Hereditary Angioedema Types I and II Clinical Trial
Official title:
An Open-label, Cross-over, Dose-ranging Study to Evaluate the Pharmacokinetics, Pharmacodynamics and Safety of the Subcutaneous Administration of a Human Plasma-derived C1-esterase Inhibitor in Subjects With Hereditary Angioedema
Verified date | January 2021 |
Source | CSL Behring |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
The aim of the study is to assess what happens to C1-esterase inhibitor that is administered under the skin of subjects with hereditary angioedema. Three different dosing regimens of C1-esterase inhibitor will be assessed. Each subject will be assigned to receive 2 of the 3 dosing regimens, each for 4 weeks. The activity and concentration of C1-esterase inhibitor in the blood will be measured during each 4-week period. The study will also examine how well C1-esterase inhibitor administered under the skin is tolerated by the subjects.
Status | Completed |
Enrollment | 18 |
Est. completion date | December 2012 |
Est. primary completion date | December 2012 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years and older |
Eligibility | Inclusion Criteria: - Males or females aged 18 years or older. - Laboratory-confirmed hereditary angioedema type I or II. - Less than two hereditary angioedema attacks per month in the last three months. - Body weight of 50.0 kg to 110.0 kg. Exclusion Criteria: - Receiving prophylactic C1-esterase inhibitor therapy. - Received C1-esterase inhibitor, ecallantide, icatibant or any blood products for the prevention or treatment of hereditary angioedema within 7 days before the screening visit. - Intends to use recombinant C1-esterase inhibitor or fresh frozen plasma for the acute treatment of hereditary angioedema during the study. - Received androgen therapy (e.g., danazol, oxandrolone, stanozolol, testosterone) within 30 days before the screening visit. - Female subjects who started taking or changed dose of any hormonal contraceptive regimen or hormone replacement therapy (i.e., estrogen/progesterone-containing products) within 3 months prior to the screening visit. - Known or suspected hypersensitivity to the study product, or to any excipients of the study product. - Pregnancy or lactation. |
Country | Name | City | State |
---|---|---|---|
Germany | Study Site | Berlin | |
Germany | Study Site | Frankfurt | |
Germany | Study Site | Mainz | |
United States | Study Site | Atlanta | Georgia |
United States | Study Site | Chevy Chase | Maryland |
United States | Study Site | Cincinnati | Ohio |
United States | Study Site | Hershey | Pennsylvania |
United States | Study Site | Toledo | Ohio |
Lead Sponsor | Collaborator |
---|---|
CSL Behring | Parexel |
United States, Germany,
Zuraw BL, Cicardi M, Longhurst HJ, Bernstein JA, Li HH, Magerl M, Martinez-Saguer I, Rehman SM, Staubach P, Feuersenger H, Parasrampuria R, Sidhu J, Edelman J, Craig T. Phase II study results of a replacement therapy for hereditary angioedema with subcuta — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Modeled C1-esterase Inhibitor Functional Activity Trough Level | Mean trough C1-esterase inhibitor functional activity of the low, medium and high subcutaneous dose regimens, based on modeling and simulation | at the fourth week of each dosing regimen | |
Secondary | As-observed C1-esterase Inhibitor Functional Activity Trough Level | Mean trough C1-esterase inhibitor functional activity of the low, medium and high subcutaneous dose regimens | during the last week of 4-week dose regimen | |
Secondary | C1-esterase Inhibitor Concentration Trough Level | Mean trough C1-esterase inhibitor concentration of the low, medium and high subcutaneous dose regimens | during the last week of 4-week dose regimen | |
Secondary | C4 Concentration Trough Level | Mean trough C4 concentration of the low, medium and high subcutaneous dose regimens | during the last week of 4-week dose regimen | |
Secondary | Change From Baseline in C1-esterase Inhibitor Functional Activity | Mean change from baseline of C1-esterase inhibitor functional activity of the low, medium and high subcutaneous dose regimens | Baseline and during the last week of 4-week dose regimen | |
Secondary | Change From Baseline in C1-esterase Inhibitor Concentration | Mean change from baseline of C1-esterase inhibitor concentration of the low, medium and high subcutaneous dose regimens | Baseline and during the last week of 4-week dose regimen | |
Secondary | Change From Baseline in C4 Concentration | Mean change from baseline of C4 concentration of the low, medium and high subcutaneous dose regimens | Baseline and during the last week of 4-week dose regimen |
Status | Clinical Trial | Phase | |
---|---|---|---|
Completed |
NCT01467947 -
Postmarketing Immunogenicity Study in HAE Subjects Treated With Berinert
|
Phase 4 | |
Completed |
NCT01912456 -
A Study to Evaluate the Clinical Efficacy and Safety of Subcutaneously Administered C1-esterase Inhibitor in the Prevention of Hereditary Angioedema
|
Phase 3 | |
Completed |
NCT01760343 -
A Study to Evaluate the Safety and Pharmacokinetics of Two Formulations of C1-esterase Inhibitor
|
Phase 1 | |
Completed |
NCT04618211 -
Dose-ranging Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema
|
Phase 2 | |
Recruiting |
NCT06343779 -
Study of Oral Deucrictibant Soft Capsule for On-Demand Treatment of Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema
|
Phase 3 | |
Completed |
NCT02316353 -
A Study to Evaluate the Long-term Clinical Safety and Efficacy of Subcutaneously Administered C1-esterase Inhibitor in the Prevention of Hereditary Angioedema
|
Phase 3 | |
Recruiting |
NCT05396105 -
Extension Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema
|
Phase 2/Phase 3 | |
Withdrawn |
NCT01832896 -
Study to Assess the Tolerability and Safety of Ecallantide in Children and Adolescents With Hereditary Angioedema
|
Phase 2 | |
Active, not recruiting |
NCT05047185 -
Dose-ranging Study of Oral PHA-022121 for Prophylaxis Against Angioedema Attacks in Patients With Hereditary Angioedema Type I or Type II
|
Phase 2 |