Hereditary Transthyretin Amyloidosis (ATTRv)TRAMANCT05560555
Retrospective Study Collecting Neurological Follow-up of Hereditary Transthyretin Amyloidosis (ATTRv) Patients Included in B3461028 and B3461045.
An observational study of Hereditary Transthyretin Amyloidosis (ATTRv) and Polyneuropathy, run by Pfizer at 3 sites in Spain.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hereditary Transthyretin Amyloidosis (ATTRv), Polyneuropathy
- What is being tested?
- Tafamidis
- How many people
- 5 participants
- Where
- 3 sitesSpain
- Study dates
- 24 Oct 2022to 15 Nov 2022
- Phase
- Observational
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
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
Who may be able to join
- 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..)
Who cannot join
- Treatment with tafamidis 61 mg < 12 months
- Neurological follow up < 12 months
- Other diagnosis for polyneuropathy
Trial locations and contacts
3 sites in 1 country.
Spain3 sites
Hospital Universitari de Bellvitge
Barcelona, 08907
Hospital Clinico San Carlos
Madrid, 28040
Hospital Son Llatzer
Palma de Mallorca, 07198
About this study
Official title: Tafamidis 61mg, Outcomes in ATTR Amyloidosis With Neurologic and Multisystemic Involvement - TRAMA
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.
- Being tested
- Tafamidis
- Medical subject headings
- Amyloidosis, Hereditary, Transthyretin-Related, Polyneuropathies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TafamidisDrug
61 milligrams (mg) as received in studies B3461028 and B3461045
| Study group | Role | What they receive |
|---|---|---|
| Retrospective cohort ATTRv and ATTRwt patients enrolled in B3461028 and B3461045 studies in Spain | Not described Receives: Drug: Tafamidis |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Neuropathy Impairment Score (NIS) at Month 12 for ATTRv
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.
Measured: Baseline and Month 12 (data collected and analyzed over 22 days)
Secondary outcomes (33)
Change in NIS for ATTRv
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.
Measured: Baseline, Month 6, 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
NIS-LL (Neuropathy Impairment Score Lower Limbs) is a clinically important, sensitive measure of neurological function in individuals, assessing sensory function, reflexes, and muscle weakness of the lower limbo. NÍS-LL assessed muscle weakness, reflexes, sensation. Each item is scored separately for left and right limbs. Components of muscle weakness:0(normal) to4(paralysis), higher score=more weakness; reflexes, sensation:0=normal, 1=decreased, or 2=absent. Total NIS-LL score range 0-88, higher score=more impairment. The rate of change was calculated from last follow-up.
Measured: 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
Norfolk Quality of Life Questionnaire for Diabetic Neuropathy is a standardized and validated instrument that assesses the effect of polyneuropathy on the functionality and quality of life of the individual. Norfolk QOL-DN: 35-item participant-rated questionnaire is used to assess impact of diabetic neuropathy on the quality of life of participants with diabetic neuropathy; Item 1 to 7: related to symptoms and presence of symptom was assessed as 1 and absence was assessed as 0. Item 8-35: related to activities of daily living and scored on a 5-point Likert scale, where 0= no problem and 4= severe problem (except item 32, where -2= much better, 0=about the same, 2=much worse). TQOL= sum of all the items, total possible score range= -4 to 138, where higher score=worse quality of life.
Measured: 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 ATTRv
COMPASS-31 (Composite Autonomic Symptom Score 31) is a questionnaire designed to assess the severity and functional ability in participants with autonomic dysfunction. COMPASS-31 total score ranged from 0 to 100; where 0=Lesser severity of dysautonomia and 100=Greater severity of dysautonomia.
Measured: Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days)
Change in Familial Amyloid Polyneuropathy Specific Rasch-Built Overall Disability Scale (FAP-RODs) for ATTRv
FAP-RODS is a questionnaire that assessed the effect of neuropathy on daily activities. FAP-RODS total score ranged from 0 to 68; where, 0=Lower ability to perform daily activities and 68=Greater ability to perform daily activities.
Measured: 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
FAP is a stage system based on symptom severity and disease progression. FAP stages included: Asymptomatic; Free ambulation (walking without support): stage 1; Supportive ambulation (walking with support): stage 2; Wheelchair-bound or bedridden: stage 3.
Measured: Month 18, 24 and 30 months 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
PND is a staging system that assess the degree of neuropathic dysfunction and its impact on ambulation. PND stages included: Asymptomatic; Stage I: Sensory disturbances, normal gait; Stage II: Sensory disturbances, altered gait not requiring support; IIIA: Gait requiring one support; IIIB: Gait requiring two supports; IV: Wheelchair or bedside. Participants who did not change to higher stages compared to the start of treatment was reported as Unchanged and those who progressed to higher stages were reported under Staging up.
