Hereditary Angioedema (HAE)Phase 2NCT01095510
CINRYZE for the Treatment of Hereditary Angioedema Attacks in Children Under the Age of 12
A Phase 2 study testing CINRYZE in people with Hereditary Angioedema (HAE), run by Shire at 11 sites in 3 countries.
Trial summary
The objectives of this study were to evaluate: (1) the dose response and (2) the pharmacokinetics (PK) and pharmacodynamics (PD) of intravenous (IV) administration of CINRYZE for the treatment of acute angioedema attacks in children above and below 25 kg and less than 12 years of age with hereditary angioedema (HAE)… Read more about this study
- Who is it for?
- Ages 2 to 11 years
- Conditions the study is for
- Hereditary Angioedema (HAE)
- What is being tested?
- CINRYZE
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 11 sitesin 3 countries
- Study dates
- 2 Jun 2010to 17 Apr 2012
- 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 2 to 11 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- To be eligible for this protocol, subjects must:
- Be at least 10 kg of body weight.
- Have a confirmed diagnosis of HAE.
- Have an acute HAE attack and be able to initiate treatment within 8 hours after onset of symptoms.
Who cannot join
- To be eligible for this protocol, subjects must not:
- Have any active infectious illness.
- Have had a prior HAE attack and/or received any C1 INH product within 7 days prior to dosing with study drug.
- Have received therapy with antifibrinolytics (e.g., tranexamic acid), androgens (e.g., danazol, oxandrolone, stanozolol, or testosterone), ecallantide (Kalbitor®), or icatibant (Firazyr®) within 7 days prior to dosing with study drug.
- Have a history of allergic reaction to C1 INH products, including CINRYZE (or any of the components of CINRYZE), or other blood products.
- Have participated in any other investigational drug evaluation within 30 days prior to dosing with study drug, or have previously received treatment with CINRYZE in this study at any time.
Trial locations and contacts
11 sites in 3 countries.
11 sites in 3 countries
United States8 sites
Institute for Asthma and Allergy, PC
Chevy Chase, Maryland, 20815
Asthma & Allergy Associates, P.C.
Colorado Springs, Colorado, 80907
AARA Research Center
Dallas, Texas, 75231
Allergy & Asthma Research Group
Eugene, Oregon, 97401
Baker Allergy, Asthma and Dermatology Research Center, LLC
Lake Oswego, Oregon, 97035
Allergy and Asthma Research Center, P.A.
San Antonio, Texas, 78229
Marycliff Allergy Specialists
Spokane, Washington, 99204
University of South Florida Asthma, Allergy and Immunology Clinical Research Unit
Tampa, Florida, 33613
Germany2 sites
Charité Universitätsmedizin Berlin, Dept. of Dermatology and Allergy
Berlin
Klinikum rechts der Isar, Technical University Munich, ENT Clinic
Munich
Hungary1 site
Semmelweis University, Allergy and Angioedema Outpatients Clinic, Kútvölgyi Clinical Center
Budapest
About this study
Official title: Open-Label, Single-Dose Study to Evaluate the Response and Pharmacokinetics/Pharmacodynamics of Different Doses of CINRYZE® [C1 Inhibitor (Human)] For Treatment of Acute Angioedema Attacks in Children Less Than 12 Years of Age With Hereditary Angioedema
The objectives of this study were to evaluate: (1) the dose response and (2) the pharmacokinetics (PK) and pharmacodynamics (PD) of intravenous (IV) administration of CINRYZE for the treatment of acute angioedema attacks in children above and below 25 kg and less than 12 years of age with hereditary angioedema (HAE); and (3) to determine the safety and tolerability following IV administration of CINRYZE in this study population.
Read the detailed description
Each subject received CINRYZE for treatment of a single acute angioedema attack.
- Conditions
- Hereditary Angioedema (HAE)
- Being tested
- CINRYZE
- Keywords
- CINRYZE, C1 INH, HAE, C1 inhibitor, Pediatric, PK/PD
- Medical subject headings
- Angioedemas, Hereditary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CINRYZEBiological
Also called C1 inhibitor [human]
| Study group | Role | What they receive |
|---|---|---|
| 500 U CINRYZE (10-25 kg body weight) | Experimental | Single IV dose of 500 U CINRYZE Receives: Biological: CINRYZE |
| 1000 U CINRYZE (10-25 kg body weight) | Experimental | Single IV dose of 1000 U CINRYZE Receives: Biological: CINRYZE |
| 1000 U CINRYZE (>25 kg body weight) | Experimental | Single IV dose of 1000 U CINRYZE Receives: Biological: CINRYZE |
| 1500 U CINRYZE (>25 kg body weight) | Experimental | Single IV dose of 1500 U CINRYZE Receives: Biological: CINRYZE |
What is being measured
The primary outcome is the main question the study is designed to answer.
Presence of Unequivocal Beginning of Relief of the Defining Attack Symptom
Measured: Within 4 hours following treatment
Secondary outcomes (3)
Time to Unequivocal Beginning of Relief of the Defining Attack Symptom
Measured: Within 4 hours following treatment
Time to Complete Resolution of the Attack
Measured: Within 1 week following treatment
Change in C1 Inhibitor (C1 INH) Antigen and Functional C1 INH Concentrations
Data was not reported due to change in planned analysis.
