Safety And Efficacy Evaluation Of Fx-1006a In Patients With V122i Or Wild-Type Transthyretin (TTR) Amyloid Cardiomyopathy
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Purpose
Open-label Safety and Efficacy Evaluation of Fx-1006a in Patients with V122i Or Wild-type Transthyretin (ttr) Amyloid Cardiomyopathy.
Patients who successfully complete Fx1B-201 will report to the clinical unit on Day 0 to sign the informed consent form and determine eligibility for Protocol Fx1B-303. In addition, on Day 0, patients will have their entrance criteria reviewed, and medical histories and demographic characteristics obtained.
The physical examination (including weight and vital signs) and the relevant end of study clinical laboratory tests (alkaline phosphatase, alanine aminotransferase, aspartate aminotransferase, blood urea nitrogen, gamma glutamyl transferase, creatinine, total bilirubin, international normalized ratio, troponin I, troponin T, and amino-terminal B-type natriuretic peptide) from Protocol Fx1B-201 will be used for Protocol Fx1B-303. If more than 30 days has elapsed between the final study visit of Protocol Fx1B-201 and Day 0 of Protocol Fx1B-303, an abbreviated physical examination (including weight and vital signs) and clinical laboratory assessments must be performed on Day 0.
Eligible patients will begin once-daily dosing with 20 mg Fx-1006A at home on Day 1 (i.e., first dose) and will return to the clinical unit for study visits every 6 months.
Adverse events (AEs) and concomitant medication use will be collected at each 6-month visit to the clinical unit. Blood draws for clinical safety laboratory tests and abbreviated physical examinations (including weight and vital signs) will also be performed at each 6-month clinic visit. ECGs will be performed every 12 months on an annual basis. A telephone call will be made at 3-month intervals between clinic visits to assess safety and use of concomitant medications.
For the evaluation of efficacy, the Patient Global Assessment, NYHA classification, KCCQ, 6-minute walk test, and efficacy-related clinical laboratory tests (serum levels of troponin T, troponin I, and NT-pro-BNP) will be determined every 6 months. In addition, echocardiograms will be performed every 12 months on an annual basis.
An end of study visit including all safety and efficacy assessments will occur upon patient completion of the study, premature withdrawal (for any reason), or in the event of program discontinuation by the Sponsor.
| Condition | Intervention | Phase |
|---|---|---|
|
ATTR-CM |
Drug: Fx-1006A |
Phase 3 |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | Open-Label Safety And Efficacy Evaluation Of Fx-1006a In Patients With V122i Or Wild-Type Transthyretin (TTR) Amyloid Cardiomyopathy |
- To obtain additional, long-term, open-label safety and efficacy data for Fx-1006A in patients with transthyretin amyloid cardiomyopathy (ATTR-CM) [ Time Frame: 3 years ] [ Designated as safety issue: Yes ]
- To continue to provide the investigational project Fx-1006A to patients with ATTR-CM who completed Protocol Fx1B-201 [ Time Frame: 3 years ] [ Designated as safety issue: Yes ]
| Enrollment: | 31 |
| Study Start Date: | September 2009 |
| Estimated Study Completion Date: | December 2020 |
| Estimated Primary Completion Date: | December 2020 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Experimental: Open-Label |
Drug: Fx-1006A
Once daily 20mg oral Fx-1006A (soft gelatin capsule)
|
Eligibility| Ages Eligible for Study: | 18 Years to 75 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Patient successfully completed Protocol Fx1B-201.
- If female; patient is post menopausal. If male, female partner is post-menopausal. If female is of child bearing potential, willing to use acceptable method of birth control up to 3 months after last dose (included female partners of male participants).
- Patient is willing to comply with protocol.
Exclusion Criteria:
- Patient did not successfully complete Fx1B-201.
- Chronic use of NSAIDS.
- Patient has a clinically significant medication condition that increases risk of study participation.
- Patient has received heart or liver transplant.
Contacts and Locations| United States, Georgia | |
| Pfizer Investigational Site | |
| Atlanta, Georgia, United States, 30322 | |
| United States, Illinois | |
| Pfizer Investigational Site | |
| Chicago, Illinois, United States, 60637 | |
| United States, Maryland | |
| Pfizer Investigational Site | |
| Baltimore, Maryland, United States, 21287 | |
| United States, Massachusetts | |
| Pfizer Investigational Site | |
| Boston, Massachusetts, United States, 02215 | |
| United States, New York | |
| Pfizer Investigational Site | |
| New York, New York, United States, 10032 | |
| Study Director: | Pfizer CT.gov Call Center | Pfizer |
More Information
Additional Information:
No publications provided
| Responsible Party: | Pfizer |
| ClinicalTrials.gov Identifier: | NCT00935012 History of Changes |
| Other Study ID Numbers: | FX1B-303, B3461026 |
| Study First Received: | July 6, 2009 |
| Last Updated: | April 29, 2013 |
| Health Authority: | United States: Food and Drug Administration |
Additional relevant MeSH terms:
|
Amyloidosis Cardiomyopathies Proteostasis Deficiencies |
Metabolic Diseases Heart Diseases Cardiovascular Diseases |
ClinicalTrials.gov processed this record on May 23, 2013