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

• ClinicalTrials.gov Identifier: NCT05560555
• Recruitment Status: Completed
• First Posted: September 29, 2022
• Last Update Posted: December 27, 2022
• Sponsor: Pfizer
• Information provided by (Responsible Party): Pfizer

Study Description

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/ophtalmo/gastrointestinal) ≥12 months.

Condition or disease	Intervention/treatment
Hereditary Transthyretin Amyloidosis (ATTRv); Polyneuropathy	Drug: Tafamidis

Study Design

• Study Type: Observational
• Actual Enrollment: 5 participants
• Observational Model: Other
• Time Perspective: Retrospective
• Official Title: Tafamidis 61mg, Resultados en Amiloidosis ATTR Con afectación neurológica y Multisistémica - TRAMA
• Actual Study Start Date: October 24, 2022
• Actual Primary Completion Date: November 15, 2022
• Actual Study Completion Date: November 15, 2022

Groups and Cohorts

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

Outcome Measures

Primary Outcome Measures :

1. Change from Baseline in Neurological Impairment score (NIS) [ Time Frame: Baseline, Month 12 ]

Secondary Outcome Measures :

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

Eligibility Criteria

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

Criteria

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

Contacts and Locations

Locations

Spain

Hospital Universitari de Bellvitge

Barcelona, Spain, 08907

Hospital Clinico San Carlos

Madrid, Spain, 28040

Hospital Son Llatzer

Palma de Mallorca, Spain, 07198

Sponsors and Collaborators

Pfizer

Investigators

• Study Director: Pfizer CT.gov Call Center; Pfizer

More Information

Additional Information:

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

• Responsible Party: Pfizer
• ClinicalTrials.gov Identifier: NCT05560555
• Other Study ID Numbers: B3461104; TRAMA ( Other Identifier: Alias Study Number )
• First Posted: September 29, 2022
• Last Update Posted: December 27, 2022
• Last Verified: December 2022

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No
• 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.

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Polyneuropathies; Amyloid Neuropathies, Familial; Amyloidosis; Proteostasis Deficiencies; Metabolic Diseases; Peripheral Nervous System Diseases; Neuromuscular Diseases; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Amyloid Neuropathies; Genetic Diseases, Inborn; Amyloidosis, Familial; Metabolism, Inborn Errors