Clinical Outcome Study of ARC1779 Injection in Patients With Thrombotic Microangiopathy

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00726544
• Date of registration: 30/07/2008
• Prospective Registration: Yes
• Primary sponsor: Archemix Corp.
• Public title: Clinical Outcome Study of ARC1779 Injection in Patients With Thrombotic Microangiopathy
• Scientific title: A Randomized, Double-blind, Placebo Controlled, Clinical Outcome Study of ARC1779 Injection in Patients With Thrombotic Microangiopathy
• Date of first enrolment: December 2008
• Target sample size: 100
• Recruitment status: Terminated
• URL: http://clinicaltrials.gov/show/NCT00726544
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
• Phase: Phase 2

Countries of recruitment

Austria, Canada, Italy, United Kingdom, United States

Key inclusion & exclusion 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
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.



Age minimum: 18 Years
Age maximum: 75 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Thrombotic Microangiopathy

Thrombotic Thrombocytopenic Purpura

Intervention(s)

Drug: ARC 1779 Placebo

Drug: ARC1779 Injection

Primary Outcome(s)

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]

Secondary Outcome(s)

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.]

Secondary ID(s)

ARC1779-006

Source(s) of Monetary Support

Please refer to primary and secondary sponsors