Clinical Outcome Study of ARC1779 Injection in Patients With Thrombotic Microangiopathy
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ClinicalTrials.gov Identifier: NCT00726544 |
Recruitment Status :
Terminated
(Enrollment into study was slower than expected.)
First Posted : August 1, 2008
Last Update Posted : November 26, 2009
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Thrombotic Microangiopathy Thrombotic Thrombocytopenic Purpura | Drug: ARC 1779 Placebo Drug: ARC1779 Injection | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 100 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Primary Purpose: | Treatment |
Official Title: | A Randomized, Double-blind, Placebo Controlled, Clinical Outcome Study of ARC1779 Injection in Patients With Thrombotic Microangiopathy |
Study Start Date : | December 2008 |
Estimated Primary Completion Date : | December 2010 |
Estimated Study Completion Date : | March 2011 |

Arm | Intervention/treatment |
---|---|
Placebo Comparator: Placebo |
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. |
Active Comparator: Low Dose |
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. |
Active Comparator: Medium Dose |
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. |
Active Comparator: High Dose |
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. |
- 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 ]
- 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. ]
- 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. ]
- 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. ]
- 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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Ages Eligible for Study: | 18 Years to 75 Years (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00726544

Responsible Party: | Dr, James Gilbert, Archemix |
ClinicalTrials.gov Identifier: | NCT00726544 |
Other Study ID Numbers: |
ARC1779-006 |
First Posted: | August 1, 2008 Key Record Dates |
Last Update Posted: | November 26, 2009 |
Last Verified: | November 2009 |
thrombocytopenia microangiopathic hemolytic anemia von Willebrand Factor ADAMTS13 |
Vascular Diseases Purpura Purpura, Thrombocytopenic Purpura, Thrombotic Thrombocytopenic Thrombotic Microangiopathies Blood Coagulation Disorders Hematologic Diseases Hemorrhage |
Pathologic Processes Skin Manifestations Thrombocytopenia Blood Platelet Disorders Immune System Diseases Thrombophilia Cardiovascular Diseases |