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Clinical Trials/NCT05560555
NCT05560555CompletedNot Applicable

Tafamidis 61mg, Outcomes in ATTR Amyloidosis With Neurologic and Multisystemic Involvement - TRAMA

Pfizer3 sites in 1 country5 target enrollmentStarted: October 24, 2022Last updated:
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Conditions
Interventions
Drugs

Trial Snapshot

Phase
Not Applicable
Status
Completed
Sponsor
Pfizer
Enrollment
5
Locations
3
Primary Endpoint
Change in Neuropathy Impairment Score (NIS) at Month 12 for ATTRv
Study OverviewStudy DesignEligibility CriteriaArms & InterventionsOutcomesInvestigatorsSitesRecords & IdentifiersSimilar Trials

Study Overview

Brief 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.

Study Design

Study Type
Observational
Observational Model
Other
Time Perspective
Retrospective

Eligibility Criteria

Ages
18 Years to — (Adult, Older Adult)
Sex
All
Accepts Healthy Volunteers
No

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

Arms & Interventions

Retrospective cohort ATTRv and ATTRwt patients enrolled in B3461028 and B3461045 studies in Spain

Intervention: Tafamidis (Drug)

Outcomes

Primary Outcomes

Change in Neuropathy Impairment Score (NIS) at Month 12 for ATTRv

Time Frame: Baseline and Month 12 (data collected and analyzed over 22 days)

NIS (Neuropathy Impairment Score) is a clinically important, sensitive measure of individual neurological function, assessing sensory function, reflexes, and muscle weakness. NIS score ranged from 0 to 244, with higher score indicating greater disability or impairment. The rate of change was calculated from last follow-up.

Secondary Outcomes

  • Change in Familial Amyloid Polyneuropathy Specific Rasch-Built Overall Disability Scale (FAP-RODs) for ATTRv(Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days))
  • Number of Participants According to Familiar Amyloidotic Polyneuropathy Stage (FAP) for ATTRv(Month 18, 24 and 30 months after the start of treatment (data collected and analyzed over 22 days))
  • Change in NIS for ATTRv(Baseline, Month 6, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days))
  • Change in COMPASS-31 for ATTRv(Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days))
  • Change in Neuropathy Impairment Score - Lower Limbs (NIS-LL) for ATTRv(Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days))
  • Change in Norfolk Quality of Life- Diabetic Neuropathy (Norfolk QOL-DN) for ATTRv(Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days))
  • Percentage of Participants Who do Not Have Stage Progression in the PND Score for ATTRv(From Baseline to Month 30 (data collected and analyzed over 22 days))
  • Number of Participants With Symptoms of Autonomic Neuropathy(Month 18, 24 and 30 after the start of treatment (data collected and analyzed over 22 days))
  • Number of Participants With Symptoms of Peripheral Neuropathy(At baseline (data collected and analyzed over 22 days))
  • Percentage of Responders to Treatment for ATTRv(Month 12 (data collected and analyzed over 22 days))
  • Ulnar/Sural Sensory Nerve Action Potential Amplitude (SNAP) for ATTRv(Month 24 and 30 after the start of treatment (data collected and analyzed over 22 days))
  • Change in NIS for ATTRwt(Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days))
  • Change in NIS-LL for ATTRwt(Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days))
  • Number of Participants With R-R Interval Variability for ATTRv(Month 18, 24 and 30 (data collected and analyzed over 22 days))
  • Number of Participants With R-R Interval Variability for ATTRwt(Month 18, 24 and 30 (data collected and analyzed over 22 days))
  • mBMI for ATTRwt(Month 18, 24 and 30 after start of treatment (data collected and analysed over 22 days))
  • Ulnar/Sural SNAP Score for ATTRwt(Month 24 and 30 after the start of treatment (data collected and analyzed over 22 days))
  • Ulnar/Peroneal CMAP Score for ATTRwt(Month 24 and 30 after the start of treatment (data collected and analyzed over 22 days))
  • Number of Participants With Carpal Tunnel Syndrome(At baseline (data collected and analyzed over 22 days))
  • Number of Participants With Lumbar Stenosis(At baseline (data collected and analyzed over 22 days))
  • Modified Body Mass Index (mBMI) for ATTRv(Month 18, 24 and 30 after start of treatment (data collected and analysed over 22 days))
  • Ulnar/Peroneal Compound Muscle Action Potential Amplitude (CMAP) for ATTRv(Month 24 and 30 after the start of treatment (data collected and analyzed over 22 days))
  • Percentage of Participants Who do Not Have Stage Progression in the PND Score for ATTRwt(From Baseline to Month 30 (data collected and analysed over 22 days))
  • Percentage of Responders to Treatment for ATTRwt(Month 12 (data collected and analyzed over 22 days))
  • Change in Norfolk QOL-DN for ATTRwt(Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days))
  • Change in COMPASS-31 for ATTRwt(Baseline, Month 6, 12, 18, 24 and 36 after treatment initiation)
  • Change in FAP-RODs for ATTRwt(Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days))
  • Number of Participants According to FAP Stage for ATTRwt(Month 18, 24 and 30 months after the start of treatment (data collected and analyzed over 22 days))
  • Number of Participants With Gastrointestinal Disturbances(Month 18, 24 and 30 after the start of treatment (data collected and analyzed over 22 days))
  • Number of Participants With Unintentional Weight Loss(Month 18, 24 and 30 after the start of treatment (data collected and analyzed over 22 days))
  • Number of Participants With Urological Disturbances(Month 18, 24 and 30 after the start of treatment (data collected and analyzed over 22 days))
  • Number of Participants With Ophthalmological Disturbances(Month 18, 24 and 30 after the start of treatment (data collected and analyzed over 22 days))
  • Number of Participants With Central Nervous System (CNS) Disturbances(Month 18, 24 and 30 after the start of treatment (data collected and analyzed over 22 days))

Investigators

Sponsor
Pfizer
Sponsor Class
Industry
Responsible Party
Sponsor

Study Sites (3)

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