Measured: From Baseline to Month 30 (data collected and analyzed over 22 days)
Percentage of Responders to Treatment for ATTRv
Percentage of responders to treatment at Month 12 was defined as participants who achieved the change from baseline of less than 4 points in the NIS and participants who achieved the change from baseline of less than 2 points in the NIS-LL were reported in this outcome measure.
Measured: Month 12 (data collected and analyzed over 22 days)
Number of Participants With R-R Interval Variability for ATTRv
Number of participants with R-R interval variability (altered/unaltered) was reported in this outcome measure.
Measured: Month 18, 24 and 30 (data collected and analyzed over 22 days)
Modified Body Mass Index (mBMI) for ATTRv
BMI was calculated by weight divided by height squared and measured as kilogram per square meter (kg/m^2). mBMI was calculated by multiplying BMI by serum albumin levels [gram/liter (g/L)].
Measured: Month 18, 24 and 30 after start of treatment (data collected and analysed over 22 days)
Ulnar/Sural Sensory Nerve Action Potential Amplitude (SNAP) for ATTRv
Sural sensory nerve action potential amplitude (in microvolts) was measured using electromyography of the left lower limb.
Measured: Month 24 and 30 after the start of treatment (data collected and analyzed over 22 days)
Ulnar/Peroneal Compound Muscle Action Potential Amplitude (CMAP) for ATTRv
Peroneal motor nerve compound muscle action potential amplitude was measured using electromyography of the left lower limb.
Measured: Month 24 and 30 after the start of treatment (data collected and analyzed over 22 days)
Change in NIS for ATTRwt
NIS (Neuropathy Impairment Score) was 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.
Measured: 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
NIS-LL (Neuropathy Impairment Score Lower Limbs) was clinically important, sensitive measure of neurological function in individuals, assessing sensory function, reflexes, and muscle weakness of the lower limbo. NÍS-LL assessed muscle weakness, reflexes, sensation. Each item scored separately for left and right limbs. Components of muscle weakness:0(normal) to4(paralysis), higher score=more weakness; reflexes, sensation:0=normal, 1=decreased, or 2=absent. Total NIS-LL score range 0-88, higher score=more impairment. The rate of change was calculated from last follow-up.
Measured: Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days)
Change in Norfolk QOL-DN for ATTRwt
Norfolk Quality of Life Questionnaire for Diabetic Neuropathy was a standardized and validated instrument that assesses the effect of polyneuropathy on the functionality and quality of life of the individual. Norfolk QOL-DN: 35-item participant-rated questionnaire used to assess impact of diabetic neuropathy on the quality of life of participants with diabetic neuropathy; Item 1 to 7: related to symptoms and presence of symptom was assessed as 1 and absence was assessed as 0. Item 8-35: related to activities of daily living and scored on a 5-point Likert scale, where 0= no problem and 4= severe problem (except item 32, where -2= much better, 0=about the same, 2=much worse). TQOL= sum of all the items, total possible score range= -4 to 138, where higher score=worse quality of life.
Measured: 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
COMPASS-31 (Composite Autonomic Symptom Score 31) was a questionnaire designed to assess the severity and functional ability in participants with autonomic dysfunction. COMPASS-31 total score ranged from 0 to 100; where 0=Lesser severity of dysautonomia and 100=Greater severity of dysautonomia.
Measured: Baseline, Month 6, 12, 18, 24 and 36 after treatment initiation
Change in FAP-RODs for ATTRwt
FAP-RODS is a questionnaire that assessed the effect of neuropathy on daily activities. FAP-RODS total score ranged from 0 to 68; where, 0=Lower ability to perform daily activities and 68=Greater ability to perform daily activities.
Measured: 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
FAP stage is a staging system based on symptom severity and disease progression. FAP stages included: Asymptomatic; Free ambulation (Walking without support): stage 1; Supportive ambulation (Walking with support): stage 2; Wheelchair-bound or bedridden: stage 3.
Measured: Month 18, 24 and 30 months 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
PND was a simple staging system according to the degree of neuropathic dysfunction and its impact on ambulation. PND stages included: Asymptomatic: I Sensory disturbances, normal gait: II Sensory disturbances, altered gait not requiring support: IIIA Gait requiring one support: IIIB Gait requiring two supports: IV Wheelchair or bedside). Participants who did not change to higher stages compared to the start of treatment was reported as Unchanged and those who progressed to higher stages were reported under Staging up.
Measured: From Baseline to Month 30 (data collected and analysed over 22 days)
Percentage of Responders to Treatment for ATTRwt
Percentage of responders to treatment was defined as participants who achieved the change from baseline of less than 4 points in the NIS and participants who achieved the change from baseline of less than 2 points in the NIS-LL were reported in this outcome measure.
Measured: Month 12 (data collected and analyzed over 22 days)
Number of Participants With R-R Interval Variability for ATTRwt
Number of participants with R-R interval variability (altered/unaltered) was reported in this outcome measure.
Measured: Month 18, 24 and 30 (data collected and analyzed over 22 days)
mBMI for ATTRwt
BMI was calculated by weight divided by height squared and measured as kilogram per square meter (kg/m^2).
Measured: Month 18, 24 and 30 after start of treatment (data collected and analysed over 22 days)
Ulnar/Sural SNAP Score for ATTRwt
Sural sensory nerve action potential amplitude (in microvolts) was measured using electromyography of the left lower limb.
Measured: Month 24 and 30 after the start of treatment (data collected and analyzed over 22 days)
Ulnar/Peroneal CMAP Score for ATTRwt
Peroneal motor nerve compound muscle action potential amplitude (in millivolts) was measured using electromyography of the left lower limb.
Measured: Month 24 and 30 after the start of treatment (data collected and analyzed over 22 days)
Number of Participants With Carpal Tunnel Syndrome
Number of participants with carpal tunnel syndrome were reported in this outcome measure.
Measured: At baseline (data collected and analyzed over 22 days)
Number of Participants With Lumbar Stenosis
Number of participants with lumbar stenosis were reported in this outcome measure.
Measured: At baseline (data collected and analyzed over 22 days)
Number of Participants With Gastrointestinal Disturbances
Number of participants with gastrointestinal disturbances were reported in this outcome measure.
Measured: Month 18, 24 and 30 after the start of treatment (data collected and analyzed over 22 days)
Number of Participants With Unintentional Weight Loss
Number of participants with unintentional weight loss were reported in this outcome measure.
Measured: Month 18, 24 and 30 after the start of treatment (data collected and analyzed over 22 days)
Number of Participants With Urological Disturbances
Number of participants with urological disturbances were reported in this outcome measure.
Measured: Month 18, 24 and 30 after the start of treatment (data collected and analyzed over 22 days)
Number of Participants With Ophthalmological Disturbances
Number of participants with ophthalmological disturbances were reported in this outcome measure.
Measured: 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
Number of participants with CNS disturbances were reported in this outcome measure.
Measured: Month 18, 24 and 30 after the start of treatment (data collected and analyzed over 22 days)
Number of Participants With Symptoms of Autonomic Neuropathy
Number of participants with symptoms of autonomic neuropathy including impaired sweating, sexual dysfunction, orthostatic hypotension were reported in this outcome measure.
Measured: 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
Number of participants with symptoms of peripheral neuropathy (allodynia and paresthesia) were reported in this outcome measure.
Measured: At baseline (data collected and analyzed over 22 days)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Sep 2022
- Study start24 Oct 2022
- Primary completion15 Nov 2022
- Study completion15 Nov 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Hereditary Transthyretin Amyloidosis (ATTRv)Studies of the same condition, open and under way.
- More trials run by PfizerThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Retrospective
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pfizer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Pfizer CT.gov Call Center, Study director, Pfizer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05560555
- Sponsor's protocol ID
- B3461104
- Other IDs
- TRAMA
- Acronym
- TRAMA
- Status last verified
- Nov 2023
- First submitted
- 26 Sep 2022
- Last updated
- 13 May 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical\_trials/trial\_data\_and\_results/data\_requests.
Study at a glance
From the registry record, last updated by the sponsor on 13 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Tafamidis 61mg, Outcomes in ATTR Amyloidosis With Neurologic and Multisystemic Involvement - TRAMA
- ClinicalTrials.gov ID
- NCT05560555
- Acronym / sponsor ID
- TRAMA / B3461104
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- PfizerIndustry (pharma, biotech, devices)
- Study start
- 24 Oct 2022 (actual)
- Primary completion
- 15 Nov 2022 (actual)
- Study completion
- 15 Nov 2022 (actual)
- First posted
- 29 Sep 2022 (actual)
- Results
- Posted 13 May 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
- Observational model
- Other
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Retrospective cohort ATTRv and ATTRwt patients enrolled in B3461028 and B3461045 studies in Spain | – | Drug: Tafamidis |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- 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
Key inclusion criteria (4 listed)
- 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..)
