A Study of the Pharmacokinetics, Pharmacodynamics, and Safety of ARC1779 Injection in Patients With Von Willebrand Disease Type 2B

This study has been withdrawn prior to enrollment.
(Sponsor decided not to go forward with the study.)
Sponsor:
Information provided by:
Archemix Corp.
ClinicalTrials.gov Identifier:
NCT00694785
First received: June 6, 2008
Last updated: August 20, 2009
Last verified: August 2009

June 6, 2008
August 20, 2009
October 2008
June 2009   (final data collection date for primary outcome measure)
To evaluate the effect of ARC1779 Injection on platelet counts in vWD-2B patients who have thrombocytopenia at baseline. [ Time Frame: 10 days ] [ Designated as safety issue: No ]
Same as current
Complete list of historical versions of study NCT00694785 on ClinicalTrials.gov Archive Site
To assess the concentration-response relationships among ARC1779 pharmacokinetic (PK) and pharmacodynamic (PD) parameters [ Time Frame: 10 days ] [ Designated as safety issue: No ]
Same as current
Not Provided
Not Provided
 
A Study of the Pharmacokinetics, Pharmacodynamics, and Safety of ARC1779 Injection in Patients With Von Willebrand Disease Type 2B
A Study of the Pharmacokinetics, Pharmacodynamics, and Safety of ARC1779 Injection in Patients With Von Willebrand Disease Type 2B

To evaluate the effect of ARC1779, a therapeutic oligonucleotide ("aptamer") in patients with Type2B von Willebrand Disease.

ARC1779 will be investigated in an open-label, uncontrolled study in up to 3 vWD-2B patients. Patients with vWD-2B will be screened for eligibility based primarily upon a single major criterion, i.e., presence of any degree of chronic thrombocytopenia. Eligible patients will be treated by intravenous infusion of ARC1779 for 72 hours.

Interventional
Phase 2
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Von Willebrand Disease
  • Drug: ARC1779

    Initial stepwise infusion of 0.14 mg/kg given over 60 minutes and subsequent continuous infusion of an additional 1.7 mg/kg given over the remaining 72 hours at a rate of 0.0004 mg/kg/min.

    Group "NT3"

  • Drug: ARC1779

    Initial stepwise infusion of 0.14 mg/kg given over 60 minutes and subsequent continuous infusion of an additional 1.3 mg/kg given over the next 72 hours.

    Group "T3"

  • Drug: ARC1779

    Initial stepwise infusion of 0.28 mg/kg given over 60 minutes and subsequent continuous infusion of an additional 3.8 mg/kg given over the remaining 72 hours at a rate of 0.0009 mg/kg/min.

    Group "NT6"

  • Drug: ARC1779

    Initial stepwise infusion of 0.28 mg/kg given over 60 minutes and subsequent continuous infusion of an additional 3.0 mg/kg given over the remaining 72 hours.

    Group "T6"

  • Experimental: Group NT3
    Patients will receive a total dose of approximately 1.7 mg/kg of ARC1779 over 72 hours to achieve a target plasma concentration of 3 mcg/mL
    Intervention: Drug: ARC1779
  • Experimental: Group T3
    Patients will receive a total dose of approximately 1.4 mg/kg of ARC1779 over 72 hours to achieve a target plasma concentration of 3 mcg/mL. The infusion of ARC1779 will be tapered by 50% from 48 hours to 60 hours and again by 50% from 60 hours to 72 hours.
    Intervention: Drug: ARC1779
  • Experimental: Group NT6
    Patients will receive a total dose of approximately 3.9 mg/kg of ARC1779 over 72 hours to achieve a target plasma concentration of 6 mcg/mL.
    Intervention: Drug: ARC1779
  • Experimental: Group T6
    Patients will receive a total dose of approximately 3.1 mg/kg of ARC1779 over 72 hours to achieve a target plasma concentration of 6 mcg/mL. The infusion of ARC1779 will be tapered by 50% from 48 hours to 60 hours and again by 50% from 60 hours to 72 hours.
    Intervention: Drug: ARC1779
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Withdrawn
2
Not Provided
June 2009   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • Male or female patients;
  • ≥ 16 to ≤ 75 years of age;
  • Diagnosis of VWD-2B according to national expert guidelines for the USA [1] and Europe [2] based on medical history and findings from a matrix of laboratory assays which may include: platelet count, concentration of VWF antigen (VWF:Ag), VWF ristocetin cofactor activity (VWF:RCo), Factor VIII (FVIII) activity, ristocetin-induced platelet aggregation (RIPA), platelet function analyzer (PFA-100®) closure time, bleeding time (BT), VWF multimer test, VWF: platelet-binding (VWF:PB) activity, etc.)
  • Thrombocytopenia (defined as a platelet count < 100 per nL on at least 2 occasions within the month preceding enrollment;
  • Female patients of reproductive age must be enrolled within 1 to 7 days of the cessation of preceding menses;
  • Female patients must be non-pregnant and willing to use effective, redundant methods of contraception (i.e., for both self and male partner) throughout the study and for at least 30 days after discontinuation of study drug treatment;
  • Male patients must agree to use a medically acceptable contraceptive (abstinence or use of a condom with spermicide) throughout the study and for at least 30 days 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:

  • Patients with a possible co-existing or alternative hematologic diagnosis which can account for the laboratory findings of thrombocytopenia, etc.;
  • Any significant medical co-morbidity which would pose an increased risk of bleeding (e.g., recent trauma or surgery, a history of gastrointestinal ulcers, etc.) or thrombosis (e.g., history of recurrent deep vein thrombosis (DVT).
Both
16 Years to 75 Years
No
Contact information is only displayed when the study is recruiting subjects
Not Provided
 
NCT00694785
ARC1779-010
Not Provided
Margaret Ragni, MD, University of Pittsburgh
Archemix Corp.
Not Provided
Not Provided
Archemix Corp.
August 2009

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP