- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00726544
Clinical Outcome Study of ARC1779 Injection in Patients With Thrombotic Microangiopathy
A Randomized, Double-blind, Placebo Controlled, Clinical Outcome Study of ARC1779 Injection in Patients With Thrombotic Microangiopathy
Study Overview
Status
Intervention / Treatment
Study Type
Enrollment (Anticipated)
Phase
- Phase 2
Contacts and Locations
Study Locations
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Vienna, Austria, 1090
- University of Vienna
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Quebec, Canada, G1J 1Z4
- CHA-Hospital de L'Enfant-Jesus
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Nova Scotia
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Halifax, Nova Scotia, Canada, B3H 2Y9
- QEII CDHA Centre
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Quebec
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Greenfield Park, Quebec, Canada, J4V 2H1
- CICM/Hospital Charles LeMoyne
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Catania, Italy, 95100
- Ospedale Ferrarotto
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Milan, Italy
- Policlinico Mangiagalli Regina Elena-Fondazione L.Villa
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Milano, Italy, 20122
- Fondazione Ospedale Maggiore Policlinico
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Reggio Emilia, Italy, 42100
- Azienda Ospedaliera S.Maria Nuova
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Rome, Italy
- Universita Cattolica del Sacro Cuore
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London, United Kingdom, W1T 4EU
- University College London Hospital
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Illinois
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Chicago, Illinois, United States, 60611
- Northwestern University
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Indiana
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Indianapolis, Indiana, United States, 46202
- Indiana University Simon Cancer Center
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Missouri
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St. Louis, Missouri, United States, 63110
- Washington University
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New York
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Valhalla, New York, United States, 10595
- New York Medical College
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Ohio
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Columbus, Ohio, United States, 43235
- The Ohio State University Research Foundation
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Pennsylvania
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Philadelphia, Pennsylvania, United States, 19104
- University of Pennsylvania
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Texas
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Houston, Texas, United States, 77030
- The Methodist Hospital
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
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.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Placebo Comparator: Placebo
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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.
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Active Comparator: Low Dose
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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.
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.
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.
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Active Comparator: Medium Dose
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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.
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.
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.
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Active Comparator: High Dose
|
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.
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.
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.
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
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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
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6 weeks post randomization
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Secondary Outcome Measures
Outcome Measure |
Time Frame |
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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.
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Once during the hospitalization period and again at the 6 week clinic visit.
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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.
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During the extended clinical follow-up for each patient from the time of the 6 week clinic visit until the study is closed.
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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.
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During initial hospitalization and at 6 week clinic visit.
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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.
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During initial hospitalization and at the 6 week clinic visit.
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Collaborators and Investigators
Sponsor
Study record dates
Study Major Dates
Study Start
Primary Completion (Anticipated)
Study Completion (Anticipated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Pathologic Processes
- Cardiovascular Diseases
- Immune System Diseases
- Hematologic Diseases
- Hemorrhage
- Blood Coagulation Disorders
- Skin Manifestations
- Thrombocytopenia
- Blood Platelet Disorders
- Thrombophilia
- Vascular Diseases
- Purpura
- Purpura, Thrombocytopenic
- Purpura, Thrombotic Thrombocytopenic
- Thrombotic Microangiopathies
Other Study ID Numbers
- ARC1779-006
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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