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| Tracking Information | |
|---|---|
| First Received Date ICMJE | February 27, 2008 |
| Last Updated Date | February 13, 2009 |
| Start Date ICMJE | June 2008 |
| Estimated Primary Completion Date | March 2009 (final data collection date for primary outcome measure) |
| Current Primary Outcome Measures ICMJE |
TTR stabilization [ Time Frame: 6 weeks ] [ Designated as safety issue: No ] |
| Original Primary Outcome Measures ICMJE | Same as current |
| Change History | Complete list of historical versions of study NCT00630864 on ClinicalTrials.gov Archive Site |
| Current Secondary Outcome Measures ICMJE |
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| Original Secondary Outcome Measures ICMJE | Same as current |
| Descriptive Information | |
| Brief Title ICMJE | The Effects of Fx-1006A on Transthyretin Stabilization and Clinical Outcome Measures in Patients With Non-V30M Transthyretin Amyloidosis |
| Official Title ICMJE | The Effects of Fx-1006A on Transthyretin Stabilization and Clinical Outcome Measures in Patients With Non-V30M Transthyretin Amyloidosis |
| Brief Summary | This is an open-label, multicenter, international study designed to determine TTR stabilization as well as Fx-1006A safety and tolerability, and its effects on clinical outcomes in patients with non-V30M TTR amyloidosis. Strong pre-clinical and clinical evidence support a daily dose of 20 mg of Fx-1006A to be the optimum dose to achieve stabilization of tetrameric TTR in ATTR-PN patients. Since disease presentation is similar between V30M and non-V30M TTR mutations associated with ATTR-PN and Fx-1006A has been shown to stabilize wild-type and V30M TTR in vitro and ex vivo, the present study is being conducted to determine the effects of Fx-1006A on TTR stabilization in ATTR-PN patients with TTR mutations other than V30M. Safety and exploratory efficacy of Fx-1006A administered once daily for 12 months will also be evaluated in this patient population. |
| Detailed Description | This is an open-label, multicenter, international study designed to determine TTR stabilization as well as Fx-1006A safety and tolerability, and its effects on clinical outcomes in patients with non-V30M TTR amyloidosis. The study will be conducted in two parts. Part 1 will include a six-week dosing period during which all enrolled patients will receive oral Fx-1006A 20 mg soft gelatin capsules once daily for six weeks. At Week 6, blood samples will be collected from each patient to determine TTR stabilization. Patients who complete the Week 6 visit will continue receiving daily oral Fx-1006A 20 mg for up to a total of 12 months during Part 2 of this study. If it is determined that a patient is not stabilized at Week 6, the patient will be discontinued from the study. During Part 2, clinical outcomes will be measured at Months 6 and 12, based on NIS, Norfolk QOL-DN, mBMI, NCS, HRDB, SF-36, Karnofsky score, and echocardiography; NT-pro-BNP and troponin I levels will be measured at Baseline, Weeks 2 and 6, and Months 3, 6, and 12. Pharmacokinetic measurements will be made using samples collected at Baseline, Week 6, and Months 6 and 12. Safety and tolerability will be assessed throughout the study based on vital signs, physical examinations, ECG, echocardiography, 24-hour Holter monitoring, clinical laboratory tests (hematology, serum chemistry, and urinalysis), and monitoring adverse events and concomitant medication use. Day 1 will be defined as administration of the first dose of study drug. Clinic Visits will be conducted during Screening (Days -30 to -1) and at Baseline (Day 0), and Week 2, and Week 6, and Months 3, 6, and 12 (± 2 weeks of the scheduled date for post-Baseline visits). Monthly telephone contacts (+ 1 week of the scheduled date) will be made during months in which no investigative site visits are scheduled (Months 4, 5, 7, 8, 9, 10, and 11) for assessment of adverse events and concomitant medications. A final telephone contact to assess adverse events and concomitant medication usage will be made 30 days after the last dose of study drug. Patients who discontinue from the study at any time following enrollment will have a final visit performed, including all safety assessments, at the time of discontinuation. Any patient discontinuing after the Month 6 visit will also have all exploratory assessments performed. |
| Study Phase | Phase II |
| Study Type ICMJE | Interventional |
| Study Design ICMJE | Treatment, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study |
| Condition ICMJE | Transthyretin-Associated Amyloidosis With Polyneuropathy |
| Intervention ICMJE | Drug: Fx-1006A |
| Study Arms / Comparison Groups | Experimental: Fx-1006A 20mg soft gelatin capsules once daily for 12 months |
| Publications * | |
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* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline. |
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| Recruitment Information | |
| Recruitment Status ICMJE | Active, not recruiting |
| Estimated Enrollment ICMJE | 24 |
| Estimated Completion Date | January 2010 |
| Estimated Primary Completion Date | March 2009 (final data collection date for primary outcome measure) |
| Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
|
| Gender | Both |
| Ages | 18 Years to 75 Years |
| Accepts Healthy Volunteers | No |
| Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects |
| Location Countries ICMJE | United States, France, Germany, Italy |
| Administrative Information | |
| NCT ID ICMJE | NCT00630864 |
| Responsible Party | Jeff Packman, Vice President, Drug Development Operations, FoldRx Pharmaceuticals, Inc. |
| Study ID Numbers ICMJE | Fx1A-201 |
| Study Sponsor ICMJE | FoldRx Pharmaceuticals |
| Collaborators ICMJE | |
| Investigators ICMJE | |
| Information Provided By | FoldRx Pharmaceuticals |
| Verification Date | February 2009 |
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ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |
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