Clinical Outcome Study of ARC1779 Injection in Patients With Thrombotic Microangiopathy

NCT ID: NCT00726544

Last Updated: 2009-11-26

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

100 participants

Study Classification

INTERVENTIONAL

Study Start Date

2008-12-31

Study Completion Date

2011-03-31

Brief Summary

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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.

Detailed Description

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Conditions

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Thrombotic Microangiopathy Thrombotic Thrombocytopenic Purpura

Keywords

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thrombocytopenia microangiopathic hemolytic anemia von Willebrand Factor ADAMTS13

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Placebo

Group Type PLACEBO_COMPARATOR

ARC 1779 Placebo

Intervention Type DRUG

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

Group Type ACTIVE_COMPARATOR

ARC1779 Injection

Intervention Type DRUG

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

Group Type ACTIVE_COMPARATOR

ARC1779 Injection

Intervention Type DRUG

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

Group Type ACTIVE_COMPARATOR

ARC1779 Injection

Intervention Type DRUG

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.

Interventions

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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.

Intervention Type 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.

Intervention Type 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.

Intervention Type 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.

Intervention Type DRUG

Eligibility Criteria

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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 antiglobulin 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.

Patients who have again become acutely ill following recent treatment and achievement of a brief remission of acute TMA may be enrolled in the study if ALL of the following conditions are met:

* Disease activity in the patient in unabated (e.g. persistent thrombocytopenia and microangiopathic hemolytic anemia with ongoing neurological symptoms and/or troponin elevation);
* The last plasma exchange of the patient's preceding course of treatment occurred at least 7 days prior;
* The patient did not undergo splenectomy during the preceding course of treatment;
* The new course of plasma exchange has not been ongoing for more than 3 days.

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).

Patients who have again become acutely ill following recent treatment and achievement of a brief remission of acute TMA may not be enrolled in the study if ANY of the following conditions are met:

* The last plasma exchange of the patient's preceding course of treatment occurred less than 7 days prior;
* The patient underwent splenectomy during the preceding course of treatment;
* The new course of plasma exchange has been ongoing for more than 3 days.
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Archemix Corp.

INDUSTRY

Sponsor Role lead

Responsible Party

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Archemix

Locations

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Northwestern University

Chicago, Illinois, United States

Site Status

Indiana University Simon Cancer Center

Indianapolis, Indiana, United States

Site Status

Washington University

St Louis, Missouri, United States

Site Status

New York Medical College

Valhalla, New York, United States

Site Status

The Ohio State University Research Foundation

Columbus, Ohio, United States

Site Status

University of Pennsylvania

Philadelphia, Pennsylvania, United States

Site Status

The Methodist Hospital

Houston, Texas, United States

Site Status

University of Vienna

Vienna, , Austria

Site Status

QEII CDHA Centre

Halifax, Nova Scotia, Canada

Site Status

CICM/Hospital Charles LeMoyne

Greenfield Park, Quebec, Canada

Site Status

CHA-Hospital de L'Enfant-Jesus

Québec, Quebec, Canada

Site Status

Ospedale Ferrarotto

Catania, , Italy

Site Status

Policlinico Mangiagalli Regina Elena-Fondazione L.Villa

Milan, , Italy

Site Status

Fondazione Ospedale Maggiore Policlinico

Milan, , Italy

Site Status

Azienda Ospedaliera S.Maria Nuova

Reggio Emilia, , Italy

Site Status

Università Cattolica del Sacro Cuore

Rome, , Italy

Site Status

University College London Hospital

London, , United Kingdom

Site Status

Countries

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United States Austria Canada Italy United Kingdom

Other Identifiers

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ARC1779-006

Identifier Type: -

Identifier Source: org_study_id