Key exclusion criteria (3 listed)
- Treatment with tafamidis 61 mg < 12 months
- Neurological follow up < 12 months
- Other diagnosis for polyneuropathy
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Tafamidis61 milligrams (mg) as received in studies B3461028 and B3461045 | Drug | Retrospective cohort ATTRv and ATTRwt patients enrolled in B3461028 and B3461045 studies in Spain |
Primary outcome
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.
33 secondary outcomes
Change in NIS for ATTRv
Time frame: Baseline, Month 6, 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
Time frame: 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
Time frame: 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 ATTRv
Time frame: Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days)
Change in Familial Amyloid Polyneuropathy Specific Rasch-Built Overall Disability Scale (FAP-RODs) for ATTRv
Time frame: 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
Time frame: Month 18, 24 and 30 months 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
Time frame: From Baseline to Month 30 (data collected and analyzed over 22 days)
Percentage of Responders to Treatment for ATTRv
Time frame: Month 12 (data collected and analyzed over 22 days)
Number of Participants With R-R Interval Variability for ATTRv
Time frame: Month 18, 24 and 30 (data collected and analyzed over 22 days)
Modified Body Mass Index (mBMI) for ATTRv
Time frame: Month 18, 24 and 30 after start of treatment (data collected and analysed over 22 days)
Ulnar/Sural Sensory Nerve Action Potential Amplitude (SNAP) for ATTRv
Time frame: Month 24 and 30 after the start of treatment (data collected and analyzed over 22 days)
Ulnar/Peroneal Compound Muscle Action Potential Amplitude (CMAP) for ATTRv
Time frame: Month 24 and 30 after the start of treatment (data collected and analyzed over 22 days)
Change in NIS for ATTRwt
Time frame: 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
Time frame: Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days)
Change in Norfolk QOL-DN for ATTRwt
Time frame: 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
Time frame: Baseline, Month 6, 12, 18, 24 and 36 after treatment initiation
Change in FAP-RODs for ATTRwt
Time frame: 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
Time frame: Month 18, 24 and 30 months 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
Time frame: From Baseline to Month 30 (data collected and analysed over 22 days)
Percentage of Responders to Treatment for ATTRwt
Time frame: Month 12 (data collected and analyzed over 22 days)
Number of Participants With R-R Interval Variability for ATTRwt
Time frame: Month 18, 24 and 30 (data collected and analyzed over 22 days)
mBMI for ATTRwt
Time frame: Month 18, 24 and 30 after start of treatment (data collected and analysed over 22 days)
Ulnar/Sural SNAP Score for ATTRwt
Time frame: Month 24 and 30 after the start of treatment (data collected and analyzed over 22 days)
Ulnar/Peroneal CMAP Score for ATTRwt
Time frame: Month 24 and 30 after the start of treatment (data collected and analyzed over 22 days)
Number of Participants With Carpal Tunnel Syndrome
Time frame: At baseline (data collected and analyzed over 22 days)
Number of Participants With Lumbar Stenosis
Time frame: At baseline (data collected and analyzed over 22 days)
Number of Participants With Gastrointestinal Disturbances
Time frame: Month 18, 24 and 30 after the start of treatment (data collected and analyzed over 22 days)
Number of Participants With Unintentional Weight Loss
Time frame: Month 18, 24 and 30 after the start of treatment (data collected and analyzed over 22 days)
Number of Participants With Urological Disturbances
Time frame: Month 18, 24 and 30 after the start of treatment (data collected and analyzed over 22 days)
Number of Participants With Ophthalmological Disturbances
Time frame: 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
Time frame: Month 18, 24 and 30 after the start of treatment (data collected and analyzed over 22 days)
Number of Participants With Symptoms of Autonomic Neuropathy
Time frame: 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
Time frame: At baseline (data collected and analyzed over 22 days)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 May 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- To obtain contact information for a study center near you, click here.Link provided by the sponsor.
- Individual participant dataSharing planned: No. Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Pfizer CT.gov Call Center study director
Pfizer
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hereditary Transthyretin Amyloidosis (ATTRv) and Polyneuropathy, run by Pfizer at 3 sites in Spain.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 13 May 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Spain. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 13 May 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Neuropathy Impairment Score (NIS) at Month 12 for ATTRv (time frame: Baseline and Month 12 (data collected and analyzed over 22 days)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 May 2024; AzTrials last refreshed its copy on 8 Nov 2026.