Clinical trial NCT05560555: Retrospective Study Collecting Neurological Follow-up of Hereditary Transthyretin Amyloidosis (ATTRv) Patients Included in B3461028 and B3461045.
Official title: Tafamidis 61mg, Outcomes in ATTR Amyloidosis With Neurologic and Multisystemic Involvement - TRAMA
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hereditary Transthyretin Amyloidosis (ATTRv)
Polyneuropathy
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2022-09-29
Enrollment: 5
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Study population: retrospective cohort 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 have kept going to their multisystemic follow-ups (neuro/ophthalmo/gastrointestinal) ≥12 months

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.


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

Primary outcomes:
measure: Change in Neuropathy Impairment Score (NIS) at Month 12 for ATTRv
timeFrame: Baseline and Month 12 (data collected and analyzed over 22 days)
Secondary outcomes:
measure: Change in NIS for ATTRv
timeFrame: Baseline, Month 6, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days)
measure: Change in Neuropathy Impairment Score - Lower Limbs (NIS-LL) for ATTRv
timeFrame: Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days)
measure: Change in Norfolk Quality of Life- Diabetic Neuropathy (Norfolk QOL-DN) for ATTRv
timeFrame: Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days)
measure: Change in COMPASS-31 for ATTRv
timeFrame: Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days)
measure: Change in Familial Amyloid Polyneuropathy Specific Rasch-Built Overall Disability Scale (FAP-RODs) for ATTRv
timeFrame: Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days)
measure: Number of Participants According to Familiar Amyloidotic Polyneuropathy Stage (FAP) for ATTRv
timeFrame: Month 18, 24 and 30 months after the start of treatment (data collected and analyzed over 22 days)
measure: Percentage of Participants Who do Not Have Stage Progression in the PND Score for ATTRv
timeFrame: From Baseline to Month 30 (data collected and analyzed over 22 days)
measure: Percentage of Responders to Treatment for ATTRv
timeFrame: Month 12 (data collected and analyzed over 22 days)
measure: Number of Participants With R-R Interval Variability for ATTRv
timeFrame: Month 18, 24 and 30 (data collected and analyzed over 22 days)
measure: Modified Body Mass Index (mBMI) for ATTRv
timeFrame: Month 18, 24 and 30 after start of treatment (data collected and analysed over 22 days)
measure: Ulnar/Sural Sensory Nerve Action Potential Amplitude (SNAP) for ATTRv
timeFrame: Month 24 and 30 after the start of treatment (data collected and analyzed over 22 days)
measure: Ulnar/Peroneal Compound Muscle Action Potential Amplitude (CMAP) for ATTRv
timeFrame: Month 24 and 30 after the start of treatment (data collected and analyzed over 22 days)
measure: Change in NIS for ATTRwt
timeFrame: Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days)
measure: Change in NIS-LL for ATTRwt
timeFrame: Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days)
measure: Change in Norfolk QOL-DN for ATTRwt
timeFrame: Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days)
measure: Change in COMPASS-31 for ATTRwt
timeFrame: Baseline, Month 6, 12, 18, 24 and 36 after treatment initiation
measure: Change in FAP-RODs for ATTRwt
timeFrame: Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days)
measure: Number of Participants According to FAP Stage for ATTRwt
timeFrame: Month 18, 24 and 30 months after the start of treatment (data collected and analyzed over 22 days)
measure: Percentage of Participants Who do Not Have Stage Progression in the PND Score for ATTRwt
timeFrame: From Baseline to Month 30 (data collected and analysed over 22 days)
measure: Percentage of Responders to Treatment for ATTRwt
timeFrame: Month 12 (data collected and analyzed over 22 days)
measure: Number of Participants With R-R Interval Variability for ATTRwt
timeFrame: Month 18, 24 and 30 (data collected and analyzed over 22 days)
measure: mBMI for ATTRwt
timeFrame: Month 18, 24 and 30 after start of treatment (data collected and analysed over 22 days)
measure: Ulnar/Sural SNAP Score for ATTRwt
timeFrame: Month 24 and 30 after the start of treatment (data collected and analyzed over 22 days)
measure: Ulnar/Peroneal CMAP Score for ATTRwt
timeFrame: Month 24 and 30 after the start of treatment (data collected and analyzed over 22 days)
measure: Number of Participants With Carpal Tunnel Syndrome
timeFrame: At baseline (data collected and analyzed over 22 days)
measure: Number of Participants With Lumbar Stenosis
timeFrame: At baseline (data collected and analyzed over 22 days)
measure: Number of Participants With Gastrointestinal Disturbances
timeFrame: Month 18, 24 and 30 after the start of treatment (data collected and analyzed over 22 days)
measure: Number of Participants With Unintentional Weight Loss
timeFrame: Month 18, 24 and 30 after the start of treatment (data collected and analyzed over 22 days)
measure: Number of Participants With Urological Disturbances
timeFrame: Month 18, 24 and 30 after the start of treatment (data collected and analyzed over 22 days)
measure: Number of Participants With Ophthalmological Disturbances
timeFrame: Month 18, 24 and 30 after the start of treatment (data collected and analyzed over 22 days)
measure: Number of Participants With Central Nervous System (CNS) Disturbances
timeFrame: Month 18, 24 and 30 after the start of treatment (data collected and analyzed over 22 days)
measure: Number of Participants With Symptoms of Autonomic Neuropathy
timeFrame: Month 18, 24 and 30 after the start of treatment (data collected and analyzed over 22 days)
measure: Number of Participants With Symptoms of Peripheral Neuropathy
timeFrame: At baseline (data collected and analyzed over 22 days)
Other outcomes:
Locations:
city: Barcelona
country: Spain
facility: Hospital Universitari de Bellvitge
city: Madrid
country: Spain
facility: Hospital Clinico San Carlos
city: Palma de Mallorca
country: Spain
facility: Hospital Son Llatzer
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: