A Study to Evaluate the Safety and Pharmacokinetics of Two Formulations of C1-esterase Inhibitor

• ClinicalTrials.gov Identifier: NCT01760343
• Recruitment Status: Completed
• First Posted: January 4, 2013
• Last Update Posted: April 5, 2013
• Sponsor: CSL Behring
• Information provided by (Responsible Party): CSL Behring

Study Description

Brief Summary:

A new formulation of Berinert (CSL830) is being investigated for the management of hereditary angioedema (HAE). The main aim of the study is to assess the safety of a single 1500 IU dose of the new formulation of Berinert. This study will also look at the pharmacokinetics of CSL830 relative to Berinert currently on the market.

Condition or disease	Intervention/treatment	Phase
Hereditary Angioedema Types I and II	Biological: Berinert; Biological: CSL830	Phase 1

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 16 participants
• Allocation: Randomized
• Intervention Model: Crossover Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: A Randomized, Double-blind, Single-center, Cross-over Study to Evaluate the Safety, Bioavailability and Pharmacokinetics of Two Formulations of C1-esterase Inhibitor Administered Intravenously
• Study Start Date: January 2013
• Actual Primary Completion Date: March 2013
• Actual Study Completion Date: March 2013

Arms and Interventions

Arm	Intervention/treatment
Experimental: Berinert, then CSL830; A single intravenous dose of Berinert at 1500 units (1500 IU), followed by a single intravenous dose of CSL830 at 1500 IU.	Biological: Berinert; Berinert is a plasma-derived C1 esterase inhibitor (human), supplied as a freeze-dried powder for reconstitution.; Biological: CSL830; CSL830 is a formulation of Berinert.
Experimental: CSL830, then Berinert; A single intravenous dose of CSL830 at 1500 IU, followed by a single intravenous dose of Berinert at 1500 IU.	Biological: Berinert; Berinert is a plasma-derived C1 esterase inhibitor (human), supplied as a freeze-dried powder for reconstitution.; Biological: CSL830; CSL830 is a formulation of Berinert.

Outcome Measures

Primary Outcome Measures :

1. Incidence of adverse events (AEs) within 24 hours of CSL830 infusion [ Time Frame: From the start of infusion to 24 hours after the end of infusion ]

Secondary Outcome Measures :

1. Incidence of adverse events (AEs) within 10 days of the CSL830 infusion [ Time Frame: From the start of infusion to 10 days after the infusion ]
2. Relative bioavailability of CSL830 versus Berinert - Cmax [ Time Frame: 240 hours ]
   Relative bioavailability in terms of maximum concentration (Cmax) of CSL830 versus Berinert
3. Relative bioavailability of CSL830 versus Berinert - AUC [ Time Frame: 240 hours ]
   Relative bioavailability in terms of area under the curve from timepoint 0 to infinity (AUC0-∞) of CSL830 versus Berinert

Eligibility Criteria

• Ages Eligible for Study: 18 Years to 45 Years (Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: Yes

Criteria

Inclusion Criteria:

• Healthy subjects without clinically significant medical conditions or laboratory abnormalities
• Male or female subjects aged 18 to 45 years inclusive, at the time of informed consent
• Non-smokers
• Body mass index of 18.0 to 29.0 kg/m2 inclusive

Exclusion Criteria:

• Previous history of clinically significant arterial or venous thrombosis, current history of a clinically significant pro-thrombotic risk, or a clinically significant abnormality on laboratory thrombotic screen at the screening visit.
• Known or suspected hypersensitivity to the investigational medicinal product (IMP), or to any excipients of the IMP.
• Female subjects who started taking or changed dose of any hormonal contraceptive regimen or hormone replacement therapy (ie, estrogen/progesterone containing products) within 3 months before the screening visit.
• Alcohol, drug, or medication abuse within one year before the study.
• Female subjects of childbearing potential (eg, not post-menopausal) either not using, or not willing to use, a medically reliable method of contraception for the entire duration of the study, or have a vasectomized partner, or not sexually abstinent for the entire duration of the study, or not surgically sterile.
• Participation in another clinical study (or use of another IMP) within 30 days (or 5 times the half-life, whichever is longer) before, or during, the study.

Contacts and Locations

Locations

Germany

Study Site

Berlin, Germany

Sponsors and Collaborators

CSL Behring

Investigators

• Study Director: Global Clinical Program Director; CSL Behring

More Information

• Responsible Party: CSL Behring
• ClinicalTrials.gov Identifier: NCT01760343 History of Changes
• Other Study ID Numbers: CSL830_1001; 2012-002429-31 ( EudraCT Number )
• First Posted: January 4, 2013
• Last Update Posted: April 5, 2013
• Last Verified: April 2013

Additional relevant MeSH terms:

Angioedema; Angioedemas, Hereditary; Hereditary Angioedema Types I and II; Vascular Diseases; Cardiovascular Diseases; Urticaria; Skin Diseases, Vascular; Skin Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases; Genetic Diseases, Inborn; Complement C1s; Complement C1 Inhibitor Protein; Complement C1 Inactivator Proteins; Immunologic Factors; Physiological Effects of Drugs; Complement Inactivating Agents; Immunosuppressive Agents