A Study of KW-3357 in Congenital Antithrombin Deficiency

A Phase I Study to Determine the Pharmacokinetic Profile, Safety and Tolerability of a Single Dose (50IU/kg) of KW-3357 in Subjects With Congenital Antithrombin Deficiency.

The aim of this study is to determine the pharmacokinetics, safety and tolerability of KW-3357 in asymptomatic subjects with congenital antithrombin deficiency.

Study Overview

• Status: Completed
• Conditions: Congenital Antithrombin Deficiency
• Intervention / Treatment: Drug: KW-3357

Detailed Description

Patients with Congenital Antithrombin Deficiency are at increased risk of venous thrombosis and pulmonary embolism especially when undergoing certain high risk procedures. Antithrombin replacement therapy is often administered during these periods, with or without low molecular weight heparin. Prior to assessing the efficacy of KW-3357, a new recombinant human antithrombin, the present study will determine it's pharmacokinetics, safety and tolerability in subjects who have Congenital Antithrombin Deficiency but who are currently asymptomatic and not undergoing a high-risk procedure. Up to 16 evaluable subjects will be enrolled at multiple investigational sites over a period of approximately 7 months.

• Study Type: Interventional
• Enrollment (Anticipated): 16
• Phase: Phase 1

Contacts and Locations

Study Locations

• France
  • Further details on request, France
• Germany
  • Further details on request, Germany
• Italy
  • Further details on request, Italy
• Sweden
  • Further details on request, Sweden
• United Kingdom
  • Further details on request, United Kingdom

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Male or female subjects at least 18 years of age with congenital Antithrombin deficiency (AT activity ≤60% of normal) in a stable condition without evidence of acute thromboembolic events
• Signed IEC-approved Informed Consent Form
• Subjects must not have received an infusion of Antithrombin for at least 14 days before Screening
• Patients of reproductive potential must agree to follow accepted birth control methods during the study

Exclusion Criteria:

• Subjects who are classified as morbidly obese (defined by the presence of a body mass index >40 kg/m2)
• Subjects who have participated in a study with an investigational drug within 30 days of Screening or within 5.5 times the elimination half-life of the investigational drug before Screening, whichever period is greater
• Subjects with any clinically relevant medical history or current condition or physical findings, ECG, or laboratory values which could interfere with the objectives of the study or the safety of the subject
• Subjects using non-steroidal anti-inflammatories, fondaparinux sodium, dabigatran or rivaroxaban or who are expected to be treated with these drugs during the study
• Subjects who have concomitant nephrotic syndrome
• Female subjects who are pregnant or lactating
• Subjects who are taking heparin, low molecular weight heparin and/or oral anticoagulants, with the exception of vitamin K antagonists (eg, warfarin)

Study Plan

How is the study designed?

Design Details

• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Other: 1; Single group	Drug: KW-3357; 50IU/mL, IV single dose

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
To evaluate the pharmacokinetic profile of a single dose (50 IU/kg) of KW 3357 in subjects with congenital AT deficiency	July 2011

Secondary Outcome Measures

Outcome Measure	Time Frame
To determine the safety and tolerability of a single dose (50 IU/kg) of KW-3357 in subjects with congenital AT deficiency	July 2011

Collaborators and Investigators

• Sponsor: Kyowa Hakko Kirin UK, Ltd.
• Investigators: Principal Investigator: Beverley Hunt, FRCP, FRCPath MD, St Thomas' Hospital, London, UK

Study record dates

Study Major Dates

• Study Start: April 1, 2009
• Primary Completion (Actual): December 1, 2010
• Study Completion (Actual): April 1, 2011

Study Registration Dates

• First Submitted: July 10, 2009
• First Submitted That Met QC Criteria: July 10, 2009
• First Posted (Estimate): July 13, 2009

Study Record Updates

• Last Update Posted (Estimate): June 17, 2011
• Last Update Submitted That Met QC Criteria: June 15, 2011
• Last Verified: March 1, 2011

More Information

Terms related to this study

• Keywords: Pharmacokinetics; Thrombosis; Antithrombin; Hereditary Antithrombin Deficiency; Congenital Antithrombin Deficiency
• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Genetic Diseases, Inborn; Blood Protein Disorders; Blood Coagulation Disorders; Thrombophilia; Antithrombin III Deficiency
• Other Study ID Numbers: 3357-EU-001; EudraCT number 2008-005504-16