N/A
N=5
Retrospective Study Collecting Neurological Follow-up of Hereditary Transthyretin Amyloidosis (ATTRv) Patients Included in B3461028 and B3461045.
Hereditary Transthyretin Amyloidosis (ATTRv) · Polyneuropathy
Bottom Line
View on ClinicalTrials.gov: NCT05560555 ↗Enrolled (actual)
5
Serious AEs
0.0%
Results posted
May 2024
Primary outcome: Primary: Change in Neuropathy Impairment Score (NIS) at Month 12 for ATTRv — 1.6 Change in score*months
Study Design & Population
- Study type
- Observational
- Phase
- N/A
- Interventions
- Tafamidis (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Pfizer
- Primary completion
- Nov 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in Neuropathy Impairment Score (NIS) at Month 12 for ATTRv |
1.6 | — |
| SECONDARY Change in NIS for ATTRv |
0.8; 2.4; 3.1; 4.7 | — |
| SECONDARY Change in Neuropathy Impairment Score - Lower Limbs (NIS-LL) for ATTRv |
1.6; 3.1; 4.7; 6.3; 9.4 | — |
| SECONDARY Change in Norfolk Quality of Life- Diabetic Neuropathy (Norfolk QOL-DN) for ATTRv |
NA | — |
| SECONDARY Change in COMPASS-31 for ATTRv |
NA | — |
| SECONDARY Change in Familial Amyloid Polyneuropathy Specific Rasch-Built Overall Disability Scale (FAP-RODs) for ATTRv |
NA | — |
| SECONDARY Number of Participants According to Familiar Amyloidotic Polyneuropathy Stage (FAP) for ATTRv |
0; 1; 0; 0; 1; 1 | — |
| SECONDARY Percentage of Participants Who do Not Have Stage Progression in the PND Score for ATTRv |
100.0; 0.0 | — |
| SECONDARY Percentage of Responders to Treatment for ATTRv |
100.0; 0.0 | — |
| SECONDARY Number of Participants With R-R Interval Variability for ATTRv |
0; 1 | — |
| SECONDARY Modified Body Mass Index (mBMI) for ATTRv |
27.7; 24.3 | — |
| SECONDARY Ulnar/Sural Sensory Nerve Action Potential Amplitude (SNAP) for ATTRv |
2.9 | — |
| SECONDARY Ulnar/Peroneal Compound Muscle Action Potential Amplitude (CMAP) for ATTRv |
1.3 | — |
| SECONDARY Change in NIS for ATTRwt |
0.4; 0.8; 1.1; 1.5; 2.3 | — |
| SECONDARY Change in NIS-LL for ATTRwt |
0.2; 0.4; 0.6; 0.8; 1.2 | — |
| SECONDARY Change in Norfolk QOL-DN for ATTRwt |
— | — |
| SECONDARY Change in COMPASS-31 for ATTRwt |
— | — |
| SECONDARY Change in FAP-RODs for ATTRwt |
— | — |
| SECONDARY Number of Participants According to FAP Stage for ATTRwt |
0; 1; 0; 0; 0; 2 | — |
| SECONDARY Percentage of Participants Who do Not Have Stage Progression in the PND Score for ATTRwt |
66.7; 33.3 | — |
| SECONDARY Percentage of Responders to Treatment for ATTRwt |
100.0; 100.0 | — |
| SECONDARY Number of Participants With R-R Interval Variability for ATTRwt |
0; 1; 0; 1 | — |
| SECONDARY mBMI for ATTRwt |
29.7; 24.0; 25.7 | — |
| SECONDARY Ulnar/Sural SNAP Score for ATTRwt |
17.0; 11.9 | — |
| SECONDARY Ulnar/Peroneal CMAP Score for ATTRwt |
5.9; 2.7 | — |
| SECONDARY Number of Participants With Carpal Tunnel Syndrome |
2; 2 | — |
| SECONDARY Number of Participants With Lumbar Stenosis |
0; 1 | — |
| SECONDARY Number of Participants With Gastrointestinal Disturbances |
1; 0; 1; 0; 2 | — |
| SECONDARY Number of Participants With Unintentional Weight Loss |
0; 0; 0 | — |
| SECONDARY Number of Participants With Urological Disturbances |
0; 0; 0; 0; 0; 1 | — |
| SECONDARY Number of Participants With Ophthalmological Disturbances |
0; 0; 0; 0; 0; 0 | — |
| SECONDARY Number of Participants With Central Nervous System (CNS) Disturbances |
0; 0; 0; 0; 0; 0 | — |
| SECONDARY Number of Participants With Symptoms of Autonomic Neuropathy |
0; 1; 0; 2; 1; 2 | — |
| SECONDARY Number of Participants With Symptoms of Peripheral Neuropathy |
1; 0 | — |
Summary
A study of patients with hereditary transthyretin amyloidosis (ATTRv) and wild-type transthyretin amyloidosis (ATTRwt) that have been enrolled in B3461028 and B3461045 studies in Spain - exposed to tafamidis 61mg for ≥12 months with polyneuropathy (PN) have kept going to their multisystemic follow-ups (neuro/ophthalmo/gastrointestinal) ≥12 months.
Eligibility Criteria
Inclusion Criteria
- Treatment with tafamidis 61 mg ≥ 12 months
- Neurological follow up ≥ 12 months
- Diagnosis of transthyretin amyloidosis with polyneuropathy (ATTR-PN) based on one of the following:
- Amplitude reduction in, at least, 2 nerves under normal value, excluding median nerve OR 50% amplitude reduction in, at least, 2 nerves on the basal value of the patient, excluding median nerve OR 2 abnormal tests detecting thin fibers alterations (through Sudo scan, RR Interval analysis, etc..)
Exclusion Criteria
- Treatment with tafamidis 61 mg < 12 months
- Neurological follow up < 12 months
- Other diagnosis for polyneuropathy
Data sourced from ClinicalTrials.gov (NCT05560555). 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.