First-in-Human and Proof-of-Mechanism Study of ARC19499 Administered to Hemophilia Patients

NCT ID: NCT01191372

Last Updated: 2021-05-05

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE1
• Total Enrollment: 17 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2010-09-15
• Study Completion Date: 2011-12-28

Brief Summary

The purpose of this study is to examine the safety, tolerability and the way the body handles various single and multiple doses of ARC19499 in patients with hemophilia.

Conditions

Hemophilia

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

saline for injection

Group Type: PLACEBO_COMPARATOR

placebo control

Intervention Type: DRUG

sterile saline for injection

ARC19499 Low Dose

Group Type: EXPERIMENTAL

ARC19499

Intervention Type: DRUG

Anti-tissue factor pathway inhibitor (TFPI) aptamer

ARC19499 Mid Dose

Group Type: EXPERIMENTAL

ARC19499

Intervention Type: DRUG

Anti-tissue factor pathway inhibitor (TFPI) aptamer

ARC19499 High Dose

Group Type: EXPERIMENTAL

ARC19499

Intervention Type: DRUG

Anti-tissue factor pathway inhibitor (TFPI) aptamer

Interventions

placebo control

sterile saline for injection

Intervention Type: DRUG

ARC19499

Anti-tissue factor pathway inhibitor (TFPI) aptamer

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

Adult male patients ≥18 to ≤75 years of age.

• Hemophilia of any type or severity.
• Patients who are negative for hepatitis B surface antigen (HBsAg), and human immunodeficiency virus (HIV) I and II antibody tests at screening.
• Male patients who, with their partners, are willing to use 2 effective, methods of contraception (i.e., for both self and partner) throughout the study and for at least 3 months after discontinuation of study drug treatment.
• All patients must be capable of understanding and complying with the protocol and must have signed the informed consent document.

Exclusion Criteria

• Female patients;
• If on a prophylactic coagulation factor concentrate regimen, inability or unwillingness to discontinue prophylaxis during participation in this study.
• Existence of other co-existing bleeding disorder (e.g., von Willebrand Disease).
• Medical history of venous or arterial thromboembolism.
• Scheduled for elective surgical procedure during the conduct of this study.
• Use of an investigational drug within 30 days of study entry.
• Transaminase values > 3 x upper limit of normal (ULN) at time of screening.
• Haemoglobin <12.0 g/dL.
• Participants who, in the opinion of the Investigator, have a significant infection or known inflammatory process on screening.
• Participants who have a clinically relevant history or presence of respiratory, gastrointestinal, renal, hepatic, lymphatic, neurological, cardiovascular, psychiatric, musculoskeletal, genitourinary, immunological, dermatological, connective tissue diseases or disorders.
• Any medical condition the investigator believes would place the patient at increased risk as a result of participation in the study e.g. history of thromboembolic disease or stroke.
• Any medication the investigator considers may increase the risk of adverse effects during the study.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Baxalta now part of Shire

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Study Director

Role: STUDY_DIRECTOR

Takeda

Locations

London, , United Kingdom

Countries

United Kingdom

Other Identifiers

2010-020373-17

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

271101

Identifier Type: OTHER

Identifier Source: secondary_id

ARC19499-001

Identifier Type: -

Identifier Source: org_study_id