Phase 4
N=15
The Effect Of Tafamidis Meglumine In Transthyretin Amyloid Polyneuropathy Patients
Transthyretin Amyloid Polyneuropathy (ATTR-PN)
Bottom Line
View on ClinicalTrials.gov: NCT04828993 ↗Enrolled (actual)
15
Serious AEs
20.0%
Results posted
Mar 2024
Primary outcome: Primary: Change From Baseline in Neuropathy Impairment Score-lower Limb (NIS-LL) Total Score at Week 72 — 2.3 Units on a scale
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 4
- Interventions
- tafamidis meglumine (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Pfizer
- Primary completion
- Feb 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in Neuropathy Impairment Score-lower Limb (NIS-LL) Total Score at Week 72 |
2.3 | — |
| SECONDARY Change From Baseline in Neuropathy Impairment Score-lower Limb (NIS-LL) Total Score at Weeks 24, and 48 |
3.0; 8.0 | — |
| SECONDARY Change From Baseline in Total Quality of Life (TQOL) of Norfolk Quality of Life - Diabetic Neuropathy (Norfolk QOL-DN) at Weeks 24, 48, and 72 |
-1.00; 2.00; 8.00 | — |
| SECONDARY Change From Baseline in 5 Domains of Norfolk QOL-DN at Weeks 24, 48, and 72 |
-3.00; 2.00; 4.00; 0.00; 1.00; 3.00 | — |
| SECONDARY Change From Baseline in Modified Body Mass Index (mBMI) at Weeks 4, 8, 12, 24, 36, 48, and 72 |
-5.9; 22.3; 9.2; 21.8; 37.0; 41.4 | — |
| SECONDARY Change From Baseline in Physical Component Summary and Mental Component Summary of 36-Item Short Form Survey (SF-36) at Weeks 24, 48, and 72 |
-2.15; -0.12; -1.60; 1.21; -2.56; -6.10 | — |
| SECONDARY Change From Baseline in EuroQoL 5 Dimensions 5 Levels (EQ-5D-5L) Index Score at Weeks 24, 48, and 72 |
0.00; -0.05; -0.16 | — |
| SECONDARY Number of Participants Reporting Treatment-Emergent Adverse Events (TEAEs) |
13 | — |
| SECONDARY Number of Participants With Categorical Vital Signs Data |
0; 0; 4; 3; 0; 6 | — |
| SECONDARY Number of Participants With Categorical Electrocardiogram (ECG) Data |
0; 0; 0; 0; 1; 1 | — |
| SECONDARY Number of Participants With Clinically Significant Echocardiography (ECHO) Value Related to Primary Diagnosis (Transthyretin Amyloidosis [ATTR]) at Baseline, Weeks 24, 48, and 72 |
9; 11; 9; 10 | — |
| SECONDARY Number of Participants With Clinical Laboratory Abnormalities |
12 | — |
| SECONDARY Transthyretin (TTR) Concentrations on Day 1 (Baseline), and at Weeks 8, 12, 24, 48, and 72 |
18.70; 23.20; 28.00; 25.70; 27.90; 26.60 | — |
| SECONDARY TTR Stabilization and Percentage and 95% CI of Responders in TTR Stabilization at Post Baseline Visit |
100.0; 100.0; 100.0; 100.0; 87.5 | — |
Summary
This is a single-arm, open-label, multicenter study designed to evaluate the efficacy, safety, tolerability as well as pharmacodynamics of tafamidis meglumine in ATTR-PN participants in China.
Approximately 10-15 participants are planned to be enrolled. All enrolled participants will receive oral tafamidis meglumine 20 mg soft capsules once daily for 72 weeks (18 months).
Eligibility Criteria
Inclusion Criteria
- Male or female participants between the ages of 18 and 80 years.
- Participants have amyloid documented by biopsy
- Participants must have a TTR mutation that is associated with ATTR-PN.
- Participants have peripheral and/or autonomic neuropathy
- Stages of disease according to symptom severity-stage I.
Exclusion Criteria
- Other acute or chronic medical or psychiatric condition including recent or active suicidal ideation or behavior or laboratory abnormality, in the judgment of the investigator, would make the participant inappropriate for entry into this study.
- Chronic use of non-protocol approved non-steroidal anti-inflammatory drugs.
- Use of diflunisal, tauroursodeoxycholate, doxycycline, inotersen, patisiran or any other TTR stabilizing agent, or experimental interventions for familial amyloidosis within 30 days prior to the study entry and/or during study participation. Participants who are taking or who have previously taken tafamidis.
Prior/Concurrent Clinical Study Experience:
- Previous administration with an investigational drug within 30 days or 5 half lives preceding the first dose of investigational product used in this study (whichever is longer).
- Participant has primary (light chain) or secondary amyloidosis.
- If female, participant is pregnant or breast feeding, or plans to be pregnant or breast feeding in the next 18 months.
- Participant has received prior liver or any other organ except cornea transplantation.
- Participant requires significant assistance with ambulation or is wheel chair bound.
- Participants with cardiomyopathy specific TTR mutations.
- Participant has other causes of sensorimotor neuropathy.
Data sourced from ClinicalTrials.gov (NCT04828993). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.