Hereditary AngioedemaPhase 2NCT01095497
A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous CINRYZE Administration
A Phase 2 study testing CINRYZE in people with Hereditary Angioedema, run by Shire at 7 sites in United States.
Trial summary
The objectives of the study are to: 1. Evaluate the safety and tolerability of CINRYZE administered by subcutaneous injection in subjects with hereditary angioedema 2. Characterize the pharmacokinetics and pharmacodynamics of CINRYZE administered by subcutaneous injection 3. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Hereditary Angioedema
- What is being tested?
- CINRYZE
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 26 participants
- Where
- 7 sitesUnited States
- Study dates
- 7 Jun 2010to 16 Dec 2010
- 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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- To be eligible for this protocol, a subject must:
- Have a confirmed diagnosis of HAE.
- During the 3 consecutive months prior to screening, have a history of less than 1 HAE attack per month (average) treated with C1INH therapy or any other blood products, ecallantide (Kalbitor), icatibant (Firazyr), antifibrinolytics (e.g., tranexamic acid), IV fluids, or narcotic analgesics.
- Agree to strictly adhere to the protocol-defined schedule of assessments and procedures.
Who cannot join
- To be eligible for this protocol, a subject must not:
- Have received C1INH therapy or any blood products for treatment or prevention of an HAE attack within 14 days prior to the first dose.
- Have received any ecallantide (Kalbitor), icatibant (Firazyr), or antifibrinolytics (e.g., tranexamic acid) within 14 days prior to the first dose.
- Have any change (start, stop, or change in dose) in androgen therapy (e.g., danazol, oxandrolone, stanozolol, testosterone) within 14 days prior to the first dose.
- If female, have started taking or changed the dose of any hormonal contraceptive regimen or hormone replacement therapy (i.e., estrogen/progestin containing products) within 3 months prior to the first dose.
- Have a history of abnormal blood clotting or other coagulopathy.
Show all 10Show fewer
- Have a history of allergic reaction to CINRYZE or other blood products.
- Be pregnant or breastfeeding.
- Have received an immunization within 30 days prior to the first dose.
- Have participated in any other investigational drug study within 30 days prior to the first dose.
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
United States7 sites
Family Allergy and Asthma Center
Atlanta, Georgia, 30342
Institute for Asthma and Allergy
Chevy Chase, Maryland, 20815
AARA Research Center
Dallas, Texas, 75231
Allergy, Asthma and Dermatology Research Center
Lake Oswego, Oregon, 97035
Allergy, Asthma and Immunology Associates
Scottsdale, Arizona, 85251
Marycliff Allergy Specialists
Spokane, Washington, 99204
Allergy and Asthma Clinical Research, Inc.
Walnut Creek, California, 94598
About this study
Official title: An Open-Label Multiple-Dose Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous (SC) Versus Intravenous (IV) Administration of CINRYZE in Adolescents and Adults With Hereditary Angioedema (HAE)
The objectives of the study are to:
- Evaluate the safety and tolerability of CINRYZE administered by subcutaneous injection in subjects with hereditary angioedema
- Characterize the pharmacokinetics and pharmacodynamics of CINRYZE administered by subcutaneous injection
- Assess the immunogenicity of CINRYZE following subcutaneous administration
- Conditions
- Hereditary Angioedema
- Being tested
- CINRYZE
- Keywords
- HAE, C1 inhibitor (C1INH), Subcutaneous
- 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
C1 esterase inhibitor (human)
| Study group | Role | What they receive |
|---|---|---|
| IV CINRYZE First, Then SC CINRYZE Dose 1 | Experimental | Not described Receives: Biological: CINRYZE |
| IV CINRYZE First, Then SC CINRYZE Dose 2 | Experimental | Not described Receives: Biological: CINRYZE |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and Severity of Adverse Events, Number of Subjects With Local Injection Site Reactions, and Number of Subjects Who Discontinue Study Drug or Withdraw From the Study.
Measured: 18 days in each treatment period
Secondary outcomes (3)
Mean Change C1 Inhibitor (C1INH)
Mean Change in Baseline in Observed Plasma Concentration of C1 Inhibitor (C1INH) Antigen. Baseline-corrected concentrations were derived by subtracting the observed pre-dose concentrations on Day 1 of each treatment period from each observed concentration.
