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

NCT01191372 | Terminated Phase 1

• 3 other identifiers: ARC19499-0012010-020373-17271101
• Study Type: interventional
• Target: 17
• Locations: 1 country
• Sites: 1

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

Keywords

Hemophilia A; Hemophilia B; pharmacokinetics; pharmacodynamics; Tissue Factor Pathway Inhibitor

Outcome Measures

Primary Outcomes (1)

• Pharmacokinetics (PK) of ARC19499

  The PK profile of ARC19499 administered by single and multiple subcutaneous injections will be characterized. The bioavailability of subcutaneously injected ARC19499 relative to that of intravenously infused ARC19499 will be determined.

  2 weeks

Secondary Outcomes (1)

• Coagulation system pharmacodynamic (PD) effects of ARC19499.

  2 weeks

Study Arms (4)

saline for injection

PLACEBO COMPARATOR

Drug: placebo control

ARC19499 Low Dose

EXPERIMENTAL

Drug: ARC19499

ARC19499 Mid Dose

EXPERIMENTAL

Drug: ARC19499

ARC19499 High Dose

EXPERIMENTAL

Drug: ARC19499

Interventions

placebo control DRUG

sterile saline for injection

saline for injection

ARC19499 DRUG

Anti-tissue factor pathway inhibitor (TFPI) aptamer

ARC19499 High Dose; ARC19499 Low Dose; ARC19499 Mid Dose

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Baxalta now part of Shire: lead

Study Sites (1)

Unknown Facility

London, United Kingdom

Location

MeSH Terms

Conditions

Hemophilia A; Hemophilia B

Interventions

ARC19499

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 26, 2010
• First Posted: August 30, 2010
• Study Start: September 15, 2010
• Primary Completion: December 28, 2011
• Study Completion: December 28, 2011
• Last Updated: May 5, 2021

Record last verified: 2021-05

Locations

GB (1)