The Effect Of Tafamidis For The Transthyretin Amyloid Polyneuropathy Patients With V30M Or Non-V30M Transthyretin

The Effect Of Transthyretin Stabilization, Safety, Tolerability, Efficacy And Pharmacokinetics Of Orally Administered Tafamidis In Transthyretin Amyloid Polyneuropathy Patients With V30M Or Non-V30M Transthyretin: A Phase 3, Open-Label(B3461010)

Status

Unknown

Phases

Phase 3

Study type

Interventional

Source

JPRN

Registry ID

JPRN-jRCT2080221636

Enrollment

Registered

2011-11-02

Start date

2011-11-02

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Transthyretin Familial Amyloid Polyneuropathy

Interventions

investigational material(s) Generic name etc : tafamidis meglumine INN of investigational material : tafamidis meglumine Therapeutic category code : 129 Other agents affecting peripheral nervous syste

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Transthyretin amyloid polyneuropathy with V30M or non-V30M transthyretin mutation.

Exclusion criteria

Exclusion criteria: -Primary amyloidosis and secondary amyloidosis. -History of liver transplant.

Design outcomes

Primary

Measure	Time frame
TTR stabilization at Week 8 compared with Baseline, as measured by a validated immunoturbidimetric assay. Same as above	—

Secondary

Measure	Time frame
Change from baseline on the following scales:Neuropathy Impairment Score;QOL;"Sigma 7 NTs NDS"as measured by nerve conduction studies(NCS),vibration detection threshold(VDT) and heart rate response to deep breathing(HRDB); Change from baseline on the following scales:"Sigma 3 NTSF NDS"as measured by cooling and heat pain thresholds utilizing CASE IV and heart rate response to deep breathing (HRDB);mBMI;Ambulatory status. Transthyretin(TTR)stabilization at each follow up visit after Week 8. Blood samples for determination of plasma levels of tafamidis will be collected at Baseline and during the visits at Week 2. lood samples for determination of plasma levels of tafamidis will be collected at Week 4. Blood samples for determination of plasma levels of tafamidis will be collected Week 8. Blood samples for determination of plasma levels of tafamidis will be collected at Week 26. Blood samples for determination of plasma levels of tafamidis will be collected at Week 52. Blood samples for determination of plasma levels of tafamidis will be collected at Week 78. Blood samples for determination of plasma levels of tafamidis will be collected at End of study. Same as above	—

Measure

Time frame

Change from baseline on the following scales:Neuropathy Impairment Score;QOL;"Sigma 7 NTs NDS"as measured by nerve conduction studies(NCS),vibration detection threshold(VDT) and heart rate response to deep breathing(HRDB); Change from baseline on the following scales:"Sigma 3 NTSF NDS"as measured by cooling and heat pain thresholds utilizing CASE IV and heart rate response to deep breathing (HRDB);mBMI;Ambulatory status. Transthyretin(TTR)stabilization at each follow up visit after Week 8. Blood samples for determination of plasma levels of tafamidis will be collected at Baseline and during the visits at Week 2. lood samples for determination of plasma levels of tafamidis will be collected at Week 4. Blood samples for determination of plasma levels of tafamidis will be collected Week 8. Blood samples for determination of plasma levels of tafamidis will be collected at Week 26. Blood samples for determination of plasma levels of tafamidis will be collected at Week 52. Blood samples for determination of plasma levels of tafamidis will be collected at Week 78. Blood samples for determination of plasma levels of tafamidis will be collected at End of study. Same as above

—

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026