A Randomized, Double-blind, Placebo-controlled, Clinical Outcome Study of ARC1779 Injection in Patients with Thrombotic Microangiopathy. - Study of ARC1779 Injection in Patients with Thrombotic Microangiopathy

• Conditions: Thrombotic microangiopathy (TMA) disorders; MedDRA version: 9.1Level: LLTClassification code 10009731Term: Coagulation disorder

• Registration Number: EUCTR2008-001872-54-GB
• Lead Sponsor: Archemix Corp

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2008-10-15
• Last Update Posted: 19 March 2012

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 100

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.
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 is 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.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

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 & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: • To evaluate the ability of ARC1779 Injection to improve clinical outcome by<br>protecting brain, heart, and kidney from damage due to formation of disseminated<br>platelet thrombi in the microcirculation;<br>• To evaluate the overall safety and tolerability of ARC1779 Injection;<br>• To assess the concentration-response of ARC1779 for efficacy- and safety-related<br>effects;<br>• To assess the concentration-response relationships among ARC1779<br>pharmacokinetic (PK) and pharmacodynamic (PD) parameters.;Secondary Objective: Exploratory objectives<br>Some patients may undergo cranial MRI once prior to, and once again ~6 weeks after the start of study drug treatment.;Primary end point(s): Composite of clinical events and biomarker evidence for injury to the target organs commonly affected by TMA, i.e. brain, heart and kidney.