Safety And Efficacy Evaluation Of Fx-1006A In Subjects With Transthyretin Amyloidosis
This is a Phase 3, open-label study designed to obtain additional, long-term, open-label safety and efficacy data for Fx-1006A and to continue to provide patients with 20 mg oral Fx-1006A (soft gel capsule) who have completed either Protocol Fx-006 (a 1 year, open-label extension study to Protocol Fx-005 which is a randomized, double-blind, placebo-controlled, 18-month study to evaluate the safety and efficacy of Fx-1006A) or Protocol Fx1A-201 (a Phase 2, open-label study to evaluate TTR stabilization as well as the safety and tolerability of Fx-1006A) until market availability of Fx-1006A in individual patients' country of residence.
Patients who successfully complete Protocol Fx-006 or Fx1A-201 will report to the clinical unit on Day 0 (Baseline) to sign the informed consent form and determine their eligibility for Protocol Fx1A-303. In addition, on Day 0 (Baseline), patients will have their entrance criteria reviewed and medical history and demography for all patients will be obtained. The relevant end of study assessments from Protocols Fx1A-201 and Fx-006 will serve as Baseline assessments for Protocol Fx1A-303 if these examinations were performed within 30 days of Day 0 (Baseline). For any patient successfully completing Protocol Fx-006, the Karnofsky Performance Scale Index will be assessed and the cranial nerve and upper limb components of the NIS will be performed and combined with the NIS-LL data from the end of study visit from Protocol Fx-006.
If there is more than 30 days between the final study visit of Fx-006 or Fx1A-201 and Day 0 (Baseline) of Fx1A-303, all Day 0 study procedures will be performed (i.e., no data from the final study visits from the previous studies will be utilized).
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 and, if female, a urine pregnancy test will be performed. An abbreviated physical examination (including weight and vitals signs) will be conducted at every other 6 month visit. A telephone call will be made at 3-month intervals between clinic visits to assess safety and the use of concomitant medications.
For the evaluation of efficacy, the NIS, Norfolk QOL-DN, and Karnofsky Performance Scale Index will be performed on an annual basis (i.e., every other 6-month visit).
An end of study visit will occur upon patient withdrawal (for any reason), program discontinuation by the Sponsor, or upon market availability of Fx-1006A in individual patients' country of residence.
|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 Transthyretin Amyloidosis (fx1a-303)|
- To obtain additional, long-term, open-label safety and efficacy data for Fx-1006A in patients with transthyretin (TTR) amyloidosis (ATTR) [ Time Frame: 10 years ] [ Designated as safety issue: Yes ]
- To continue to provide the investigational product Fx-1006A until its market availability to patients with ATTR who have completed Protocol Fx-006 or Protocol Fx1A-201 [ Time Frame: 10 years ] [ Designated as safety issue: Yes ]
|Study Start Date:||August 2009|
|Estimated Study Completion Date:||December 2021|
|Estimated Primary Completion Date:||December 2021 (Final data collection date for primary outcome measure)|
Please refer to this study by its ClinicalTrials.gov identifier: NCT00925002
|Contact: Pfizer CT.gov Call Center||1-800-718-1021|
|United States, Maryland|
|Johns Hopkins Hospital||Active, not recruiting|
|Baltimore, Maryland, United States, 21287|
|FLENI (Fundacion Lucha contra las Enfermedades Neurologicas Infantiles)||Active, not recruiting|
|Buenos Aires, Argentina, C1428AQK|
|FLENI Florencia Picone||Active, not recruiting|
|Ciudad de Buenos Aires, Argentina, C1428AQK|
|Hospital Universitario Clementino Fraga Filho||Recruiting|
|Rio de Janeiro, RJ, Brazil, 21941-913|
|CHU Henri Mondor||Active, not recruiting|
|Creteil cedex, France, 94010|
|Universitatsklinikum Muenster||Active, not recruiting|
|Muenster, Germany, 48149|
|University Hospital Medizinische Klinik und Polikinik B||Active, not recruiting|
|Munster, Germany, 48129|
|Centro per lo Studio e la Cura delle Amiloidosi Sistemiche IRCCS Policlinico San Matteo||Active, not recruiting|
|Pavia, Italy, 27100|
|IRCCS - Policlinico San Matteo||Active, not recruiting|
|Pavia, Italy, 27100|
|Hospital de Santa Maria||Active, not recruiting|
|Lisbon, Portugal, 1649-028|
|Serviço de Neurologia, piso 7, Hospital de Santa Maria||Active, not recruiting|
|Lisbon, Portugal, 1649-035|
|Unidade Clinica de Paramiloidose, EPE - Centro Hospitalar do Porto, Hospital Geral de Santo Antonio||Active, not recruiting|
|Porto, Portugal, 4099-001|
|Umea University Hospital||Active, not recruiting|
|Umea, SE, Sweden, 901 87|
|Neuroscience Center||Active, not recruiting|
|Umea, Sweden, 90185|
|Norrland's University Hospital||Active, not recruiting|
|Umea, Sweden, SE-901 85|
|Study Director:||Pfizer CT.gov Call Center||Pfizer|