| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT00791492 | An Extension of Study Fx-005 Evaluating Long-Term Safety And Clinical Outcomes Of Fx-1006A In Patients With Transthyretin Amyloid Polyneuropathy | PHASE2 | COMPLETED | 86 | — | — | Jul 1, 2008 | Oct 1, 2010 | Dec 17, 2012 | 11 | Argentina, Brazil +4 |
| NCT00409175 | Safety and Efficacy Study of Fx-1006A in Patients With Familial Amyloidosis | PHASE2 | COMPLETED | 128 | — | — | Jan 1, 2007 | May 1, 2009 | Dec 17, 2012 | 10 | United States, Argentina +7 |
Response to treatment indicated by either improvement(decrease from baseline) or stabilization(change from baseline of 0 to less than\[\<\] 2) in NIS-LL score,based on mean of 2 scores in 1 week period.NIS-LL assessed muscle weakness,reflexes,sensation.Each item scored separately for left,right limbs.Components of muscle weakness:0(normal)-4(paralysis),higher score=more weakness;reflexes,sensation:0=normal,1=decreased,or 2=absent.Total NIS-LL score range 0-88,higher score=more impairment. For tafamidis-tafamidis group, NIS-LL baseline value of previous study FX-005(NCT00409175) used as reference.
Response to treatment indicated by either improvement(decrease from baseline) or stabilization(change from baseline of 0 to less than\[\<\] 2) in NIS-LL score,based on mean of 2 scores in 1 week period.NIS-LL assessed muscle weakness,reflexes,sensation.Each item scored separately for left,right limbs.Components of muscle weakness:0(normal)-4(paralysis),higher score=more weakness;reflexes,sensation:0=normal,1=decreased,or 2=absent.Total NIS-LL score range 0-88,higher score=more impairment. For tafamidis-tafamidis group, NIS-LL baseline value of previous study FX-005(NCT00409175) used as reference.
Norfolk QOL-DN: 35-item participant-rated questionnaire used to assess impact of diabetic neuropathy on the quality of life of participants with diabetic neuropathy; Item 1 to 7: related to symptoms and presence of symptoms was assessed as 1 and absence was assessed as 0. Item 8-35: related to activities of daily living and scored on a 5-point Likert scale, where 0= no problem and 4= severe problem (except item 32, where -2= much better, 0=about the same, 2=much worse). TQOL= sum of all the items, total possible score range= -2 to 138, where higher score=worse quality of life.
Norfolk QOL-DN: 35-item participant-rated questionnaire used to assess impact of diabetic neuropathy on the quality of life of participants with diabetic neuropathy; Item 1 to 7: related to symptoms and presence of symptoms was assessed as 1 and absence was assessed as 0. Item 8-35: related to activities of daily living and scored on a 5-point Likert scale, where 0= no problem and 4= severe problem (except item 32, where -2= much better, 0=about the same, 2=much worse). TQOL= sum of all the items, total possible score range= -2 to 138, where higher score=worse quality of life.
Response to treatment was indicated by either improvement (decrease from baseline) or stabilization (change from baseline of 0 to less than\[\<\] 2) in NIS-LL score, based on mean of 2 scores in 1 week period. NIS-LL: assessed muscle weakness, reflexes, sensation. Each item scored separately for left, right limbs. Components of muscle weakness scored on 0(normal) to 4(paralysis) scale, higher score=greater weakness. Components of reflexes, sensation scored 0=normal, 1=decreased, or 2=absent. Total NIS-LL score range 0-88, higher score=greater impairment.
Norfolk QOL-DN: 35-item participant-rated questionnaire used to assess impact of diabetic neuropathy on the quality of life of participants with diabetic neuropathy; Item 1 to 7: related to symptoms and presence of symptom was assessed as 1 and absence was assessed as 0. Item 8-35: related to activities of daily living and scored on a 5-point Likert scale, where 0= no problem and 4= severe problem (except item 32, where -2= much better, 0=about the same, 2=much worse). TQOL= sum of all the items, total possible score range= -2 to 138, where higher score=worse quality of life.
| Arm | Type | Description |
|---|---|---|
| Fx-1006A | EXPERIMENTAL | - |
| 1. | EXPERIMENTAL | Fx-1006A |
| 2. | PLACEBO_COMPARATOR | Placebo |
| Name | Type | Description |
|---|---|---|
| Fx-1006A | DRUG | Fx-1006A 20mg soft gelatin capsule administered orally once daily (at the same time each day) for 12 months. |
| Placebo | DRUG | Fx-1006A 20mg or matched placebo once daily (at the same time each day) for a period of 18 Months |
Inclusion Criteria: Male and non-pregnant female patients meeting all of the following criteria are eligible for enrollment in this study: * Patient has completed the Month 18 visit of Study Fx-005. * If female, patient is post-menopausal, surgically sterilized, or willing to use an acceptable met...