sites in 7 countries.
Trial summary
Primary Objective: To evaluate the efficacy of recombinant human C1 inhibitor (rhC1INH) in the prophylaxis of angioedema attacks in patients with HAE Secondary Objective: To evaluate the safety and immunogenicity of recombinant human C1 inhibitor (rhC1INH) in the prophylaxis of angioedema attacks in patients with HAE Read more about this study
- Who is it for?
- Ages 13 years and older
- Conditions the study is for
- Hereditary Angioedema
- What is being tested?
- Recombinant human C1 inhibitor
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 32 participants
- Where
- 10 sitesin 7 countries
- Study dates
- Dec 2014to Sep 2016
- 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 13 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients fulfilling the following criteria at Screening are eligible for participation in the study:
- Age 13 years or older
- Laboratory confirmed diagnosis of HAE
- A history of frequent HAE attacks (at least 4 attacks per month across a minimum of 3 consecutive months).
- Female patients of childbearing potential who are sexually active must be willing to use an acceptable form of contraception.
- Provided written informed consent (and written assent for minors)
- Willingness and ability to comply with all protocol procedures
Who cannot join
- Patients who meet any of the following criteria at Screening are to be excluded from study participation:
- Patients with medical history of allergy to rabbits or rabbit-derived products (including rhC1INH)
- Diagnosis of acquired angioedema (AAE)
- Patients who are pregnant, or breastfeeding, or are currently intending to become pregnant
- Treatment with any investigational drug in the past 30 days
- Patients with any condition or treatment that, in the opinion of the Investigator, might interfere with the evaluation of study objectives
- Patients currently treated with angiotensin-converting enzyme (ACE) inhibitors
Trial locations and contacts
10 sites in 7 countries.
10 sites in 7 countries
United States4 sites
Baker Allergy, Asthma and Dermatology Research Center
Lake Oswego, Oregon, 97035
Washington University Division of Allergy and Immunology
St Louis, Missouri, 63141
University of South Florida Asthma, Allergy and Immunology Clinical Research Unit
Tampa, Florida, 33613
University of South Florida, Asthma, Allergy & Immunology Clinical Research Unit
Tampa, Florida, 33613
Czechia1 site
Faculty Hospital by St. Anna Brno, Department of clinical Immunology and Allergology
Brno, 65691
Italy1 site
Azienda Ospedaliera Universitaria Luigi Sacco Di Milano
Milan, 20157
North Macedonia1 site
PHI University Clinic of Dermatology
Skopje, 1000
Romania1 site
SC Centrul Clinic Mediquest SRL
Sângeorgiu de Mureş, Mureș County, 547530
About this study
Official title: A Phase 2 Multicenter, Randomized, Double-Blind, Placebo-Controlled, 3-Period Crossover Study to Evaluate the Efficacy and Safety of Recombinant Human C1 Inhibitor in the Prophylaxis of Angioedema Attacks in Patients With Hereditary Angioedema (HAE)
Primary Objective:
To evaluate the efficacy of recombinant human C1 inhibitor (rhC1INH) in the prophylaxis of angioedema attacks in patients with HAE
Secondary Objective:
To evaluate the safety and immunogenicity of recombinant human C1 inhibitor (rhC1INH) in the prophylaxis of angioedema attacks in patients with HAE
Read the detailed description
Study Design:
This is a multi-center, randomized, double-blind, placebo-controlled, 3-period crossover study of rhC1INH in prophylaxis of angioedema attacks in patients with HAE.
Medical screening (clinical and laboratory parameters) will be performed and patient medical history specific to HAE attacks will be collected to assess eligibility. Each patient will receive three 4 week periods of treatment twice weekly.
- Conditions
- Hereditary Angioedema
- Being tested
- Recombinant human C1 inhibitor, Placebo
- Keywords
- HAE, Hereditary Angioedema, Prophylaxis
- Medical subject headings
- Angioedemas, Hereditary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Recombinant human C1 inhibitorBiological
Also called rhC1INH, Ruconest, Conestat alfa
PlaceboOther
Also called Saline
| Study group | Role | What they receive |
|---|---|---|
| rhC1INH twice weekly | Experimental | rhC1INH administered twice weekly Receives: Biological: Recombinant human C1 inhibitor |
| rhC1INH once weekly | Experimental | rhC1INH administered once weekly Receives: Biological: Recombinant human C1 inhibitor |
| Placebo (Saline) twice weekly | Placebo comparator | Placebo (Saline) administered twice weekly Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of HAE Attacks
Average number of HAE attacks normalized to a 28 day period
Measured: 28 days
Secondary outcomes (2)
Number of Participants With Adverse Events
Number of participants that experienced Treatment Emergent Adverse Events observed in safety population
Measured: 20 weeks
Percentage of Participants Achieving at Least 50% Reduction in Number of Attacks
Percentage of participants achieving at least 50% reduction in the number of attacks normalized to a 28-day period as compared to the placebo treatment period
Measured: 28 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Sep 2014 (estimated)
- Study startDec 2014
- Primary completionMay 2016
- Study completionSep 2016
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 (2)Papers the sponsor has linked to this study.
