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

Sponsored by:	Archemix Corp.
Information provided by:	Archemix Corp.
ClinicalTrials.gov Identifier:	NCT00694785

Purpose

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

Condition	Intervention	Phase
Von Willebrand Disease	Drug: ARC1779	Phase II

Genetics Home Reference related topics: hemophilia von Willebrand disease

Drug Information available for: Liothyronine sodium Triiodothyronine

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
Official Title:	A Study of the Pharmacokinetics, Pharmacodynamics, and Safety of ARC1779 Injection in Patients With Von Willebrand Disease Type 2B

Further study details as provided by Archemix Corp.:

Primary Outcome Measures:

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 ]

Secondary Outcome Measures:

To assess the concentration-response relationships among ARC1779 pharmacokinetic (PK) and pharmacodynamic (PD) parameters [ Time Frame: 10 days ] [ Designated as safety issue: No ]

Estimated Enrollment:	2
Study Start Date:	October 2008
Estimated Primary Completion Date:	June 2009 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Group NT3: Experimental 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	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"
Group T3: Experimental 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.	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"
Group NT6: Experimental 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.	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"
Group T6: Experimental 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.	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"

Detailed Description:

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.

Eligibility

Ages Eligible for Study:	16 Years to 75 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

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).

Contacts and Locations

No Contacts or Locations Provided

More Information

Responsible Party:	University of Pittsburgh ( Margaret Ragni, MD )
Study ID Numbers:	ARC1779-010
Study First Received:	June 6, 2008
Last Updated:	October 1, 2008
ClinicalTrials.gov Identifier:	NCT00694785
Health Authority:	United States: Food and Drug Administration

Study placed in the following topic categories:

Von Willebrand Disease
Thrombocytopathy
Hemorrhagic Disorders
Genetic Diseases, Inborn
Hematologic Diseases

Blood Platelet Disorders
Blood Coagulation Disorders
Hemostatic Disorders
Von Willebrand disease

Additional relevant MeSH terms:

Blood Coagulation Disorders, Inherited
Coagulation Protein Disorders

ClinicalTrials.gov processed this record on January 16, 2009