Ignite Creation Date:
2024-05-05 @ 5:12 PM
Last Modification Date:
2024-10-26 @ 9:29 AM
Study NCT ID:
NCT00409175
Status:
COMPLETED
Last Update Posted:
2012-12-17
First Post:
2006-12-06
Brief Title:
Safety and Efficacy Study of Fx-1006A in Patients With Familial Amyloidosis
Sponsor:
Pfizer
Organization:
Pfizer
Study Overview
Official Title:
Safety and Efficacy of Orally Administered Fx-1006A in Patients With Familial Amyloid Polyneuropathy FAP A Randomized Double-blind Placebo-controlled Study
Status:
COMPLETED
Status Verified Date:
2012-11
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This study will examine whether Fx-1006A is effective in halting the progression of Familial Amyloid Polyneuropathy FAP
Deposition of TTR amyloid is associated with a variety of human diseases Deposition of amyloid fibrils of variant TTR primarily V30M in peripheral nerve tissue produces the condition called FAP
The prevention of the formation of amyloid by stabilization of the TTR native state should constitute an effective therapy for amyloid diseases Therapeutic intervention with a TTR stabilizer drug such as Fx-1006A is hypothesized to stop progression of the disease in FAP patients FAP is a uniformly fatal disease and Fx-1006A is intended to halt the relentless neurological deterioration FAP patients experience
This Phase 23 study will enroll early to mid-stage FAP patients in order to interrupt and stabilize the disease at a point in time where progression of motor and autonomic dysfunction can be maximally effected Male and female patients with FAP with documented V30M TTR mutation will receive Fx-1006A or placebo once daily for a period of eighteen 18 months
Deposition of TTR amyloid is associated with a variety of human diseases Deposition of amyloid fibrils of variant TTR primarily V30M in peripheral nerve tissue produces the condition called FAP
The prevention of the formation of amyloid by stabilization of the TTR native state should constitute an effective therapy for amyloid diseases Therapeutic intervention with a TTR stabilizer drug such as Fx-1006A is hypothesized to stop progression of the disease in FAP patients FAP is a uniformly fatal disease and Fx-1006A is intended to halt the relentless neurological deterioration FAP patients experience
This Phase 23 study will enroll early to mid-stage FAP patients in order to interrupt and stabilize the disease at a point in time where progression of motor and autonomic dysfunction can be maximally effected Male and female patients with FAP with documented V30M TTR mutation will receive Fx-1006A or placebo once daily for a period of eighteen 18 months
Detailed Description:
Deposition of TTR amyloid is associated with a variety of human diseases Deposition of amyloid fibrils of variant TTR primarily V30M in peripheral nerve tissue produces the condition called FAP
The prevention of the formation of amyloid by stabilization of the TTR native state should constitute an effective therapy for amyloid diseases Therapeutic intervention with a TTR stabilizer drug such as Fx-1006A is hypothesized to stop progression of the disease in FAP patients FAP is a uniformly fatal disease and Fx-1006A is intended to halt the relentless neurological deterioration FAP patients experience
This Phase 23 study will enroll early to mid-stage FAP patients in order to interrupt and stabilize the disease at a point in time where progression of motor and autonomic dysfunction can be maximally effected Male and female patients with FAP with documented V30M TTR mutation will receive Fx-1006A or placebo once daily for a period of eighteen 18 months
The prevention of the formation of amyloid by stabilization of the TTR native state should constitute an effective therapy for amyloid diseases Therapeutic intervention with a TTR stabilizer drug such as Fx-1006A is hypothesized to stop progression of the disease in FAP patients FAP is a uniformly fatal disease and Fx-1006A is intended to halt the relentless neurological deterioration FAP patients experience
This Phase 23 study will enroll early to mid-stage FAP patients in order to interrupt and stabilize the disease at a point in time where progression of motor and autonomic dysfunction can be maximally effected Male and female patients with FAP with documented V30M TTR mutation will receive Fx-1006A or placebo once daily for a period of eighteen 18 months
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| B3461020 | None | None | None |