Measured: Pre-dose, 2, 4, 8 hours post dose on Day 1; Day 2, 3, 5, 8
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Mar 2010 (estimated)
- Study start2 Jun 2010
- Primary completion17 Apr 2012
- Study completion17 Apr 2012
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 (1)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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 9 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
- NCT01095510
- Sponsor's protocol ID
- 0624-203
- Other IDs
- 2011-000369-11
- Status last verified
- May 2021
- First submitted
- 24 Mar 2010
- Last updated
- 3 Jun 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Lumry W, Soteres D, Gower R, Jacobson KW, Li HH, Chen H, Schranz J. Safety and efficacy of C1 esterase inhibitor for acute attacks in children with hereditary angioedema. Pediatr Allergy Immunol. 2015 Nov;26(7):674-80. doi: 10.1111/pai.12444. Epub 2015 Aug 11.
PubMed 26171584
Study at a glance
From the registry record, last updated by the sponsor on 3 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open-Label, Single-Dose Study to Evaluate the Response and Pharmacokinetics/Pharmacodynamics of Different Doses of CINRYZE® [C1 Inhibitor (Human)] For Treatment of Acute Angioedema Attacks in Children Less Than 12 Years of Age With Hereditary Angioedema
- ClinicalTrials.gov ID
- NCT01095510
- Acronym / sponsor ID
- 0624-203
- Conditions
- Hereditary Angioedema (HAE)
- Keywords
- CINRYZE, C1 INH, HAE, C1 inhibitor, Pediatric, PK/PD
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- ShireIndustry (pharma, biotech, devices)
- Study start
- 2 Jun 2010 (actual)
- Primary completion
- 17 Apr 2012 (actual)
- Study completion
- 17 Apr 2012 (actual)
- First posted
- 30 Mar 2010 (estimated)
- Results
- Posted 25 Jul 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The objectives of this study were to evaluate: (1) the dose response and (2) the pharmacokinetics (PK) and pharmacodynamics (PD) of intravenous (IV) administration of CINRYZE for the treatment of acute angioedema attacks in children above and below 25 kg and less than 12 years of age with hereditary angioedema (HAE); and (3) to determine the safety and tolerability following IV administration of CINRYZE in this study population.
Detailed description
Each subject received CINRYZE for treatment of a single acute angioedema attack.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| 500 U CINRYZE (10-25 kg body weight) | Experimental | Biological: CINRYZESingle IV dose of 500 U CINRYZE |
| 1000 U CINRYZE (10-25 kg body weight) | Experimental | Biological: CINRYZESingle IV dose of 1000 U CINRYZE |
| 1000 U CINRYZE (>25 kg body weight) | Experimental | Biological: CINRYZESingle IV dose of 1000 U CINRYZE |
| 1500 U CINRYZE (>25 kg body weight) | Experimental | Biological: CINRYZESingle IV dose of 1500 U CINRYZE |
Who may qualify
- Ages
- 2 to 11 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Be at least 10 kg of body weight.
- Have a confirmed diagnosis of HAE.
- Have an acute HAE attack and be able to initiate treatment within 8 hours after onset of symptoms.
Key exclusion criteria (6 listed)
- Have any active infectious illness.
- Have had a prior HAE attack and/or received any C1 INH product within 7 days prior to dosing with study drug.
- Have received therapy with antifibrinolytics (e.g., tranexamic acid), androgens (e.g., danazol, oxandrolone, stanozolol, or testosterone), ecallantide (Kalbitor®), or icatibant (Firazyr®) within 7 days prior to dosing with study drug.
- Have a history of allergic reaction to C1 INH products, including CINRYZE (or any of the components of CINRYZE), or other blood products.
- Have participated in any other investigational drug evaluation within 30 days prior to dosing with study drug, or have previously received treatment with CINRYZE in this study at any time.
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 |
|---|---|---|
| CINRYZEAlso: C1 inhibitor [human] | Biological | 1000 U CINRYZE (10-25 kg body weight), 1000 U CINRYZE (>25 kg body weight), 1500 U CINRYZE (>25 kg body weight), 500 U CINRYZE (10-25 kg body weight) |
Primary outcome
Presence of Unequivocal Beginning of Relief of the Defining Attack Symptom
Time frame: Within 4 hours following treatment
3 secondary outcomes
Time to Unequivocal Beginning of Relief of the Defining Attack Symptom
Time frame: Within 4 hours following treatment
Time to Complete Resolution of the Attack
Time frame: Within 1 week following treatment
Change in C1 Inhibitor (C1 INH) Antigen and Functional C1 INH Concentrations
Time frame: Pre-dose, 2, 4, 8 hours post dose on Day 1; Day 2, 3, 5, 8
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Jun 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 26171584Lumry W, Soteres D, Gower R, Jacobson KW, Li HH, Chen H, Schranz J. Safety and efficacy of C1 esterase inhibitor for acute attacks in children with hereditary angioedema. Pediatr Allergy Immunol. 2015 Nov;26(7):674-80. doi: 10.1111/pai.12444. Epub 2015 Aug 11.
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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
11 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing CINRYZE in people with Hereditary Angioedema (HAE), run by Shire at 11 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 6 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 3 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 11 sites in United States, Germany, Hungary. 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 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 25 Jul 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?
Presence of Unequivocal Beginning of Relief of the Defining Attack Symptom (time frame: Within 4 hours following treatment). 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 NCT01095510, which also lists 1 linked publication.
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 3 Jun 2021.