Measured: 18 days in each treatment period
Mean Change C4 Compliment
Mean Change in Baseline in Observed Plasma Concentration of C4 Compliment. Baseline-corrected concentrations were derived by subtracting the observed pre-dose concentrations on Day 1 of each treatment period from each observed concentration.
Measured: 18 days in each treatment period
Number of Participants With C1 Inhibitor (INH) Antibodies
Measured: 18 days in each treatment period
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 start7 Jun 2010
- Primary completion16 Dec 2010
- Study completion16 Dec 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.
- 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
- Other
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 26 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
- NCT01095497
- Sponsor's protocol ID
- 0624-200
- Other IDs
- SPD616-200
- Status last verified
- Jun 2021
- First submitted
- 22 Feb 2010
- Last updated
- 9 Jul 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Jul 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Multiple-Dose Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous (SC) Versus Intravenous (IV) Administration of CINRYZE in Adolescents and Adults With Hereditary Angioedema (HAE)
- ClinicalTrials.gov ID
- NCT01095497
- Acronym / sponsor ID
- 0624-200
- Conditions
- Hereditary Angioedema
- Keywords
- HAE, C1 inhibitor (C1INH), Subcutaneous
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 26 participants (actual)
- Lead sponsor
- ShireIndustry (pharma, biotech, devices)
- Study start
- 7 Jun 2010 (actual)
- Primary completion
- 16 Dec 2010 (actual)
- Study completion
- 16 Dec 2010 (actual)
- First posted
- 30 Mar 2010 (estimated)
- Results
- Posted 8 Mar 2012
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 the study are to:
- Evaluate the safety and tolerability of CINRYZE administered by subcutaneous injection in subjects with hereditary angioedema
- Characterize the pharmacokinetics and pharmacodynamics of CINRYZE administered by subcutaneous injection
- Assess the immunogenicity of CINRYZE following subcutaneous administration
Study design
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| IV CINRYZE First, Then SC CINRYZE Dose 1 | Experimental | Biological: CINRYZE |
| IV CINRYZE First, Then SC CINRYZE Dose 2 | Experimental | Biological: CINRYZE |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Have a confirmed diagnosis of HAE.
- During the 3 consecutive months prior to screening, have a history of less than 1 HAE attack per month (average) treated with C1INH therapy or any other blood products, ecallantide (Kalbitor), icatibant (Firazyr), antifibrinolytics (e.g., tranexamic acid), IV fluids, or narcotic analgesics.
- Agree to strictly adhere to the protocol-defined schedule of assessments and procedures.
Key exclusion criteria (10 listed)
- Have received C1INH therapy or any blood products for treatment or prevention of an HAE attack within 14 days prior to the first dose.
- Have received any ecallantide (Kalbitor), icatibant (Firazyr), or antifibrinolytics (e.g., tranexamic acid) within 14 days prior to the first dose.
- Have any change (start, stop, or change in dose) in androgen therapy (e.g., danazol, oxandrolone, stanozolol, testosterone) within 14 days prior to the first dose.
- If female, have started taking or changed the dose of any hormonal contraceptive regimen or hormone replacement therapy (i.e., estrogen/progestin containing products) within 3 months prior to the first dose.
- Have a history of abnormal blood clotting or other coagulopathy.
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 |
|---|---|---|
| CINRYZEC1 esterase inhibitor (human) | Biological | IV CINRYZE First, Then SC CINRYZE Dose 1, IV CINRYZE First, Then SC CINRYZE Dose 2 |
Primary outcome
Incidence and Severity of Adverse Events, Number of Subjects With Local Injection Site Reactions, and Number of Subjects Who Discontinue Study Drug or Withdraw From the Study.
Time frame: 18 days in each treatment period
3 secondary outcomes
Mean Change C1 Inhibitor (C1INH)
Time frame: 18 days in each treatment period
Mean Change C4 Compliment
Time frame: 18 days in each treatment period
Number of Participants With C1 Inhibitor (INH) Antibodies
Time frame: 18 days in each treatment period
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Jul 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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
7 sites in 1 country: 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, run by Shire at 7 sites in United States.
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 10 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 9 Jul 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United States. 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 8 Mar 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?
Incidence and Severity of Adverse Events, Number of Subjects With Local Injection Site Reactions, and Number of Subjects Who Discontinue Study Drug or Withdraw From the Study. (time frame: 18 days in each treatment period). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 9 Jul 2021.