Retrospective Study Collecting Neurological Follow-up of Hereditary Transthyretin Amyloidosis (ATTRv) Patients Included in B3461028 and B3461045. (TRAMA)

Tafamidis 61mg, Resultados en Amiloidosis ATTR Con afectación neurológica y Multisistémica - 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/ophtalmo/gastrointestinal) ≥12 months.

Study Overview

• Status: Completed
• Conditions: Polyneuropathy; Hereditary Transthyretin Amyloidosis (ATTRv)
• Intervention / Treatment: Drug: Tafamidis

• Study Type: Observational
• Enrollment (Actual): 5

Contacts and Locations

Study Locations

• Spain
  • Barcelona, Spain, 08907
    • Hospital Universitari de Bellvitge
  • Madrid, Spain, 28040
    • Hospital Clinico San Carlos
  • Palma de Mallorca, Spain, 07198
    • Hospital Son Llatzer

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 99 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• 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/ophtalmo/gastrointestinal) ≥12 months

Description

Inclusion Criteria:

• Treatment with tafamidis 61 mg ≥ 12 months
• Neurological follow up ≥ 12 months
• Diagnosis of transthyretin amyloidosis with polyneuropayhy (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 Sudoscan, RR Interval análisis, etc..)

Exclusion Criteria:

• Treatment with tafamidis 61 mg < 12 months
• Neurological follow up < 12 months
• Other diagnosis for polyneuropathy

Study Plan

How is the study designed?

Design Details

• Observational Models: Other
• Time Perspectives: Retrospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Retrospective cohort ATTRv and ATTRwt patients enrolled in B3461028 and B3461045 studies in Spain	Drug: Tafamidis; 61 milligrams (mg) as received in studies B3461028 and B3461045

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Change from Baseline in Neurological Impairment score (NIS)	Baseline, Month 12

Secondary Outcome Measures

Outcome Measure	Time Frame
To describe extra-cardiac clinical features and clinical management of the Spanish ATTRv and ATTRwt patients with mixed phenotype treated with tafamidis 61 mg.	Baseline through month 36
Change From Baseline in Neuropathy Impairment Score - Lower Limbs [NIS (LL)	Baseline through Month 36
Change from Baseline in Norfolk Quality of Life- Diabetic Neuropathy (Norfolk QOL-DN)	Baseline through month 36
Change From Baseline in composite autonomic symptom scale 31 (Compass-31)	Baseline through month 36
Change from Baseline in Modified Body Mass Index (mBMI)	Baseline through month 36
Change from Baseline in Sudomotor Response	Baseline through month 36

Collaborators and Investigators

• Sponsor: Pfizer

Publications and helpful links

Helpful Links

• To obtain contact information for a study center near you, click here.

Study record dates

Study Major Dates

• Study Start (Actual): October 24, 2022
• Primary Completion (Actual): November 15, 2022
• Study Completion (Actual): November 15, 2022

Study Registration Dates

• First Submitted: September 26, 2022
• First Submitted That Met QC Criteria: September 26, 2022
• First Posted (Actual): September 29, 2022

Study Record Updates

• Last Update Posted (Actual): December 27, 2022
• Last Update Submitted That Met QC Criteria: December 23, 2022
• Last Verified: December 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Neuromuscular Diseases; Neurodegenerative Diseases; Peripheral Nervous System Diseases; Proteostasis Deficiencies; Metabolism, Inborn Errors; Heredodegenerative Disorders, Nervous System; Amyloidosis, Familial; Amyloid Neuropathies; Amyloidosis; Polyneuropathies; Amyloid Neuropathies, Familial
• Other Study ID Numbers: B3461104; TRAMA (Other Identifier: Alias Study Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No
• IPD Plan Description: 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.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No