Clinical Outcome Study of ARC1779 Injection in Patients With Thrombotic Microangiopathy - Full Text View

Sponsored by:	Archemix Corp.
Information provided by:	Archemix Corp.
ClinicalTrials.gov Identifier:	NCT00726544

Purpose

The purpose of this ascending-dose research study is to determine whether the administration of ARC1779 Injection improves subject's health profile by protecting the brain, heart, and kidney from damage due to formation of small blood clots in blood vessels. It will also determine the safety of ARC1779 Injection, how ARC1779 Injection enters and leaves the blood and tissue over time, and its effect on laboratory tests related to blood clotting, heart and brain function, and other body systems.

Condition	Intervention	Phase
Thrombotic Microangiopathy Thrombotic Thrombocytopenic Purpura	Drug: ARC 1779 Placebo Drug: ARC1779 Injection	Phase II

Genetics Home Reference related topics: factor V Leiden thrombophilia hemophilia thrombotic thrombocytopenic purpura

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Randomized, Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Placebo Control, Parallel Assignment, Safety/Efficacy Study
Official Title:	A Randomized, Double-Blind, Placebo Controlled, Clinical Outcome Study of ARC1779 Injection in Patients With Thrombotic Microangiopathy

Further study details as provided by Archemix Corp.:

Primary Outcome Measures:

The incidence of the clinical composite of death (all-cause mortality), stroke, coma, seizures, renal failure, or acute myocardial infarction (AMI) [ Time Frame: 6 weeks post randomization ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Neurocognitive function is to be assessed with the CogState® test system. [ Time Frame: Once during the hospitalization period and again at the 6 week clinic visit. ] [ Designated as safety issue: No ]
The incidence of death, stroke, or acute renal failure/injury requiring dialysis is to be assessed. [ Time Frame: During the extended clinical follow-up for each patient from the time of the 6 week clinic visit until the study is closed. ] [ Designated as safety issue: No ]
Safety- and efficacy-related clinical laboratory parameters and biomarkers will be analyzed in relation to ARC1779 exposure in terms of the dose administered and the observed plasma concentration. [ Time Frame: During initial hospitalization and at 6 week clinic visit. ] [ Designated as safety issue: Yes ]
The incidence of the composite of complications associated with plasma exchange therapy (i.e., catheter-related infection, thrombosis, internal hemorrhage, or pneumothorax) is to be assessed. [ Time Frame: During initial hospitalization and at the 6 week clinic visit. ] [ Designated as safety issue: Yes ]

Estimated Enrollment:	100
Study Start Date:	December 2008
Estimated Study Completion Date:	March 2011
Estimated Primary Completion Date:	December 2010 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Placebo: Placebo Comparator	Drug: ARC 1779 Placebo ARC1779 Injection or placebo is administered intravenously to patients as an initial loading dose followed by continuous infusion of up to 14 days plus 2 day taper.
Low Dose: Active Comparator	Drug: ARC1779 Injection ARC1779 Injection or placebo is administered intravenously to patients as an initial loading dose followed by continuous infusion of up to 14 days plus 2 day taper. Treatment with ARC1779 Injection is to be given at low dosage that is intended to produce target steady-state ARC1779 plasma concentrations during infusion of 3μg/mL.
Medium Dose: Active Comparator	Drug: ARC1779 Injection ARC1779 Injection or placebo is administered intravenously to patients as an initial loading dose followed by continuous infusion of up to 14 days plus 2 day taper. Treatment with ARC1779 Injection is to be given at low dosage that is intended to produce target steady-state ARC1779 plasma concentrations during infusion of 6μg/mL.
High Dose: Active Comparator	Drug: ARC1779 Injection ARC1779 Injection or placebo is administered intravenously to patients as an initial loading dose followed by continuous infusion of up to 14 days plus 2 day taper. Treatment with ARC1779 Injection is to be given at low dosage that is intended to produce target steady-state ARC1779 plasma concentrations during infusion of 12μg/mL.

Eligibility

Ages Eligible for Study:	18 Years to 75 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Male or female;
≥18 to ≤75 years of age;
Diagnosis of TMA based on presence of:
Thrombocytopenia, defined as a platelet count <100 x 109 per liter;
Microangiopathic hemolytic anemia, defined by negative findings on direct antiglobin test, and evidence of accelerated red blood cell (RBC) production and destruction); AND
Absence of a clinically apparent alternative explanation for thrombocytopenia and anemia, e.g., disseminated intravascular coagulation (DIC), eclampsia, HELLP syndrome, Evans syndrome;
Females: non-pregnant and commit to use of 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;
Males: commit to use of 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;
Not received an unlicensed investigational agent (drug, device, or blood-derived product) within 30 days prior to randomization, and may not receive such an investigational agent in the 30 days post-randomization (note: investigational use for treatment of TMA of a licensed immunomodulator, e.g., rituximab, is permitted at any time relative to randomization);
Capable of understanding and complying with the protocol, and he/she (or a legal representative) must have signed the informed consent document prior to performance of any study-related procedures.

Exclusion Criteria:

Females: pregnant or <24 hours post-partum, or breastfeeding;
History of bleeding diathesis or evidence of active abnormal bleeding within the previous 30 days;
Disseminated malignancy or other co-morbid illness limiting life expectancy to ≤3 months independent of the TMA disorder.
Diagnosis other than TMA which can account for the findings of thrombocytopenia and hemolytic anemia (e.g., DIC, HELLP syndrome, Evans syndrome);
Diagnosis of DIC verified by laboratory values for D-dimer, fibrinogen, prothrombin time (PT), and activated partial thromboplastin time (aPTT).

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00726544

Contacts

Contact: James Gilbert, MD

+1 617-621-7700

jgilbert@archemix.com

Locations

United States, Indiana
Archemix Clinical Trial Site	Recruiting
Indianapolis, Indiana, United States, 46202

Sponsors and Collaborators

Archemix Corp.

More Information

Responsible Party:	Archemix ( Dr, James Gilbert )
Study ID Numbers:	ARC1779-006
Study First Received:	July 30, 2008
Last Updated:	January 6, 2009
ClinicalTrials.gov Identifier:	NCT00726544
Health Authority:	United States: Food and Drug Administration; United Kingdom: Medicines Healthcare Products Regulatory Agency; Austria: Austrian Agency for Health and Food Safety; Switzerland: Swissmedic; Canada: Health Canada; Italy: Ministry of Health

Keywords provided by Archemix Corp.:

thrombocytopenia
microangiopathic hemolytic anemia
von Willebrand Factor
ADAMTS13

Study placed in the following topic categories:

Von Willebrand Disease
Purpura
Hematologic Diseases
Thrombophilia
Blood Platelet Disorders
Blood Coagulation Disorders
Anemia
Vascular Diseases
Anemia, Hemolytic
Hemostatic Disorders

Purpura, Thrombotic Thrombocytopenic
Purpura, Thrombocytopenic
Thrombosis
Thrombotic thrombocytopenic purpura, acquired
Thrombocytopathy
Signs and Symptoms
Embolism and Thrombosis
Thrombocytopenia
Embolism
Von Willebrand disease

Additional relevant MeSH terms:

Skin Manifestations
Immune System Diseases
Cardiovascular Diseases

ClinicalTrials.gov processed this record on January 14, 2009