A Phase 2 Pilot Study of the Safety, Pharmacokinetics, and Pharmacodynamics of ARC1779 Injection in Patients with von Willebrand Factor-Related Platelet Function Disorders

• Conditions: von Willebrand Factor- related platelet function disorders : thrombotic thrombocytopenic purpura(TTP) in remission, acute TTP, and von Willebrand Disease Type- 2b (vWD-2b).; MedDRA version: 9.1Level: LLTClassification code 10037563Term: Purpura thrombopenic thrombotic; MedDRA version: 9.1Level: LLTClassification code 10047715Term: Von Willebrand's disease

• Registration Number: EUCTR2007-004371-19-AT
• Lead Sponsor: Archemix Corp.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2007-12-12
• Last Update Posted: 19 March 2012

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 28

Inclusion Criteria

All patients:
•Male or female;
•= 18 to = 75 years of age;
•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 and Familial 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.

TPP remission patients: History of an episode of acute TTP at any time in the past.

Acute TPP patients:
Any episode, first or relapse, with presence of all of the following:
• Microangiopathic hemolytic anemia (schistocytosis present, Coombs test negative);
• Severe thrombocytopenia;
• Clinical diagnosis of either a primary or secondary form of TTP:
- Primary TTP: e.g., familial TTP (Upshaw-Schulman syndrome), or acquired idiopathic TTP, or atypical HUS”;
- Secondary TTP: e.g., TTP occurring post-bone marrow transplant, druginduced TTP, lupus-related TTP, etc.;

vWD-2b patients: Confirmed diagnosis of vWD-2b; Urine pregnancy tests will be repeated in female vWD-2b patients on Day 1 pre-dose for each treatment period.

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

All patients:
•History of recent trauma or surgery;
•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.

Study & Design

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