Hereditary AngioedemaPhase 2NCT01426763
A Study to Evaluate the Safety and Pharmacology of Subcutaneous Administration of CINRYZE With Recombinant Human Hyaluronidase
A Phase 2 study testing CINRYZE with rHuPH20 in people with Hereditary Angioedema, run by Shire at 4 sites in United States.
Trial summary
The objectives of the study are to: 1. Evaluate the safety and tolerability of subcutaneously administered CINRYZE with recombinant human hyaluronidase (rHuPH20) in subjects with hereditary angioedema (HAE) who previously participated in CINRYZE Study 0624-200 (NCT01095497) 2. 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 with rHuPH20
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 4 sitesUnited States
- Study dates
- 12 Sep 2011to 28 Nov 2011
- 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:
- Provide informed consent/assent, as appropriate.
- Have previously participated in CINRYZE Study 0624-200 and completed the subcutaneous therapy period in that study.
- During the 3 consecutive months prior to screening, have a history of less than 1 HAE attack per month (average) that required treatment with C1 INH therapy or other blood products.
- Agree to avoid his/her known HAE triggers during the study to the best of his/her ability.
Who cannot join
- To be eligible for this protocol, a subject must not:
- Have received C1 INH therapy or any blood products for treatment or prevention of an HAE attack within 14 days prior to the first dose of study drug.
- Have received any ecallantide (Kalbitor), icatibant (Firazyr), or antifibrinolytics (e.g., tranexamic acid) within 14 days prior to the first dose of study drug.
- 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 of study drug.
- 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 of study drug.
- Have a history of abnormal blood clotting.
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- Have a history of allergic reaction to products containing C1 INH or other blood products.
- Have a known allergy to hyaluronidase or any other ingredient in rHuPH20.
- Be pregnant or breastfeeding.
- Have received an investigational study drug within 30 days prior to the first dose of study drug.
Trial locations and contacts
4 sites in 1 country.
United States4 sites
ViroPharma Investigational Site
Atlanta, Georgia, 30342
ViroPharma Investigational Site
Dallas, Texas, 75231
ViroPharma Investigational Site
Scottsdale, Arizona, 85251
ViroPharma Investigational Site
Walnut Creek, California, 94598
About this study
Official title: An Open-label Multiple-dose Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Administration of CINRYZE® (C1 Esterase Inhibitor [Human]) With Recombinant Human Hyaluronidase (rHuPH20) in Subjects With Hereditary Angioedema
The objectives of the study are to:
- Evaluate the safety and tolerability of subcutaneously administered CINRYZE with recombinant human hyaluronidase (rHuPH20) in subjects with hereditary angioedema (HAE) who previously participated in CINRYZE Study 0624-200 (NCT01095497)
- Characterize the pharmacokinetics and pharmacodynamics of subcutaneously administered CINRYZE with rHuPH20
- Assess the immunogenicity of CINRYZE following subcutaneous (SC) administration of CINRYZE with rHuPH20
- Conditions
- Hereditary Angioedema
- Being tested
- CINRYZE with rHuPH20
- Keywords
- HAE, C1 esterase inhibitor, C1 INH, Subcutaneous, hyaluronidase, rHuPH20
- Medical subject headings
- Angioedemas, Hereditary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CINRYZE with rHuPH20Biological
Also called C1 esterase inhibitor (human), Recombinant human hyaluronidase
| Study group | Role | What they receive |
|---|---|---|
| SC CINRYZE with rHuPH20 Dose Level 1 | Experimental | Subcutaneous injection of 1000 Units of CINRYZE with 20,000 Units of rHuPH20 twice weekly for two weeks Receives: Biological: CINRYZE with rHuPH20 |
| SC CINRYZE with rHuPH20 Dose Level 2 | Experimental | Subcutaneous injection of 2000 Units of CINRYZE with 40,000 Units of rHuPH20 twice weekly for two weeks Receives: Biological: CINRYZE with rHuPH20 |
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 Participants With Local Injection Site Reactions, and Number of Participants Who Discontinue Study Drug or Withdraw From the Study
Measured: 18 days
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
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
Number of Subjects With C1 INH Antibodies
Measured: Day 1 (pre-dose), Day 18 (168 h post Dose 4), and 30 (±2) days after the last dose of study drug (Dose 4)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Aug 2011 (estimated)
