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

• Status: Completed
• Trial end date: 2008-12-01
• Target enrollment: 0
• Participant gender: All

Summary

To evaluate the overall safety and tolerability of ARC1779, a therapeutic oligonucleotide ("aptamer") in patients with three types of von Willebrand Factor related platelet disorders.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Archemix Corp.
• Treatments: Deamino Arginine Vasopressin

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.