Home
Search
Study Topics
Glossary
|
|
|
|
|
|
Sponsored by: |
Archemix Corp. |
---|---|
Information provided by: | Archemix Corp. |
ClinicalTrials.gov Identifier: | NCT00632242 |
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.
Condition | Intervention | Phase |
---|---|---|
Purpura, Thrombotic Thrombocytopenic Von Willebrand Disease Type-2b |
Drug: ARC1779 |
Phase II |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Single Group Assignment, Safety/Efficacy Study |
Official 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 |
Estimated Enrollment: | 28 |
Study Start Date: | January 2008 |
Study Completion Date: | December 2008 |
Primary Completion Date: | December 2008 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
---|---|
TTP Remission Cohort 1: Experimental
Patients will receive a total dose of 0.47 mg/kg of ARC1779 over 4 hours to achieve a target plasma concentration of 6 mcg/mL
|
Drug: ARC1779
Initial stepwise infusion of 0.23 mg/kg given over 30 minutes and subsequent continuous infusion of an additional 0.24 mg/kg given over 4 hours at a rate of 0.001 mg/kg/min.
|
TTP Remission Cohort 2: Experimental
Patients will receive a total dose of 1.67 mg/kg of ARC1779 over 24 hours to achieve a target plasma concentration of 6 mcg/mL
|
Drug: ARC1779
Initial stepwise infusion of 0.23 mg/kg given over 30 minutes and subsequent continuous infusion of an additional 1.44 mg/kg given over 24 hours at a rate of 0.001 mg/kg/min.
|
TTP Remission Cohort 3: Experimental
Patients will receive a total dose of 3.34 mg/kg of ARC1779 over 24 hours to achieve a target plasma concentration of 12 mcg/mL
|
Drug: ARC1779
Initial stepwise infusion of 0.46 mg/kg over 30 minutes and subsequent continuous infusion of an additional 2.88 mg/kg given over 24 hours at a rate of 0.002 mg/kg/min.
|
Acute TTP Cohort 4: Experimental
Patients will receive up to a total dose of 40.78 mg/kg of ARC1779 for ≤ 14 days to achieve a target plasma concentration of 12 mcg/mL
|
Drug: ARC1779
Initial stepwise infusion of 0.23 mg/kg given over 30 minutes and subsequent continuous infusion of an additional 1.44 mg/kg given over 24 hours at a rate of 0.001 mg/kg/min to produce a target plasma concentration of 6 mcg/mL. Continuous infusion of ARC1779 Injection may continue for ≤ 14 days. After initial 24 hours dose may be titrated to achieve a target plasma concentration of 12 mcg/mL as needed, on the basis of clinical and laboratory data, according to the Investigator's judgment. |
vWD-Type2b Cohort 5: Experimental
Subjects will receive either ARC1779, desmopressin or a combination of ARC1779 and desmopressin in a 3-period crossover design. The maximum dose of ARC1779 will be 0.47 mg/kg to achieve a target plasma concentration of 6 mcg/mL.
|
Drug: ARC1779
In one period of the sequence, ARC1779 will be administered to all subjects as a stepwise infusion of 0.23 mg/kg over 30 minutes and subsequent continuous infusion of an additional 0.24 mg/kg given over 4 hours at a rate of 0.001 mg/kg/min in combination with a dummy 30-minute infusion of placebo. In another period, subjects will receive a single infusion of desmopressin at a dose of 0.4 mcg/kg given over 30 minutes in combination with a dummy 30-minute stepwise infusion followed by 4-hour continuous infusion of placebo. In the one other period, subjects will receive the combination of desmopressin at a dose of 0.4 mcg/kg given over 30 minutes and ARC1779 given as a stepwise infusion of 0.23 mg/kg over 30 minutes and subsequent continuous infusion of an additional 0.24 mg/kg given over 4 hours at a rate of 0.001 mg/kg/min
|
ARC1779 Injection will be investigated in 4 cohorts of TTP patients as an uncontrolled, open-label study. Patients with vWD-2b will be enrolled in an additional cohort in a randomized, blinded, double-dummy, and placebo-controlled study.
Collectively, patients representing 3 different vWF-related platelet function disorders: TTP in remission, acute TTP, and vWD-2b will be treated in a total of 5 cohorts. Three cohorts will consist of patients who are status post an episode of TTP ("TTP Remission Cohorts") and will be treated with ARC1779 Injection in a dose- and duration-escalation design. In parallel, a single cohort of patients with acute TTP ("Acute TTP Cohort") will be treated according to an individual patient titration-to-response paradigm. This cohort will be opened for enrollment at the beginning of the study and closed after all of the other cohorts are completed. Also in parallel, a single cohort of patients with vWD-2b ("vWD-2b Cohort") will begin enrollment at the commencement of the study and continue independently of the course of the TTP Remission Cohorts. Up to 4 patients will be included in each of the TTP cohorts. The vWD-2b Cohort is expected to consist of up to 12 vWD-2b patients.
Ages Eligible for Study: | 18 Years to 75 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Acute TTP - any episode, first or relapse, with presence of all of the following:
Exclusion Criteria:
Responsible Party: | Archemix Corp. ( James Gilbert, MD, Chief Medical Officer ) |
Study ID Numbers: | ARC1779-004 |
Study First Received: | March 3, 2008 |
Last Updated: | January 8, 2009 |
ClinicalTrials.gov Identifier: | NCT00632242 |
Health Authority: | Austria: Agency for Health and Food Safety |
Von Willebrand Disease Purpura Hematologic Diseases Thrombophilia Blood Coagulation Disorders Blood Platelet Disorders Vascular Diseases Hemostatic Disorders Purpura, Thrombotic Thrombocytopenic Purpura, Thrombocytopenic Thrombosis |
Thrombotic thrombocytopenic purpura, acquired Thrombocytopathy Signs and Symptoms Embolism and Thrombosis Thrombocytopenia Hemorrhagic Disorders Genetic Diseases, Inborn Embolism Deamino Arginine Vasopressin Von Willebrand disease |
Skin Manifestations Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Cardiovascular Diseases |