- Study start12 Sep 2011
- Primary completion28 Nov 2011
- Study completion28 Nov 2011
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
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Halozyme Therapeutics
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01426763
- Sponsor's protocol ID
- 0624-204
- Other IDs
- SPD616-204
- Status last verified
- Jun 2021
- First submitted
- 25 Aug 2011
- Last updated
- 19 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 19 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 Administration of CINRYZE® (C1 Esterase Inhibitor [Human]) With Recombinant Human Hyaluronidase (rHuPH20) in Subjects With Hereditary Angioedema
- ClinicalTrials.gov ID
- NCT01426763
- Acronym / sponsor ID
- 0624-204
- Conditions
- Hereditary Angioedema
- Keywords
- HAE, C1 esterase inhibitor, C1 INH, Subcutaneous, hyaluronidase, rHuPH20
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- ShireIndustry (pharma, biotech, devices)
- Collaborators
- Halozyme Therapeutics
- Study start
- 12 Sep 2011 (actual)
- Primary completion
- 28 Nov 2011 (actual)
- Study completion
- 28 Nov 2011 (actual)
- First posted
- 31 Aug 2011 (estimated)
- Results
- Posted 4 Jan 2013
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 subcutaneously administered CINRYZE with recombinant human hyaluronidase (rHuPH20) in subjects with hereditary angioedema (HAE) who previously participated in CINRYZE Study 0624-200 (NCT01095497)
- Characterize the pharmacokinetics and pharmacodynamics of subcutaneously administered CINRYZE with rHuPH20
- Assess the immunogenicity of CINRYZE following subcutaneous (SC) administration of CINRYZE with rHuPH20
Study design
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| SC CINRYZE with rHuPH20 Dose Level 1 | Experimental | Biological: CINRYZE with rHuPH20Subcutaneous injection of 1000 Units of CINRYZE with 20,000 Units of rHuPH20 twice weekly for two weeks |
| SC CINRYZE with rHuPH20 Dose Level 2 | Experimental | Biological: CINRYZE with rHuPH20Subcutaneous injection of 2000 Units of CINRYZE with 40,000 Units of rHuPH20 twice weekly for two weeks |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Provide informed consent/assent, as appropriate.
- Have previously participated in CINRYZE Study 0624-200 and completed the subcutaneous therapy period in that study.
- During the 3 consecutive months prior to screening, have a history of less than 1 HAE attack per month (average) that required treatment with C1 INH therapy or other blood products.
- Agree to avoid his/her known HAE triggers during the study to the best of his/her ability.
Key exclusion criteria (10 listed)
- Have received C1 INH therapy or any blood products for treatment or prevention of an HAE attack within 14 days prior to the first dose of study drug.
- Have received any ecallantide (Kalbitor), icatibant (Firazyr), or antifibrinolytics (e.g., tranexamic acid) within 14 days prior to the first dose of study drug.
- 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 of study drug.
- 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 of study drug.
- Have a history of abnormal blood clotting.
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 |
|---|---|---|
| CINRYZE with rHuPH20Also: C1 esterase inhibitor (human), Recombinant human hyaluronidase | Biological | SC CINRYZE with rHuPH20 Dose Level 1, SC CINRYZE with rHuPH20 Dose Level 2 |
Primary outcome
Incidence and Severity of Adverse Events, Number of Participants With Local Injection Site Reactions, and Number of Participants Who Discontinue Study Drug or Withdraw From the Study
Time frame: 18 days
3 secondary outcomes
Mean Change C1 Inhibitor (C1INH)
Time frame: 18 days
Mean Change C4 Compliment
Time frame: 18 days
Number of Subjects With C1 INH Antibodies
Time frame: Day 1 (pre-dose), Day 18 (168 h post Dose 4), and 30 (±2) days after the last dose of study drug (Dose 4)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 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
4 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 with rHuPH20 in people with Hereditary Angioedema, run by Shire at 4 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 5 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 19 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 4 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 4 Jan 2013). 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 Participants With Local Injection Site Reactions, and Number of Participants Who Discontinue Study Drug or Withdraw From the Study (time frame: 18 days). 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 19 Jul 2021.