ARC1779 Injection in Patients With Von Willebrand Factor-Related Platelet Function Disorders

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00632242
• Date of registration: 03/03/2008
• Prospective Registration: No
• Primary sponsor: Archemix Corp.
• Public title: ARC1779 Injection in Patients With Von Willebrand Factor-Related Platelet Function Disorders
• Scientific title: A Phase 2 Pilot Study of the Safety, Pharmacokinetics, and Pharmacodynamics of ARC1779 Injection in Patients With Von Willebrand Factor-Related Platelet Function Disorders
• Date of first enrolment: January 2008
• Target sample size: 28
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00632242
• Study type: Interventional
• Study design: Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 2

Countries of recruitment

Austria

Key inclusion & exclusion criteria

Inclusion Criteria:

- Male or female;

- Age 18-75 years;

- vWD-2b - confirmed diagnosis, or;

- TTP Remission - prior episode(s) of primary acute TTP, or;

- Acute TTP - any episode, first or relapse, with presence of all of the following:

1. Microangiopathic hemolytic anemia (schistocytosis present, Coombs test
negative);

2. Severe thrombocytopenia;

3. Clinical diagnosis of either a primary or secondary form of TTP:(1) Primary TTP:
e.g., familial TTP (Upshaw-Schulman syndrome), or acquired idiopathic TTP, or
"atypical HUS"; (2) Secondary TTP: e.g., TTP occurring post-bone marrow
transplant, drug-induced TTP, lupus-related TTP, etc.;

- Negative qualitative urine drug test at screening, and no history of alcohol or drug
abuse;

- Not considering or scheduled to undergo any surgical procedure during the duration of
the study;

- Has not donated or lost more than a unit of blood within 30 days prior to screening
visit;

- Has not received an experimental drug within 30 days prior to screening;

- Female patients must be non-pregnant [for TTP Remission and vWD-2b Cohorts, a serum
pregnancy test at screening and a urine pregnancy test at Day 1 pre-dose must be
negative; for the Acute TTP Cohort, a serum pregnancy test at Day 1 pre-dose must be
negative], and willing to use effective, redundant methods of contraception (i.e.,
for both self and male partner) throughout the study and for at least 30 days after
participation. If possible, the treatment will be initiated within 5 days of the
cessation of the preceding menstrual period;

- Male patients must agree to use a medically acceptable contraceptive (abstinence or
use of a condom with spermicide) throughout the study and for at least 30 days after
participation;

- Patients must be capable of understanding and complying with the protocol and must
have signed the informed consent document prior to performance of any study-related
procedures.

Exclusion Criteria:

- History of recent surgery or trauma;

- Any major, active health problem, e.g., cancer or heart disease, which could render
the patient medically unstable during the period of participation in the study.



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

Health Condition(s) or Problem(s) studied

Von Willebrand Disease Type-2b

Purpura, Thrombotic Thrombocytopenic

Intervention(s)

Drug: ARC1779

Primary Outcome(s)

To establish the overall safety and tolerability of ARC1779 in three varieties of von Willebrand Factor (vWF)-related platelet function disorders [Time Frame: 28 days]

Secondary Outcome(s)

To characterize the pharmacokinetic (PK) profile of ARC1779 intravenous (IV) infusion in patient groups [Time Frame: 28 days]

To assess the concentration- and dose-response relationships among ARC1779 PK and PD parameters. [Time Frame: 28 days]

To characterize the pharmacodynamic (PD) profile of ARC1779 in patients with vWF-related platelet function disorders with respect to parameters of platelet function and vWF activity [Time Frame: 28 days]

Secondary ID(s)

ARC1779-004

Source(s) of Monetary Support

Please refer to primary and secondary sponsors