- Other trials for Hereditary AngioedemaStudies 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 2
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 32 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pharming Technologies B.V.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02247739
- Sponsor's protocol ID
- C1 3201
- Status last verified
- Nov 2017
- First submitted
- 16 Sep 2014
- Last updated
- 8 Dec 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
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 36326435Riedl MA, Grivcheva-Panovska V, Moldovan D, Baker J, Yang WH, Giannetti BM, Reshef A, Andrejevic S, Lockey RF, Hakl R, Kivity S, Harper JR, Relan A, Cicardi M. Recombinant human C1 esterase inhibitor for prophylaxis of hereditary angio-oedema: a phase 2, multicentre, randomised, double-blind, placebo-controlled crossover trial. Lancet. 2017 Sep 30;390(10102):1595-1602. doi: 10.1016/S0140-6736(17)31963-3. Epub 2017 Jul 25.
PubMed 28754491
Study at a glance
From the registry record, last updated by the sponsor on 8 Dec 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Multicenter, Randomized, Double-Blind, Placebo-Controlled, 3-Period Crossover Study to Evaluate the Efficacy and Safety of Recombinant Human C1 Inhibitor in the Prophylaxis of Angioedema Attacks in Patients With Hereditary Angioedema (HAE)
- ClinicalTrials.gov ID
- NCT02247739
- Acronym / sponsor ID
- C1 3201
- Conditions
- Hereditary Angioedema
- Keywords
- HAE, Hereditary Angioedema, Prophylaxis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 32 participants (actual)
- Lead sponsor
- Pharming Technologies B.V.Industry (pharma, biotech, devices)
- Study start
- Dec 2014
- Primary completion
- May 2016 (actual)
- Study completion
- Sep 2016 (actual)
- First posted
- 25 Sep 2014 (estimated)
- Results
- Posted 8 Dec 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Primary Objective:
To evaluate the efficacy of recombinant human C1 inhibitor (rhC1INH) in the prophylaxis of angioedema attacks in patients with HAE
Secondary Objective:
To evaluate the safety and immunogenicity of recombinant human C1 inhibitor (rhC1INH) in the prophylaxis of angioedema attacks in patients with HAE
Detailed description
Study Design:
This is a multi-center, randomized, double-blind, placebo-controlled, 3-period crossover study of rhC1INH in prophylaxis of angioedema attacks in patients with HAE.
Medical screening (clinical and laboratory parameters) will be performed and patient medical history specific to HAE attacks will be collected to assess eligibility. Each patient will receive three 4 week periods of treatment twice weekly.
Study design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| rhC1INH twice weekly | Experimental | Biological: Recombinant human C1 inhibitorrhC1INH administered twice weekly |
| rhC1INH once weekly | Experimental | Biological: Recombinant human C1 inhibitorrhC1INH administered once weekly |
| Placebo (Saline) twice weekly | Placebo comparator | Other: PlaceboPlacebo (Saline) administered twice weekly |
Who may qualify
- Ages
- 13 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- Patients fulfilling the following criteria at Screening are eligible for participation in the study:
- Age 13 years or older
- Laboratory confirmed diagnosis of HAE
- A history of frequent HAE attacks (at least 4 attacks per month across a minimum of 3 consecutive months).
- Female patients of childbearing potential who are sexually active must be willing to use an acceptable form of contraception.
Key exclusion criteria (7 listed)
- Patients who meet any of the following criteria at Screening are to be excluded from study participation:
- Patients with medical history of allergy to rabbits or rabbit-derived products (including rhC1INH)
- Diagnosis of acquired angioedema (AAE)
- Patients who are pregnant, or breastfeeding, or are currently intending to become pregnant
- Treatment with any investigational drug in the past 30 days
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 |
|---|---|---|
| Recombinant human C1 inhibitorAlso: rhC1INH, Ruconest, Conestat alfa | Biological | rhC1INH once weekly, rhC1INH twice weekly |
| PlaceboAlso: Saline | Other | Placebo (Saline) twice weekly |
Primary outcome
Number of HAE Attacks
Time frame: 28 days
Average number of HAE attacks normalized to a 28 day period
2 secondary outcomes
Number of Participants With Adverse Events
Time frame: 20 weeks
Percentage of Participants Achieving at Least 50% Reduction in Number of Attacks
Time frame: 28 days
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Dec 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- 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 28754491Riedl MA, Grivcheva-Panovska V, Moldovan D, Baker J, Yang WH, Giannetti BM, Reshef A, Andrejevic S, Lockey RF, Hakl R, Kivity S, Harper JR, Relan A, Cicardi M. Recombinant human C1 esterase inhibitor for prophylaxis of hereditary angio-oedema: a phase 2, multicentre, randomised, double-blind, placebo-controlled crossover trial. Lancet. 2017 Sep 30;390(10102):1595-1602. doi: 10.1016/S0140-6736(17)31963-3. Epub 2017 Jul 25.
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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.
The record lists no contact. Use the official record to reach the sponsor.
10 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Recombinant human C1 inhibitor in people with Hereditary Angioedema, run by Pharming Technologies B.V. at 10 sites in 7 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 7 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 8 Dec 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States, Canada, Czechia, Italy, North Macedonia and 2 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. 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 Dec 2017). 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?
Number of HAE Attacks (time frame: 28 days). 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 NCT02247739, which also lists 2 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 Dec 2017.