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: 9.1 Level: LLT Classification code 10037563 Term: Purpura thrombopenic thrombotic MedDRA version: 9.1 Level: LLT Classification code 10047715 Term: Von Willebrand's disease
Conditions
Interventions
Product Name: ARC1779 Injection
Product Code: ARC1779
Pharmaceutical Form: Solution for injection
Current Sponsor code: ARC1779
Other descriptive name: ARC 1779
Concentration unit: mg/ml milligram(s)/
Sponsors
Archemix Corp.
Eligibility
Sex/Gender
All
Inclusion criteria
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
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: • To establish the overall safety and tolerability of ARC1779 Injection in patients with vWF-related platelet function disorders, including patients with TTP in remission, patients experiencing a current episode of acute TTP, patients with familial TTP characterized by chronic/recurrent thrombocytopenia, and patients with vWD-2b; • To characterize the PD profile of ARC1779 Injection in patients with vWF-related platelet function disorders with respect to parameters of platelet function and vWF activity; • To characterize the PK and PD profiles of ARC1779 following SC injection; • To assess the concentration- and dose-response relationships among ARC1779 PD and PK parameters.;Secondary Objective: ;Primary end point(s): Safety Variables For all patients, the safety of ARC1779 administration will be assessed through evaluation of the following: - Adverse events - Clinical laboratory tests (serum chemistries, urinalysis) - Coagulation tests (PT, aPTT) - Hematological profile (blood group typing, complete blood cell count with WBC differential and platelet count) - Vital signs - ECG recordings for PR, QRS, and QT intervals and evidence of arrhythmias Pharmcokinetic variables for all patients receiving ARC1779: Cmax; Tmax; t½ß; t½a, AUCp, AUC0-t, AUC0-last and AUC0-8; ?z; CL, Vss and Vz. Plasma samples will be analyzed for quantification of ARC1779 concentrations using a validated high performance liquid chromatography (HPLC) method. Pharmacodynamics: The PD variables to be measured are: platelet counts, vWF activity (by ELISA directed toward the A1 domain), vWF antigen, vWF multimer gel electrophoresis, shear-dependent platelet function (PFA-100®), shear-dependent platelet adhesion (cone and plate analyzer), and multiplate platelet function analysis. | — |
Countries
Austria
Outcome results
None listed